BridgeBio Pharma Grows Pipeline to 20+ Genetic Medicines with Four Assets Focused on Treating Genetically Driven Diseases at Their Source

New assets span multiple modalities and target an endocrine disorder, inherited eye disease, genetically driven deafness and autism spectrum disorder

SAN FRANCISCO – January 13, 2020 – BridgeBio Pharma, Inc. (NASDAQ: BBIO) today disclosed four additional assets in its pipeline, expanding the number of disclosed programs to over 20 potential medicines. BridgeBio’s world-class team of experienced drug developers are advancing these therapies with the aim of delivering life-changing medicines to patients. BridgeBio founder and CEO Neil Kumar, Ph.D., will discuss these new assets and BridgeBio’s pipeline during a presentation at the 38th Annual J.P. Morgan Healthcare Conference today at 3 PM (PST) in San Francisco.

The presentation will be webcast live and can be accessed at www.bridgebio.com on the For Investors page under News & Events or here.

“The range of disorders driven by genetic mutations is vast, and our increasing understanding of the biology of such conditions is allowing us to better engineer potential therapies designed to alleviate genetically driven diseases,” said Dr. Kumar, CEO of BridgeBio. “We are excited to unveil these four programs we’ve had the privilege of working on, taking advantage of research done by companies and academic investigators on the leading edge of scientific discovery. Our teams of experts in cardiovascular and renal disease, gene therapy, and ophthalmology identified these opportunities and have been working to advance them, in some cases for more than a year.”

Encaleret is a small molecule antagonist of the calcium sensing receptor targeting conditions related to hypoparathyroidism including Autosomal Dominant Hypocalcemia Type 1 (ADH1). Individuals with ADH1 typically have low serum calcium and high urine calcium due to gain-of-function mutations in the calcium sensing receptor. BridgeBio completed the submission of an Investigational New Drug (IND) application to the US Food and Drug Administration in late 2019 to support initial development of encaleret. A Phase 2b study of encaleret in ADH1 is planned to initiate in early 2020 at the National Institutes of Health (NIH) and expected to provide proof-of-concept data in 2021. Encaleret is housed in BridgeBio subsidiary Calcilytix Therapeutics. 

BBP-551 is a novel treatment for the genetically determined retinal diseases phenotypically classified as Retinitis Pigmentosa and Leber’s Congenital Amaurosis and genotypically caused by mutations of Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransferase (LRAT).   Each of these inborn errors of metabolism disrupts key steps within the visual cycle in regenerating 11-cis-retinal, leading to a deficiency in the molecule necessary for rhodopsin formation and subsequent visual signal transduction.  Both lead to progressive visual loss and retinal degeneration and, eventually, blindness.  The therapy originated in the lab of Dr. Krzysztof Palczewski, Ph.D. and was advanced by David Saperstein, M.D., both at the University of Washington.  BBP-551 has been granted Orphan Drug Designation in the United States and European Union and Fast Track Designation in the United States. BBP-551 is housed in BridgeBio subsidiary Retinagenix Therapeutics.

BBP-815 is an AAV gene therapy for the treatment for nonsyndromic hearing loss caused by recessive mutations in the TMC1 gene. TMC1 encodes the mechanosensory ion channel that converts sound vibrations in the inner ear into electrical signals in sensory hair cells. Mutations in the TMC1 gene prevent sound from eliciting the appropriate electrical response in the hair cells, resulting in moderate to severe hearing loss, often present early in life. By replacing the dysfunctional TMC1 protein using AAV gene therapy, BBP-815 is aiming to repair the deficient inner ear sensory cells at the source of this disease. The initial work for this treatment was performed by Jeffrey Holt, Ph.D, Professor of Otolaryngology and Neurology at Boston Children’s Hospital and Harvard Medical School. Dr. Holt is a world expert in hearing loss gene therapy and elucidated TMC1’s role in hearing. BBP-815 is housed in BridgeBio subsidiary Audition Therapeutics.

BBP-472 is a novel treatment designed to balance kinase signaling in the brain for the treatment of children with autism-spectrum disorders (ASD) characterized by loss of the PTEN protein. This program, currently in the lead-finding phase, is focused on advancing a brain-permeable inhibitor of PI3KB, a kinase shown to signal unabatedly in the absence of PTEN. Although PI3KB inhibitors designed to stay out of the brain are being tested experimentally for cancer indications, a PI3KB inhibitor engineered for brain penetrance has not been advanced to date, representing a major unmet need for this pediatric population. 

About BridgeBio Pharma

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, visit bridgebio.com.

BridgeBio Pharma Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to expectations, plans and prospects regarding the preclinical and clinical development plans, clinical trial designs, clinical and therapeutic potential, and strategy of BridgeBio’s product candidates, including, but not limited to, encaleret, BBP-551, BBP-815 and BBP-472, BridgeBio’s ability to continue to rapidly identify and develop therapeutic innovations, the timing and acceptance of Calcilytix’s Investigational New Drug (IND) application for encaleret, BridgeBio’s subsidiaries’ ability to advance their product candidates through clinical development, the timing and expected results of the Phase 2b study of encaleret in ADH1, BridgeBio’s subsidiaries’ ability to maintain current and enter into new collaborations with partners, including Calcilytix’s partnership with the National Institutes of Health, the ability of BBP-551 to retain Orphan Drug Designation in the United States and European Union and Fast Track Designation in the United States, and the timing of these events, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by  a number of risks, uncertainties and assumptions, including, but not limited to, the estimated therapeutic potential of BridgeBio’s subsidiaries’ product candidates, the timing and acceptance of Calcilytix’s IND for encaleret, the timing and success of the Phase 2b study of encaleret in ADH1, Calcilytix’s ability to maintain and derive benefits from its partnership with the NIH, and BridgeBio’s ability to support its subsidiaries’ product candidates and portfolios and advance their product candidates through clinical development, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma, Inc.’s most recent Quarterly Report on Form 10-Q and our other SEC filings. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Media Contact:
Grace Rauh, BridgeBio Pharma
grace.rauh@bridgebio.com
917-232-5478

Investor Contact:
John Grimaldi, Burns McClellan
jgrimaldi@burnsmc.com
212-213-0006 x362

Source: BridgeBio Pharma

BridgeBio Pharma’s Gene Therapy Subsidiaries Enter Strategic Partnership with Catalent for Dedicated Gene Therapy Development and Manufacturing Capacity

  • The deal is designed to accelerate BridgeBio’s gene therapy programs
  • The dedicated suite will support clinical and commercial supply needs for BridgeBio’s gene therapy programs for congenital adrenal hyperplasia (BBP-631) and Canavan disease (BBP-812)

PALO ALTO, Calif. – January 10, 2020 – BridgeBio Pharma, Inc. (NASDAQ: BBIO) today announced a collaboration agreement with Catalent to establish dedicated gene therapy development and manufacturing capacity at Catalent’s Paragon Gene Therapy clinical and commercial manufacturing center in Harmans, Maryland. The agreement is intended to support the clinical and commercial manufacturing needs for BridgeBio’s gene therapy product candidates for congenital adrenal hyperplasia, BBP-631, and Canavan disease, BBP-812.

Catalent’s commercial facility is fully compliant with cGMP requirements and allows for up to 5000 liters of production. The over 400,000 square feet footprint is complete with all necessary support functions for storage and fill finish for final product supply.

“Having flexibility and greater certainty in manufacturing capacity is critical to success in gene therapy,” said Eric David, M.D., J.D., CEO of BridgeBio’s gene therapy subsidiaries. “Catalent’s Paragon Gene Therapy arm has been our trusted partner for almost two years, and this expansion of our relationship is intended to allow for smoother clinical and commercial development, as well as an acceleration of our pipeline programs, helping us move faster to address critical unmet health needs for patients and their families.”

“Catalent’s expertise in the cGMP manufacturing of viral vectors complements our internal investment in the CMC process and analytical development to support our gene therapy portfolio,” said Fred Porter, Ph.D., senior vice president of CMC and technical development of BridgeBio’s gene therapy subsidiaries. “Securing dedicated capacity for the delivery of clinical and commercial supply is critical to our long-term strategy.”

Pete Buzy, president of Paragon Gene Therapy, commented, “It is Catalent’s continued goal to grow with our customers and to be able to offer them secure, state-of-the-art gene therapy facilities for their critical clinical and commercial needs. For gene therapies, the manufacturing scale-up process is complex and unique. Therefore, for our partners, having access to advanced adeno-associated virus production expertise and experience is vital to progress these pioneering treatments towards commercialization and the patients who need them.”

About BridgeBio Pharma

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development. For more information, visit bridgebio.com.

About Paragon Gene Therapy

Paragon Gene Therapy, a unit of Catalent Biologics, is an industry leader focusing on transformative technologies, including adeno-associated virus (AAV) gene therapies, next-generation vaccines, and oncology immunotherapies. Paragon Gene Therapy has facilities in Harmans and Baltimore, Maryland, which are dedicated to process development through commercial manufacturing of most scalable AAV platforms across multiple serotypes. Since 2016, Paragon Gene Therapy has completed over 100 clinical GMP AAV batches across 40 programs.

About Catalent

Catalent is the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, gene therapies, and consumer health products. With over 85 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable global clinical and commercial product supply. Catalent employs nearly 13,000 people, including over 2,400 scientists and technicians, at more than 35 facilities, and in fiscal year 2019 generated over $2.5 billion in annual revenue. Catalent is headquartered in Somerset, New Jersey. For more information, visit www.catalent.com.

BridgeBio Pharma Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to expectations, plans and prospects regarding the clinical development plans, clinical trial designs, the clinical and therapeutic potential, and the strategy of BridgeBio’s gene therapy product candidates, the clinical and commercial manufacturing supply needs of BridgeBio’s gene therapy product candidates, the ability of Catalent to provide adequate clinical and commercial supplies of active pharmaceutical ingredient to BridgeBio’s gene therapy product candidates that adhere to current good manufacturing practices (cGMP) regulations, the success of the collaboration agreement with Catalent, and the timing of these events, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by  a number of risks, uncertainties and assumptions, including, but not limited to, Catalent’s ability to provide adequate clinical and commercial supply of active pharmaceutical ingredient for BridgeBio’s gene therapy product candidates that comply with cGMP regulations, BridgeBio’s ability to maintain the collaboration agreement with Catalent, the benefits that will be derived from the collaboration agreement with Catalent, BridgeBio’s ability to support its gene therapy portfolio and advance its gene therapy product candidates through clinical development, and the novel nature of gene therapy technology and the related heightened challenges for obtaining and maintaining regulatory approval, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma, Inc.’s most recent Quarterly Report on Form 10-Q and our other SEC filings. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact:
BridgeBio Pharma, Inc.
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

Source: BridgeBio Pharma, Inc.

PellePharm Initiates Phase 2 Clinical Trial of Patidegib Topical Gel for People with High Frequency Basal Cell Carcinoma

SAN FRANCISCO – January 8, 2020 – PellePharm, Inc., a BridgeBio Pharma, Inc. (Nasdaq: BBIO) company, today announced it has dosed the first two participants in a Phase 2 clinical trial of Patidegib Topical Gel, 2%, vs. vehicle gel for people with non-Gorlin High-Frequency Basal Cell Carcinoma (HF-BCC). HF-BCC is a rare disease that causes a higher than average number of BCCs to develop, specifically in the facial area. PellePharm is a late clinical-stage biopharmaceutical company committed to targeting rare forms of basal cell carcinoma (BCC).

“There are approximately 35,000 people with HF-BCC in the United States. Their quality of life is significantly altered due to the multiple, invasive surgeries they must undergo during their treatment process,” said Sanuj K. Ravindran, M.D., president and chief executive officer of PellePharm. “Our goal is to provide people living with HF-BCC better, non-surgical options, and we are pleased to have initiated our multicenter Phase 2 trial to further evaluate Patidegib Topical Gel.”

The randomized, double-blinded, stratified, vehicle-controlled Phase 2 trial is evaluating the safety and efficacy of Patidegib Topical Gel 2% applied twice daily to the face over nine months vs. vehicle gel. The primary endpoint of the study is the number of surgically eligible basal cell carcinoma (nSEB) that develop on the face of participants over the nine-month period. The primary endpoint will be assessed by imaging and tracking of BCCs consistently throughout the study in order to identify nSEBs, consistent with the methods employed in the ongoing Phase 3 study of Patidegib topical gel for people living with Gorlin Syndrome. Approximately 40 participants will be enrolled in the Phase 2 trial.

“People with non-Gorlin HF-BCC are phenotypically similar to people with Gorlin Syndrome with respect to their BCCs, but are not born with a germline PTCH1 mutation. Those with HF-BCC are faced with the challenge of frequent BCC surgeries, which can be debilitating, painful and disfiguring, particularly to the face,” said Srikanth Pendyala, M.D., vice president of clinical development at PellePharm. “Due to the success we have found with enrolling our Gorlin Syndrome Phase 3 pivotal trial, we are thrilled about this important Phase 2 study milestone, and hope that by initiating this trial, we are closer to being able to mitigate the significant burden of frequent surgeries for those living with HF-BCC.”

PellePharm recently completed enrollment for its Phase 3 clinical trial of Patidegib Topical Gel 2% for people living with Gorlin Syndrome. PellePharm entered into a strategic collaboration with LEO Pharma in November 2018, which includes an option for LEO Pharma to acquire PellePharm.

About Patidegib

Patidegib Topical Gel, an investigational treatment, is designed to reduce the BCC tumor burden in people living with Gorlin Syndrome and High Frequency BCC (HF-BCC) by blocking the disease at its source within the hedgehog signaling pathway. Patidegib Topical Gel has shown early promise in a Phase 2 clinical study for the mitigation of BCC tumors in Gorlin Syndrome. The topical formulation of Patidegib was developed with a goal of providing the clinical activity previously demonstrated by oral Patidegib in Phase 1 trials and a favorable tolerability profile without the adverse systemic side effects observed with the oral class of hedgehog inhibitors. The topical gel formulation is stable at room temperature for at least two years, potentially making it an option for ongoing, at-home management of Gorlin Syndrome and HF-BCC. PellePharm has received both Orphan Drug Designation and Breakthrough Therapy Designation for Patidegib Topical Gel in Gorlin Syndrome from the FDA, as well as Orphan Drug Designation in Gorlin Syndrome from EMA’s Committee for Orphan Medicinal Products in the EU.

About Gorlin Syndrome

Gorlin Syndrome is a rare, genetic disease characterized by constitutional, heritable mutations in one allele of the tumor suppressor gene encoding PATCHED1 (PTCH1), which acts as the primary inhibitor of the hedgehog signaling pathway. This leads to the formation of multiple basal cell carcinomas (BCCs), often on the face.

With no FDA-approved drugs available for people living with Gorlin Syndrome, the standard of care for treating BCCs is surgery. People with severe Gorlin Syndrome may have as many as 30 surgeries per year, which can be repetitive, scarring and disfiguring. Approximately 10,000 people in the United States, or one in 31,000, are believed to be affected by Gorlin Syndrome. Gorlin Syndrome is known by several names, including Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome (BCNS) and Nevoid Basal Cell Carcinoma Syndrome (NBCCS).

About High Frequency Basal Cell Carcinoma (HF-BCC)

HF-BCC, like Gorlin Syndrome, is a rare disease which is characterized by the development of an abnormally high number of basal cell carcinomas (BCCs). Unlike people with Gorlin Syndrome, people with HF-BCC are not born with a germline PTCH1 mutation and do not suffer from the other systemic manifestations of Gorlin Syndrome. The current standard of care for people living with HF-BCC is BCC surgery.

About PellePharm

Founded by world leaders in hedgehog pathway signaling, PellePharm, a BridgeBio company, is committed to targeting rare forms of basal cell carcinoma, including Gorlin Syndrome and High Frequency Basal Cell Carcinoma (HF-BCC), at their source. PellePharm’s mission is to improve the quality of life for those suffering from Gorlin Syndrome and HF-BCC by providing an easy-to-use topical gel that could potentially reduce the need for regular, painful and disfiguring surgeries. Patidegib topical gel is a topical formulation of a proprietary hedgehog inhibitor.

About LEO Pharma

LEO Pharma A/S helps people achieve healthy skin. The company is a leader in medical dermatology with a robust R&D pipeline, a wide range of therapies and a pioneering spirit. Founded in 1908 and owned by the LEO Foundation, LEO Pharma has devoted decades of research and development to advance the science of dermatology, setting new standards of care for people with skin conditions. LEO Pharma is headquartered in Denmark with a global team of 6,000 people, serving 76 million patients in 130 countries. In 2018, the company generated net sales of DKK 10,410 million.

About BridgeBio Pharma

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development.

BridgeBio Pharma Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to expectations, plans and prospects regarding PellePharm’s clinical development plans, clinical trial designs, the clinical and therapeutic potential of Patidegib Topical Gel, the results of clinical trials, the funding by LEO Pharma of PellePharm’s pivotal Phase 3 trial of Patidegib Topical Gel in people living with Gorlin Syndrome, PellePharm’s Phase 2 clinical trial of Patidegib Topical Gel 2% in people living with High Frequency Basal Cell Carcinoma, enrollment of PellePharm’s Phase 2 clinical trial, and the timing of these events, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by  a number of risks, uncertainties and assumptions, including, but not limited to, PellePharm’s ability to enroll participants in and generate data from the ongoing Phase 2 and Phase 3 trials, the benefits that will be derived from these trials, the progress and results of the trials, PellePharm’s ability to advance Patidegib Topical Gel in clinical development in accordance with its plans, the timing of these events, and PellePharm’s ability to maintain its collaboration with LEO Pharma, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma Inc.’s most recent Quarterly Report on Form 10-Q and our other SEC filings. Moreover, PellePharm operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of PellePharm’s management as of the date of this release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Media Contact:
Lauren Barbiero
W2O Pure
(646) 564-2156
lbarbiero@w2ogroup.com

BridgeBio Pharma’s QED Therapeutics Receives Fast Track Designation for Infigratinib in Adults with First-Line Advanced or Metastatic Cholangiocarcinoma and Orphan Drug Designation for Infigratinib for Treatment of Cholangiocarcinoma

The PROOF trial, a Phase 3 trial of infigratinib in first-line cholangiocarcinoma, is currently enrolling

SAN FRANCISCO – January 6, 2020 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) subsidiary QED Therapeutics announced today that it has secured both Fast Track Designation in adults with first-line advanced or metastatic cholangiocarcinoma and Orphan Drug Designation for infigratinib for treatment of cholangiocarcinoma. In addition, the company announced that enrollment is ongoing and patient dosing has started in the PROOF trial, a Phase 3 clinical trial evaluating oral infigratinib, an investigational drug, in adults for first-line treatment of advanced cholangiocarcinoma with FGFR2 (fibroblast growth factor receptor 2) gene fusions or translocations.

Cholangiocarcinoma, a cancer of the bile ducts of the liver, is a serious and often fatal disease which affects approximately 20,000 people in the United States and European Union each year. FGFR2 genetic aberrations are present in approximately 15% to 20% of people who have this disease. Currently, treatment options are limited, and the 5-year survival rate is only 9%.1

Infigratinib received Fast Track Designation for first-line treatment of adult patients with unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or translocations.

“We believe that Fast Track and Orphan Drug Designations for infigratinib for the treatment of cholangiocarcinoma underscores the need for new, targeted treatments for genetically-driven subsets of this cancer, particularly for adults with first-line advanced or metastatic cholangiocarcinoma,” said Susan Moran, MD, MSCE, chief medical officer for QED. “Fast Track designation will enhance our interaction with the FDA on our first-line advanced or metastatic cholangiocarcinoma program and may help us get this medicine to patients more quickly.”

The PROOF trial will enroll approximately 384 patients with first-line cholangiocarcinoma with FGFR2 fusions or translocations, as determined by molecular profiling. The primary endpoint is progression-free survival compared to standard of care chemotherapy (gemcitabine and cisplatin). Patients will be randomized 2:1 to infigratinib versus standard of care.

“Importantly, in this trial, patients who are assigned to receive standard of care will be allowed to cross over and receive infigratinib if they do not respond to chemotherapy,” said Stacie Lindsey, president of the Cholangiocarcinoma Foundation. “Having a crossover option is very significant to patients and including it in the design of this trial demonstrates that QED is listening to them.”

For additional information on the PROOF trial, including eligibility, patients should ask their physician, visit clinicaltrials.gov, or email PROOF301@QEDtx.com

For more information on molecular profiling, patients can find resources from the Cholangiocarcinoma Foundation at https://cholangiocarcinoma.org/mutationsmatter

About Orphan Drug Designation

Under the FDA’s Orphan Drug Designation program, orphan drug designation is granted by the FDA to drugs or biologics intended to treat rare diseases or conditions. The designation allows the drug developer to be eligible for a seven-year period of U.S. marketing exclusivity upon approval of the drug, if the drug receives the first FDA approval for the rare disease or condition, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About U.S. FDA’s Fast Track Designation Program

The FDA’s Fast Track program was established to facilitate the development and expedite the review of drugs with the potential to treat serious conditions and address an unmet medical need. Companies that receive Fast Track designation are provided the opportunity for more frequent interactions with FDA during clinical development and are eligible for accelerated approval and/or priority review, if relevant criteria are met. Additionally, companies that receive Fast Track designation are allowed to submit completed sections of their New Drug Application (NDA) or Biologics License Application (BLA) for the drug on a rolling basis, resulting in the potential for an expedited FDA review process.

About QED Therapeutics

QED Therapeutics, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on precision medicine for FGFR-driven diseases. Our lead investigational candidate is infigratinib (BGJ398), an orally administered, FGFR1-3 selective tyrosine kinase inhibitor that has shown activity that we believe to be meaningful in clinical measures, such as overall response rate, in patients with chemotherapy-refractory cholangiocarcinoma with FGFR2 fusions and advanced urothelial carcinoma with FGFR3 genomic alterations. In addition to the first-line cholangiocarcinoma program and the Phase 3 PROOF trial, QED has also studied infigratinib in a Phase 2, open-label study in second and later-line cholangiocarcinoma and intends to pursue an NDA filing for this indication in 2020. QED Therapeutics is also evaluating infigratinib in preclinical studies for the treatment of achondroplasia. We plan to conduct further clinical trials to evaluate the potential for infigratinib to treat patients with other FGFR-driven tumor types and rare disorders.

For more information on QED Therapeutics, please visit the company’s website at www.qedtx.com

About BridgeBio Pharma, Inc.

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development.

BridgeBio Pharma Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.  These forward-looking statements, including statements relating to expectations, plans, and prospects regarding QED Therapeutics’ regulatory approval process, clinical trial designs, clinical development plans, clinical trial results, timing and completion of clinical trials, clinical and therapeutic potential of infigratinib, the availability of marketing exclusivity and other benefits under the FDA’s Orphan Drug Designation program, and the availability of accelerated approval or priority review under the FDA’s Fast Track program, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, QED Therapeutics’ ability to initiate and continue its ongoing and planned clinical trials of infigratinib, the availability of data from these trials, its ability to advance infigratinib in clinical development according to its plans, and the timing of these events, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma Inc.’s most recent Quarterly Report on Form 10-Q and our other SEC filings. Moreover, QED Therapeutics operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

_________________________

1Dhanasekaran, R., Hemming, A. W., Zendejas, I., George, T., Nelson, D. R., Soldevila-Pico, C., Firpi, R. J., Morelli, G., Clark, V., Cabrera, R. “Treatment outcomes and prognostic factors of intrahepatic cholangiocarcinoma”. Oncology Reports 29.4 (2013): 1259-1267.

QED Contact:

Carolyn Hawley
Canale Communications
carolyn@canalecomm.com
858-354-3581

BridgeBio Pharma, Inc. To Present At Upcoming Investor Conference

PALO ALTO, Calif., December 19, 2019 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) announced today that it will present at the 38th Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2020 at 3 PM (PST) in San Francisco.

The presentation will be webcast live and can be accessed at www.bridgebio.com on the For Investors page under News & Events. The webcast will be available for replay for 30 days.

About BridgeBio Pharma

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development. For more information, please visit www.bridgebio.com.

Investor contact:
BridgeBio Pharma, Inc.
ir@bridgebio.com

Media contact:
BridgeBio Pharma, Inc.
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

Source: BridgeBio Pharma, Inc.

BridgeBio Pharma’s Origin Biosciences Enters Partnership with Medison To Commercialize BBP-870 In Israel for MoCD Type A

BOSTON, December 18, 2019 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) subsidiary Origin Biosciences, a company focused on developing and commercializing a treatment for Molybdenum Cofactor Deficiency (MoCD) Type A, and Medison Pharma Ltd., a leading commercial biotech partner that operates in Israel, Canada and Central and Eastern European countries, have entered into an exclusive license agreement under which Medison received rights from Origin to distribute, market, sell and otherwise commercialize Origin’s drug product known as fosdenopterin (BBP-870/ORGN001) in Israel.

Fosdenopterin is a cPMP replacement therapy designed to treat patients with MoCD Type A. Origin recently initiated the rolling submission of a New Drug Application (NDA) with the US Food and Drug Administration (FDA) for fosdenopterin. Subsequent to FDA approval, Medison will be responsible for seeking the requisite regulatory approval and, once approved, commercializing fosdenoptrin in Israel. Origin will receive an upfront cash payment and will receive milestone payments upon the achievement of certain milestone events and ongoing royalties on net sales of fosdenopterin in Israel.

“Our partnership with Medison marks an important step in Origin’s global commercial strategy and strengthens our ability to make fosdenopterin available to infants and children suffering from MoCD Type A,” said Matt Outten, Chief Commercial Officer at BridgeBio. “Medison has a strong commercial organization in Israel with a proven track record of successfully marketing orphan disease products. Its commercial infrastructure is uniquely suited to support patients suffering from rare diseases like MoCD Type A. We look forward to collaborating with Medison to enhance our efforts to help patients by targeting MoCD Type A at its source.”

“Our promise is to assure that every patient under our responsibility has access to the best available treatments,” said Meir Jakobsohn, the founder and CEO of Medison Pharma. “The partnership with Origin Biosciences echoes our shared internal values of scientific excellence.”

Fosdenopterin has received Orphan Drug Designation in the US and Europe, and Rare Pediatric Disease Designation and Breakthrough Therapy Designation in the United States. Since the NDA for fosdenopterin is seeking approval of treatment for patients with a serious and life-threatening disease with no other treatment options (MoCD Type A), it is also eligible for Priority Review Designation, which, if granted, may further expedite the NDA review time for the potential approval of this new medicine in the United States.

About Molybdenum Cofactor Deficiency (MoCD) Type A

MoCD Type A is an ultra-rare, autosomal recessive, inborn error of metabolism caused by disruption in molybdenum cofactor (MoCo) synthesis that is vital for sulfite oxidase (SOX) activity. Patients are often infants with severe encephalopathy and intractable seizures. Disease progression is rapid with a high infant mortality rate.1,2 Those who survive beyond the first few months experience profuse developmental delays and suffer the effects of irreversible neurological damage, including brain atrophy with white matter necrosis, dysmorphic facial features, and spastic paraplegia.1,4 Clinical presentation that can be similar to hypoxic-ischemic encephalopathy (HIE) or other neonatal seizure disorders may lead to misdiagnosis and underdiagnosis.2,3 Immediate testing for elevated sulfite levels and S-sulfocysteine in the urine and very low serum uric acid may help with suspicion of MoCD Type A.2,4

About Origin Biosciences

Origin Biosciences, a subsidiary of BridgeBio Pharma Inc., is a biotechnology company focused on developing and commercializing a treatment for MoCD Type A. Origin is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring a safe, effective treatment for MoCD Type A to market as quickly as possible. For more information on Origin Biosciences, please visit the company’s website at www.origintx.com.

About Medison

Medison, is one of the world’s largest commercial partners of leading global biotech companies, providing the complete spectrum of integrated services for international companies looking to enter or expand their presence in Israel, Canada and CEE markets. Medison operates a corporate venture arm with a dedicated research and evaluation team boasting deep scientific and commercial backgrounds. Medison also operates a scouting program to cater its partners and is an active investor in life science projects around drug development and digital health.

About BridgeBio Pharma

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development.

BridgeBio Pharma Forward Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to Origin Biosciences’ clinical development plans, including its clinical development plans for BBP-870 (ORGN001), clinical trial results, timing and completion of clinical trials and regulatory submissions, competitive environment and clinical and therapeutic potential of BBP-870, the potential approval of BBP-870 by the FDA and subsequent development and commercialization by Medison of BBP-870 under the exclusive license agreement and our ability to receive milestone and royalty payments under the agreement, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, Origin Biosciences’ ability to continue its planned clinical development and regulatory submissions for BBP-870 and the timing and success of any such continued clinical development and planned regulatory submissions, as well as those set forth in the Risk Factors section of BridgeBio Pharma Inc.’s most recent Quarterly Report on Form 10-Q and our other SEC filings. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

  1. Spiegel R, Schwahn B, Scribner C L, Confer N. A natural history study of molybdenum cofactor (MoCo) and isolated sulfite oxidase deficiencies (ISOD). https://origintx.com/posters/
  2. Mechler K, Mountford WK, Hoffmann GF, Ries M. Ultra-orphan diseases: a quantitative analysis of the natural history of molybdenum cofactor deficiency. Genet Med. 2015;17(12):965-970.
  3. Durmaz MS, Özbakır B. Molybdenum cofactor deficiency: neuroimaging findings. Radiol Case Rep. 2018;13(3):592-595.
  4. Schwahn BC, Van Spronsen FJ, Belaidi AA, et al. Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study. Lancet. 2015;386(10007):1955-1963.

For more information:

Origin Biosciences
Grace Rauh
grace.rauh@bridgebio.com
(917) 232-5478

Medison Pharma LTD.
Gil Gurfinkel
VP Corporate Development
Gil@medison.co.il

Source: BridgeBio Pharma, Inc., Medison Pharma Ltd.

BridgeBio Pharma, Inc. Appoints Industry Leader Jennifer Cook as New Board Member

Genentech veteran brings global commercial expertise to Board of Directors and will serve as a Special Advisor

PALO ALTO, Calif., December 16, 2019 — BridgeBio Pharma, Inc. (NASDAQ: BBIO) today announced it has appointed Jennifer Cook to its Board of Directors and will leverage her considerable expertise as a special advisor to the senior management team. Her counsel will help BridgeBio expand its capacity to develop treatments for genetic diseases as quickly and safely as possible.

“Jennifer is a seasoned leader in the biotech and pharmaceutical spaces, with decades of experience taking drugs from research to commercialization,” said Richard Scheller, Ph.D., chairman of research and development at BridgeBio. “During our time at Roche and Genentech, I saw her launch new medicines and drive growth for both companies. I am confident that her appointment to the board will strengthen BridgeBio’s experience and capabilities in drug commercialization.”

Cook brings more than 30 years of experience in the global biotech and pharmaceutical industry. Her work includes serving as Chief Executive Officer of GRAIL, Inc., leading Roche Pharma’s European commercial business, heading global clinical operations for Roche product development, and overseeing Genentech’s U.S. Immunology and Ophthalmology Business Unit. Cook was honored as the Woman of the Year by the Healthcare Businesswomen’s Association in 2016 and was named one of the Most Influential Women in Business by The San Francisco Business Times in 2012. Cook joins current board members Neil Kumar, Charles Homcy, Richard Scheller, Eric Aguiar, James Momtazee, and Ali Satvat. She will also serve as a special advisor to Kumar, Ph.D., the founder and CEO of BridgeBio.

“As BridgeBio evolves into a commercial company that serves patients with genetic diseases and supports the physicians who care for them, we require more expertise and guidance as it relates to commercial activity,” said Dr. Kumar. “Jennifer brings that expertise and more, having run a massive commercial organization in Europe. She also offers strategic perspectives on how to manage commercial infrastructure in a company like ours, which has a strong central team supporting a growing network of affiliate companies. Jennifer is a leader I admire and I look forward to partnering with and learning from her.” 

“What interests me most about BridgeBio is its remarkable people and their focus on turning early-stage innovation in academic laboratories and leading medical institutions into drug discovery programs as quickly and efficiently as possible,” said Cook. “I look forward to applying my experience in biotech product development and commercialization to the important therapeutic areas BridgeBio is currently working on to help the team bring these treatments to patients.”

About BridgeBio Pharma

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development. For more information, please visit www.bridgebio.com.

Contact:
BridgeBio Pharma, Inc.
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

Source: BridgeBio Pharma, Inc.

BridgeBio Pharma, Inc. Appoints Eli Wallace as Chief Scientific Officer in Residence for Oncology Programs

Dr. Wallace brings over two decades of experience in oncology drug discovery to leadership team

PALO ALTO, Calif. – December 9, 2019 – BridgeBio Pharma, Inc. (NASDAQ: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, announced it has appointed Eli Wallace, Ph.D., as its chief scientific officer in residence for oncology effective immediately. In this new role, Dr. Wallace will oversee BridgeBio’s ongoing discovery efforts in targeted oncology while driving the identification and adoption of new oncology programs.

“Eli is a brilliant medicinal chemist with an outstanding record of accomplishment in drug development at Array BioPharma and Peloton Therapeutics,” said Frank McCormick, Ph.D., chairman of oncology at BridgeBio. “Eli is one of the most productive discovery scientists in oncology and will lead our efforts to bring drugs against KRAS, GPX4, and other cancer targets into the clinic. I am delighted he has joined our team and look forward to working with him to bring these new therapies to patients.”

Dr. Wallace is a strategic executive leader with 24 years of experience in drug discovery and development, having overseen entire research organizations at companies such as Peloton Therapeutics and Array BioPharma. He has a proven track record leading teams that progressed 13 small molecule drugs into clinical development. In his most recent role as chief scientific officer at Peloton, he and his team discovered and developed a first-in-class HIF2a inhibitor for renal cell carcinoma through clinical proof-of-concept. As one of the original employees of Peloton Therapeutics, Dr. Wallace helped build the research team from six to 30 scientists. Prior to his tenure at Peloton, Dr. Wallace worked in the medicinal chemistry department at Array BioPharma where he rose through the ranks to director. He led multiple research projects, which produced eight new chemical entities that have entered human clinical trials for the treatment of cancer, including Mektovi®, which was approved to treat melanoma in 2018. Specializing in chemistry, Dr. Wallace has co-authored 25 original research articles and invited reviews, and is co-inventor on 79-issued U.S. patents.

Dr. Wallace will be part of the growing team of experienced drug developers working at BridgeBio that includes biotech leaders like Drs. Frank McCormick, Richard Scheller, chairman of research and development at BridgeBio, and Charles Homcy, chairman of pharmaceuticals at BridgeBio.

“We are lucky to partner with Dr. Wallace as he steps in to lead the development of our early stage oncology portfolio,” said BridgeBio CEO and founder Neil Kumar, Ph.D. “Dr. Wallace is a scientist and drug developer we have admired for a very long time. In our book, what matters most is the development of meaningful medicines, something he has done time and again in his career.”

KRAS is a key driver of a number of large cancer indications with high unmet need, including non-small cell lung cancers, pancreatic adenocarcinomas and colorectal adenocarcinomas. GPX4 is a key tumor dependency that was recently identified as a priority oncology target based on high-impact academic reports.

“I am excited to join Drs. Frank McCormick, Richard Scheller, Charles Homcy, and Neil Kumar, and the rest of the talented BridgeBio team,” Dr. Wallace said. “BridgeBio is the perfect setting to apply my experience in small molecule drug discovery to challenging targets that will provide new, meaningful therapeutics to patients afflicted with cancer. When I saw the targets and the promising chemistry underpinning the company’s oncology programs, I knew this was where I wanted to be.”

About BridgeBio Pharma

BridgeBio is a team of experienced drug discoverers, developers, and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development. For more information, please visit www.bridgebio.com.

Contact
BridgeBio Pharma, Inc.
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

Source: BridgeBio Pharma, Inc.

BridgeBio Pharma Reports Inducement Grants under Nasdaq Listing Rule 5635(c)(4)

Palo Alto, CA, December 5, 2019 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, today announced that on November 22, 2019, the compensation committee of BridgeBio’s board of directors granted eight new employees options to purchase an aggregate of 62,745 shares of the Company’s common stock with a per share exercise price of $31.14 and restricted stock units for an aggregate of 23,980 shares of the Company’s common stock. All of the above-described awards were made under BridgeBio’s 2019 Inducement Equity Plan (the Plan).

The above-described awards were each granted as an inducement material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4), and were granted pursuant to the terms of the Plan. The Plan was adopted by BridgeBio’s board of directors in November 2019.

About BridgeBio

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development.

Contact
BridgeBio Pharma, Inc.
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

Source: BridgeBio Pharma, Inc.

BridgeBio Pharma’s Origin Biosciences Initiates Rolling Submission Of New Drug Application With The U.S. FDA For BBP-870 For The Treatment Of MoCD Type A

BOSTON, December 3, 2019 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) subsidiary Origin Biosciences has initiated a rolling submission of a New Drug Application (NDA) with the United States Food and Drug Administration (FDA) for fosdenopterin (BBP-870/ORGN001) for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A.

Currently, there are no approved therapies that alter the course of MoCD Type A, which results in severe and irreversible neurological injury. Fosdenopterin, an investigative cPMP substrate replacement therapy, aims to reduce buildup of toxic sulfites and alleviate central nervous system symptoms in infants and children with MoCD Type A.

“Initiation of our rolling NDA submission is a significant milestone for our company and an important step in providing a therapy to patients and their families living with MoCD Type A,” said Origin Biosciences CEO Neil Kirby, Ph.D. “We look forward to making fosdenopterin available to infants and children with this devastating disease as soon as possible as they currently have no treatment options that target the disease at its source.”

“BridgeBio was founded to develop breakthrough medicines for patients with genetic diseases. As we begin our first rolling new drug application with the FDA, we are taking a significant step forward for patients by targeting MoCD Type A at its source through the provision of the monophosphate cPMP. We want to thank patients and their families, the scientists, and all others involved who helped us reach this critical moment,” BridgeBio CEO Neil Kumar, Ph.D., said. “Our growing company has more than 15 drug discovery and development programs. We hope Origin’s NDA will be the first of many we submit to the FDA in our pursuit of life-changing therapies for patients, including an anticipated submission in 2020 from BridgeBio subsidiary QED Therapeutics for infigratinib for second line treatment of cholangiocarcinoma.”

The regulatory submission will primarily be supported by data from two ongoing trials, a global Phase 2 clinical trial (NCT02629393) and a global Phase 2/3 clinical trial (NCT02047461). Under its rolling review process, the FDA can review components of a marketing application as they are submitted rather than requiring all components to be received prior to initiating review, potentially allowing for faster review of the application.

BBP-870 has received Orphan Drug Designation in the US and Europe, and Rare Pediatric Disease Designation and Breakthrough Therapy Designation in the US. Since the NDA for fosdenopterin is seeking approval of treatment for patients with a serious and life-threatening disease with no other treatment options (MoCD Type A), it is also eligible for Priority Review Designation, which, if granted, may further expedite the NDA review time for the NDA approval of this new medicine.

About Molybdenum Cofactor Deficiency (MoCD) Type A

MoCD Type A is an ultra-rare, autosomal recessive, inborn error of metabolism caused by disruption in molybdenum cofactor (MoCo) synthesis that is vital for sulfite oxidase (SOX) activity. Patients are often infants with severe encephalopathy and intractable seizures. Disease progression is rapid with a high infant mortality rate.1,2  Those who survive beyond the first few months experience profuse developmental delays and suffer the effects of irreversible neurological damage, including brain atrophy with white matter necrosis, dysmorphic facial features, and spastic paraplegia.1,4 Clinical presentation that can be similar to hypoxic-ischemic encephalopathy (HIE) or other neonatal seizure disorders may lead to misdiagnosis and underdiagnosis.2,3 Immediate testing for elevated sulfite levels and S-sulfocysteine in the urine and very low serum uric acid may help with suspicion of MoCD Type A.2,4

About Origin Biosciences

Origin Biosciences, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on developing and commercializing a treatment for MoCD Type A. Origin is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring a safe, effective treatment for MoCD Type A to market as quickly as possible. For more information on Origin Biosciences, please visit the company’s website at www.origintx.com.

About BridgeBio Pharma

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development.

BridgeBio Pharma Forward Looking Statements

This press release contains forward-looking statements.  Statements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.  These forward-looking statements, including statements relating to Origin Biosciences’ clinical development plans, including its plans to initiate a rolling NDA submission for BBP-870 (ORGN001), clinical trial results, timing and completion of clinical trials and regulatory submissions, competitive environment and clinical and therapeutic potential of BBP-870, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made.  Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, Origin Biosciences’ ability to continue its planned clinical development and regulatory submissions for BBP-870 and the timing and success of any such continued clinical development and planned regulatory submissions, as well as those set forth in the Risk Factors section of BridgeBio Pharma Inc.’s most recent Quarterly Report on Form 10-Q and our other SEC filings.  Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

1Spiegel R, Schwahn B, Scribner C L, Confer N. A natural history study of molybdenum cofactor (MoCo) and isolated sulfite oxidase deficiencies (ISOD). https://origintx.com/posters/

2Mechler K, Mountford WK, Hoffmann GF, Ries M. Ultra-orphan diseases: a quantitative analysis of the natural history of molybdenum cofactor deficiency. Genet Med. 2015;17(12):965-970.

3Durmaz MS, Özbakır B. Molybdenum cofactor deficiency: neuroimaging findings. Radiol Case Rep. 2018;13(3):592-595.

4Schwahn BC, Van Spronsen FJ, Belaidi AA, et al. Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study. Lancet. 2015;386(10007):1955-1963.

Contact:
Grace Rauh
grace.rauh@bridgebio.com
(917) 232-5478