BridgeBio Pharma LLC Signs Joint Collaboration Agreement with Cincinnati Children’s to Accelerate Development of Genetic Disease Therapeutics

PALO ALTO, Calif. and CINCINNATI, Nov. 12, 2018 /PRNewswire/ — BridgeBio Pharma LLC and Cincinnati Children’s Hospital Medical Center today announced they are entering into a research collaboration to identify and develop genetic disease therapies. This partnership will combine BridgeBio’s strengths in therapeutic development for diseases of high unmet need with Cincinnati Children’s world-class researchers and clinicians. As a global research center of excellence, Cincinnati Children’s represents an ideal partner for BridgeBio and reinforces BridgeBio’s commitment to collaborating with leading researchers toward the mutual goal of discovering new therapies targeting genetic diseases at the source.

“BridgeBio is privileged to partner with leading experts at Cincinnati Children’s, an institution whose impact on science and patients we admire. We hope we can help advance the mission by partnering to move therapies forward to patients,” said Neil Kumar, co-founder and CEO of BridgeBio.

“We are excited to launch this new alliance with BridgeBio to bring new and innovative therapies to some of the most vulnerable patients impacted by rare and complex genetic diseases,” said Margaret Hostetter, MD, Chief Medical Officer of Cincinnati Children’s and Director, Cincinnati Children’s Research Foundation.

“This collaboration exemplifies Cincinnati Children’s commitment to accelerating discoveries towards commercialization through win-win academic-industry partnerships,” added Mike Pistone, Director of Acceleration for Cincinnati Children’s Innovation Ventures, the group managing this program.

Under the terms of the agreement, BridgeBio will have the opportunity to sponsor and collaboratively develop selected research programs. The agreement represents a flexible model that facilitates accelerated genetic disease research.

About BridgeBio Pharma

BridgeBio finds, develops, and delivers breakthrough medicines for genetic diseases. The company bridges remarkable advancements in genetic science with the entrepreneurial engine required to rapidly create lifesaving medicines for patients with unmet needs. Founded in 2015 by a team of industry veterans, the company has built a robust portfolio of 19 transformative drugs ranging from pre-clinical to late stage development in multiple therapeutic areas including oncology, cardiology, dermatology, and endocrinology. The company’s focus on scientific excellence and rapid execution aims to translate today’s discoveries into tomorrow’s medicines. For additional information, visit BridgeBio.com.

Eidos Therapeutics Announces Positive Phase 2 Data for AG10 in Symptomatic Patients with Mutant or Wild-Type TTR Amyloid Cardiomyopathy

SAN FRANCISCO, Nov. 10, 2018 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced positive results of its Phase 2 clinical trial studying AG10 in subjects with symptomatic transthyretin (TTR) amyloidosis cardiomyopathy (ATTR-CM). The data were presented in a late-breaking Featured Science oral presentation at the American Heart Association (AHA) Scientific Sessions. AG10 was well tolerated, demonstrated >90% TTR average stabilization at day 28, and increased serum TTR concentrations, a prognostic indicator of survival in ATTR-CM, in a dose-dependent manner. Subject to discussions with regulatory agencies, these data support the advancement of AG10 into Phase 3 pivotal trials planned to be initiated in the first half of 2019.

“These data demonstrate that AG10 is well tolerated in symptomatic patients with ATTR-CM with clear evidence of drug activity in all actively treated subjects,” said Jonathan Fox, MD, PhD, president and chief medical officer of Eidos. “The consistently high levels of TTR stabilization, in all actively treated subjects and across the entire dosing interval, were correlated with statistically significant and dose-dependent increases in serum TTR concentrations. We observed normalized serum TTR levels in 100% of patients treated with AG10. We believe these data provide clinical proof-

of-concept for AG10 in ATTR-CM patients. As reflected in these data, AG10’s TTR-stabilizing properties continue to hold great promise that it could become a best-in-class treatment for ATTR-CM.”

Phase 2 Clinical Trial

This Phase 2 clinical trial was a randomized, double-blind, placebo-controlled, multi-center study that enrolled patients with symptomatic ATTR-CM, both wild-type and mutant. Eligible patients had confirmed ATTR-CM and NYHA Class II or III symptoms, and at least one prior heart failure hospitalization or active treatment for chronic heart failure. The 49 enrolled subjects were randomly assigned in a 1:1:1 fashion to treatment with placebo, 400 mg AG10 twice daily, or 800 mg AG10 twice daily for 28 days. Results from the study showed the following:

  • The study met its primary objective of establishing that AG10 was well tolerated with no safety signals of potential clinical concern related to the administration of AG10 in symptomatic ATTR-CM patients. One serious adverse event (SAE) of dyspnea deemed unrelated to study drug was observed in one actively-treated subject (3%) and SAEs of atrial fibrillation, congestive heart failure, and cellulitis were observed in two placebo-treated subjects (12%). The overall rate of adverse events (AEs) was 69% in subjects administered 800 mg bid AG10, 63% in subjects administered 400 mg bid AG10, and 88% in subjects administered placebo.
  • As compared to placebo, subjects treated with AG10 demonstrated a statistically significant increase in serum TTR concentrations (p<0.0001), a prognostic indicator of survival in ATTR-CM patients, in a dose-dependent manner. Subjects administered 800 mg AG10 twice daily, 400 mg AG10 twice daily, and placebo exhibited mean changes in TTR concentration from baseline of +50%, +36% and -7%, respectively, at day 28.
  • All subjects administered AG10 had serum TTR concentrations within the normal range at day 28, whereas 31% of subjects administered placebo had serum TTR concentrations below the normal range on day 28.
  • AG10 administration resulted in near-complete stabilization of TTR at day 28 (>90%, on average), across the dosing interval in all actively treated subjects as measured by established ex vivo assays.

“We know that stabilizing TTR can lead to clinical benefit in ATTR patients, that treatment with a stabilizer increases TTR levels in ATTR-CM patients, and that higher serum concentrations of TTR are associated with a better prognosis in ATTR-CM,” said Dr. Daniel Judge, M.D., professor in the division of cardiology at the Medical University of South Carolina. “These Phase 2 data provide compelling evidence that AG10 stabilizes TTR to a high degree and restores serum TTR levels to normal even in patients carrying destabilizing mutations, suggesting the potential for the molecule to become an effective disease-modifying therapy for ATTR patients.”

Investor Conference Call and Webcast Details

Eidos management will host an investor conference call and webcast on Monday, November 12 at 8am ET to review the Phase 2 data. To participate in the conference call, dial +1-844-293-0174 (U.S. toll free) or +1-916-582-3546 (international), conference ID 8594856. The webcast will be available live and for replay on the company’s website at ir.eidostx.com.

About AG10

AG10 is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to amyloidosis, or ATTR. AG10 is currently being studied in an open-label extension of a Phase 2 clinical trial in patients with ATTR cardiomyopathy.

AG10 was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a “rescue mutation” because it has been shown to prevent ATTR in individuals carrying pathogenic, or disease-causing, mutations in the TTR gene. To our knowledge, AG10 is the only TTR stabilizer in development that has been observed to mimic the “super-stabilizing” properties of this rescue mutation.

About transthyretin amyloidosis (ATTR)

ATTR represents a significant unmet need of a comparatively large patient population in the context of rare genetic diseases with an inadequate current standard of care. There are three distinct diseases that comprise the ATTR family: wild-type ATTR cardiomyopathy (ATTRwt-CM), mutant ATTR cardiomyopathy (ATTRm-CM), and ATTR polyneuropathy (ATTR-PN). The worldwide prevalence of each disease is approximately 400,000 patients, 40,000 patients and 10,000 patients, respectively.

All three forms of ATTR are progressive and fatal. For patients with ATTRwt-CM and ATTRm-CM, symptoms usually manifest later in life (age 50+), with median survival of three to five years from diagnosis. ATTR-PN either presents in a patient’s early 30s or later (age 50+), and results in a median life expectancy of five to ten years from diagnosis. Progression of all forms of ATTR causes significant morbidity, impacts productivity and quality of life, and creates a significant economic burden due to the costs associated with progressively greater patient needs for supportive care.

About Eidos Therapeutics

Eidos Therapeutics is a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR). For more information, please visit www.eidostx.com.

Forward-Looking Statements

This release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act. All statements other than statements of historical facts, including the statements about the potential therapeutic and clinical benefits of AG10, its potential to become a best-in-class treatment for ATTR-CM, future clinical milestones of AG10, the timing of these events, the indications we intend to pursue and our possible clinical or other business strategies, are forward-looking statements. Forward-looking statements can be identified by terms such as “believes,” “expects,” “plans,” “potential,” “would” or similar expressions and the negative of those terms. These forward-looking statements are based on our management’s current beliefs and assumptions about future events and on information currently available to management.

Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These risks include, but are not limited to, risks and uncertainties related to: our limited operating history and historical losses, our liquidity to fund the development of our other product candidates through current and future milestones, our ability to raise additional funding to complete the development of AG10, our dependence on the success of AG10, results from our clinical trials and pre-clinical studies and those of third parties working in the same area as our product candidate, our ability to advance AG10 in clinical development in accordance with our plans, and our dependence on third parties in connection with our manufacturing, clinical trials and pre-clinical studies. Additional risks and uncertainties that could affect our future results are included in the section titled “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2018, which is available on the SEC’s website at www.sec.gov and our website at eidostx.com. Additional information on potential risks will be made available in other filings that we make from time to time with the SEC. In addition, any forward-looking statements contained in this press release are based on assumptions that we believe to be reasonable as of this date. Except as required by law, we assume no obligation to update these forward-looking statements, or to update the reasons if actual results differ materially from those anticipated in the forward-looking statements.

Media Contact: Carolyn Hawley

Canale Communications 619-849-5382

Carolyn@canalecomm.com

Investor Contact: Alex Gray

Burns McClellan 212-213-0006

agray@burnsmc.com

Eidos Therapeutics Receives Positive Opinion for Orphan Designation from the European Medicines Agency for AG10, a Potent Oral Stabilizer for the Treatment of Transthyretin Amyloidosis

SAN FRANCISCO, Oct. 26, 2018 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), announced today that the Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA) adopted a positive opinion for the designation of Eidos’ product candidate, AG10, as an orphan medicinal product for the treatment of transthyretin (TTR) amyloidosis (ATTR). In addition, the EMA also granted a product-specific pediatric investigational plan waiver to Eidos for AG10.

TTR normally circulates as a four-part molecule or tetramer, but in ATTR the molecule dissociates into individual monomers, which are unstable and aggregate as amyloid fibrils. Eidos’ lead product candidate, AG10, is designed to target ATTR at its source by stabilizing tetrameric TTR in the blood.

“We are pleased to have received Orphan Drug Designation from the FDA and now a positive opinion from the EMA as well. Eidos is excited about the opportunity to work with the ATTR patient and provider community on the further development of AG10 for ATTR,” noted Jonathan Fox, M.D., Ph.D., president and chief medical officer of Eidos. “Importantly, AG10 has demonstrated potent stabilization of tetrameric TTR in our clinical program, and we believe maximizing the level of TTR stabilization will lead to optimal clinical benefit for patients living with this devastating disease.”

Orphan Designation by the European Commission provides regulatory and financial incentives for the development of medicines that treat a

life-threatening or chronically debilitating condition affecting less than five in 10,000 people in the European Union (EU). Orphan Designation provides Eidos with certain benefits, including EU market exclusivity upon regulatory approval, if received, reductions in EMA application fees, and access to protocol assistance. Eidos is pursuing AG10 for the treatment of ATTR cardiomyopathy (ATTR-CM) and ATTR polyneuropathy (ATTR-PN), both of which are progressive, fatal diseases. The company plans to initiate Phase 3 studies in each indication in the first half of 2019.

About AG10

AG10 is an investigational, orally administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to amyloidosis, or ATTR. AG10 is currently being examined in a Phase 2 clinical trial in patients with ATTR cardiomyopathy and symptomatic heart failure. Results from this trial will be presented on November 10, 2018 at the American Heart Association’s Annual Scientific Sessions.

AG10 was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a “rescue mutation” because it has been shown to prevent ATTR in individuals also carrying a pathogenic, or disease-causing, mutation in the other copy of their TTR gene. To our knowledge, AG10 is the only TTR stabilizer in development that has been observed to mimic the “super-stabilizing” properties of this rescue mutation.

About Eidos Therapeutics

Eidos Therapeutics is a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR). For more information, please visit www.eidostx.com.

Forward-Looking Statements

This release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act. All statements other than statements of historical facts, including the statements about future clinical milestones of AG10, including the completion of our ongoing Phase 2 clinical trial and availability of data therefrom, the initiation of Phase 3 clinical trials, the timing of these events, the indications we intend to pursue, the potential benefits available to us from orphan drug designations for AG10, and our possible clinical or other business strategies, are forward-looking statements. Forward-looking statements can be identified by terms such as “believes,” “expects,” “plans,” “potential,” “would” or similar expressions and the negative of those terms. These forward-looking statements are based on our management’s current beliefs and assumptions about future events and on information currently available to management.

Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These risks include, but are not limited to, risks and uncertainties related to: our limited operating history and historical losses, our liquidity to fund the development of our other product candidates through current and future milestones, our ability to raise additional funding to complete the development and any commercialization of our product candidates, our dependence on the success of our lead product candidate, AG10, results from our clinical trials and pre-clinical studies and those of third parties working in the same area as our product candidate and our dependence on third parties in connection with our manufacturing, clinical trials and pre-clinical studies. Additional risks and uncertainties that could affect our future results are included in the section titled “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2018, which is available on the SEC’s website at www.sec.gov and our website at eidostx.com. Additional information on potential risks will be made available in other filings that we make from time to time with the SEC. In addition, any forward-looking statements contained in this press release are based on assumptions that we believe to be reasonable as of this date. Except as required by law, we assume no obligation to update these forward-looking statements, or to update the reasons if actual results differ materially from those anticipated in the forward-looking statements.

Media Contact:

Carolyn Hawley, Canale Communications, (619) 849-5382, carolyn@canalecomm.com

For Investors

Alex Gray, Burns McClellan, (212) 213-0006, agray@burnsmc.com

Data Published in Nature Communications Demonstrates CoA Therapeutics’ Potential to Treat Genetically Driven Pantothenate Kinase-Associated Neurodegeneration (PKAN)

PALO ALTO, Calif., Oct. 23, 2018 /PRNewswire/ — CoA Therapeutics, a company developing small molecules that target Coenzyme-A for patients with PKAN and other diseases of CoA sequestration, today announced key data published by researchers from St. Jude Children’s Research Hospital in Nature Communications demonstrating the potential of small molecule activators of pantothenate kinase for the treatment of pantothenate kinase-associated neurodegeneration (PKAN) and other diseases involving CoA sequestration. In addition, the company announced that it has nominated the development candidate molecule and will commence IND-enabling studies. Finally, CoA announced the formation of a scientific advisory board that will help guide future development.

PKAN is a neurological disorder that arises from mutations in the human PANK2 gene, which codes for an enzyme called pantothenate kinase 2 (PanK2). The mutations lead to progressive difficulty with movement, speech and vision, usually beginning in childhood. There are currently no treatments approved for PKAN, which typically results in death in early adulthood.

The study, authored by St. Jude researcher Suzanne Jackowski, Ph.D., describes the discovery of a new class of compounds, the pantazines, that are designed to allosterically modulate pantothenate kinases, allowing PanK enzymes to make more CoA than they would typically make in an untreated system. The specific tool compound used in the study, PZ-2891, was found to be orally bioavailable and cross the blood brain barrier, a critical characteristic for treating neurological disorders like PKAN. In addition, in a mouse model of brain CoA deficiency, animals on PZ-2891 therapy gained weight, had improved locomotor activity and a longer life span, suggesting the compound could be a novel treatment approach for PKAN. For her role in advancing research in the field, Dr. Jackowski was awarded the Lippman Medal from the International Association for Cellular Coenzymes in July 2018.

“Dr. Jackowski’s novel approach to modulating PanK enzymes is the scientific foundation of CoA Therapeutics. In this study, we see the potential of novel allosteric modulators of PanK in increasing brain CoA levels resulting in phenotypic correction in knock-out mice. These compounds have been designed to be highly brain penetrating which is critical for diseases primarily affecting the brain such as PKAN,” said Shafique Virani, M.D., chief executive officer for CoA Therapeutics. “We will be moving rapidly to translate this exciting science to support entry of this therapeutic approach into clinical trials in PKAN and other diseases of CoA sequestration.”

Dr. Jackowski, who is a member of the faculty of St. Jude Children’s Research Hospital, will be joining CoA’s scientific advisory board. In addition to Dr. Jackowski, Hyder Jinnah, M.D., professor at Emory University School of Medicine; Nicola Longo, M.D., Ph.D., professor of pediatrics at the University of Utah, and Vernon R. Sutton, M.D., professor of molecular and human genetics at Baylor College of Medicine will join the advisory board.

About CoA Therapeutics 
CoA Therapeutics, a subsidiary of BridgeBio Pharma, is developing small molecules as targeted therapeutics for PKAN and other diseases involving CoA sequestration. The company’s mission is to improve and prolong the lives of patients suffering from PKAN and other CoA-implicated genetic disorders. Founded in 2017, CoA Therapeutics is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring a safe, effective treatment to market as quickly as possible. To learn more, visit www.coatherapeutics.com.

QED Therapeutics Presents Data for Infigratinib in Cholangiocarcinoma in Late Breaking Abstract at the European Society of Medical Oncology 2018 Congress

SAN FRANCISCO, Oct. 19, 2018 /PRNewswire/ — QED Therapeutics today announced the presentation of positive, updated interim data of infigratinib (BGJ398), an orally administered, selective fibroblast growth factor receptor (FGFR) 1-3 tyrosine kinase inhibitor, for the treatment of advanced or metastatic cholangiocarcinoma (bile duct cancer) during a late-breaking poster discussion at the European Society for Medical Oncology (ESMO).

In the open-label trial, which enrolled 71 patients with FGFR2 fusions/translocations, the confirmed overall response rate (cORR) was 26.9% (95% CI 16.8-39.1%) for all patients with the potential for confirmation (n=67) and an additional 56.7% experienced stable disease as their best response, resulting in a disease control rate of 83.6%. For patients who had received one or fewer prior treatment regimens, the cORR was 39.3% (n=28), whereas patients who had received two or more treatment regimens had a cORR of 17.9%. Median progression free survival was 6.8 months (95% CI 95% CI 5.3-7.6) and median overall survival was 12.5 months (95% CI 9.9-16.6 months).

“These updated data provide the most extensive experience for an FGFR inhibitor in advanced cholangiocarcinoma,” said Milind Javle, M.D., professor, gastrointestinal medical oncology, The University of Texas MD Anderson Cancer Center and investigator on the Phase 2 infigratinib (BGJ398) study. “The high disease control rate and impressive overall survival are promising in a disease with no currently approved targeted treatments.”

“These results show that infigratinib has strong potential to make a real difference in the lives of people with cholangiocarcinoma,” said Susan Moran, M.D., M.S.C.E., chief medical officer of QED Therapeutics. “Importantly, we have initiated a pivotal, Phase 3 study in first-line cholangiocarcinoma in the hopes of offering patients an upfront chemotherapy-free treatment option.”

The study also demonstrated that infigratinib-associated toxicity is manageable. Most common treatment-emergent adverse events (TEAE) were hyperphosphatemia (73.2%), fatigue (49.3%), stomatitis (45.1%), alopecia (38.0%) and constipation (35.2%). Grade 3/4 TEAEs occurred in 47 patients (66.2%), including hypophosphatemia (14.1%), hyperphosphatemia (12.7%) and hyponatremia (11.3%).

Cholangiocarcinoma affects approximately 8,000 to 10,000 patients a year in the United States. Currently, treatment options are limited, and survival rates are generally poor.

FGFR alterations have been implicated as an oncogenic driver across multiple solid tumors and hematological cancers – including roughly one out of every five cases of cholangiocarcinoma and urothelial carcinoma. Activating mutations in FGFRs are also found in multiple forms of pediatric skeletal dysplasias, including achondroplasia, which affects up to one out of every 15,000 live births.

A Phase 3 study comparing infigratinib to standard of care chemotherapy for first line cholangiocarcinoma has been initiated by QED Therapeutics.

Full poster available at: www.QEDTx.com/ESMO2018/

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, an orally administered FGFR1-3 tyrosine kinase inhibitor that has shown meaningful clinical activity in patients with chemotherapy-refractory cholangiocarcinoma with FGFR2 fusions and advanced urothelial carcinoma with FGFR3 genomic alterations. QED is also evaluating infigratinib in preclinical studies for the treatment of achondroplasia and other skeletal dysplasias. We plan to conduct further clinical trials to evaluate infigratinib in additional FGFR-driven tumor types and rare disorders.

PellePharm Presents Updated Data from Two Phase 2 Studies Demonstrating the Potential of Patidegib Topical Gel to Treat Basal Cell Carcinomas in Patients with Gorlin Syndrome and Patients with Non-Gorlin Sporadic BCCs

SAN FRANCISCO–(BUSINESS WIRE)–PellePharm, a late clinical-stage biopharmaceutical company committed to targeting rare skin conditions at their source, today presented updated clinical data from two Phase 2 studies of patidegib topical gel in a poster session at NORD’s Rare Diseases and Orphan Products Breakthrough Summit 2018 in Washington, D.C. Results of the studies showed clinical clearance and prevention of basal cell carcinoma (BCC) tumors after 6 months of treatment with patidegib topical gel in patients with Gorlin Syndrome, a rare genetic disease; and both clinical and histologic clearance after 3 months of treatment in patients with sporadic, nodular BCCs.

“We are pleased by these positive results showing the potential of patidegib topical gel in preventing new facial BCCs in patients with Gorlin Syndrome, potentially reducing the number of surgeries and resulting facial scarring that patients experience,” said Sanuj Ravindran, M.D., president and chief executive officer of PellePharm. “Based on these Phase 2 findings, we intend to initiate a randomized, one-year registrational Phase 3 trial of patidegib topical gel at the end of 2018 in patients with Gorlin Syndrome, and we also plan to study patidegib topical gel in non-Gorlin patients with high-frequency BCCs.”

Title: Hedgehog Inhibition by Topical Patidegib Reduces Facial BCC Burden in Patients with Gorlin Syndrome
Date and Time: Monday, October 15 – Tuesday, October 16, 2018
Poster Number: #71

The data presented at the NORD Summit are from two Phase 2 trials of topical patidegib gel – a randomized, double-blind, placebo-controlled, study in patients with Gorlin Syndrome in the UK and in a study in patients with sporadic, nodular BCCs in the US. In the Gorlin Phase 2 trial, 17 patients with a total of 85 surgically eligible BCCs (SEBs) applied patidegib topical gel (administered in strengths of 2% and 4%) or a topical vehicle control twice daily for 6 months to their face for prevention and to 5 SEBs for treatment.

The Phase 2 Gorlin trial showed that for prevention of surgically-eligible BCCs (SEBs), patients in the topical control group developed an average of 1.4 new SEBs in 6 months. In contrast, patients treated with patidegib topical gel 2% and 4% developed only 0.4 SEBs in Intent to Treat analysis (p=0.096) and only 0.3 SEBs in Per-Protocol analysis (p=0.008). Additionally, clinical clearance of tumors was observed in 27% (12 out of 45 SEBs) of the patidegib topical gel treated subjects compared to no tumors with clinical clearance in subjects in the vehicle group (N=16 SEBs, P=0.02).

In the U.S. trial, 36 non-Gorlin patients with sporadic, nodular BCCs applied patidegib topical gel 2%, 4%, or topical control to BCCs for 3 months. Use of patidegib topical gel 2% was significantly more effective in clinical and histologic clearance of BCCs after 3 months compared with the topical vehicle gel (p=0.045). This finding correlated with a decrease in hedgehog biomarker in BCC tumors after three months in patients using patidegib topical gel.

Across both studies, patients using topical patidegib gel did not experience any of the significant side effects characteristic of oral hedgehog inhibitors (e.g., hair loss, taste loss or frequent muscle cramps). Patients treated with patidegib topical gel 4% experienced mild skin irritation (e.g., redness, itching and swelling), but those using patidegib topical gel 2% did not.

About Patidegib

Patidegib topical gel has shown early promise in a Phase 2 clinical study for the mitigation of BCC tumors in Gorlin Syndrome by blocking the disease at its source within the hedgehog signaling pathway. Topical patidegib gel was developed to provide the efficacy previously demonstrated by oral patidegib in Phase 1 trials without the adverse systemic side effects. Patidegib’s gel formulation is stable at room temperature for at least two years, making it a viable potential therapy for ongoing, at-home management of Gorlin Syndrome. Patidegib has received both Orphan Drug Designation and Breakthrough Therapy Designation from the FDA.

About Gorlin Syndrome

Gorlin Syndrome is a rare, genetic, disease where patients are born with mutations in 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, often on the face.

With no FDA-approved drugs available for Gorlin Syndrome BCCs, the standard of care 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 (BCCs)

High-Frequency BCC, like Gorlin Syndrome, is a rare disease which is characterized by the development of an abnormally high number of BCCs. Unlike patients with Gorlin Syndrome, patients with high-frequency BCC are not born with a germline PTCH1 mutation and do not suffer from the other systemic manifestations of Gorlin Syndrome. The standard of care for patients with high-frequency BCC is surgery.

About PellePharm

Founded by world leaders in hedgehog pathway signaling, PellePharm is a biotechnology company committed to targeting rare, genetic skin diseases, including Gorlin Syndrome and High-Frequency Basal Cell Carcinomas (BCCs), at their source. PellePharm’s mission is to improve the quality of life for those suffering from Gorlin Syndrome and High-Frequency BCCs by providing an easy-to-use topical gel that eliminates the need for regular, painful surgeries. Patidegib topical gel is a first-in-class topical formulation of a proprietary hedgehog inhibitor.

Contacts

W2O Group
Peter Duckler, 310-774-3425
pduckler@w2ogroup.com

Eidos Therapeutics to Present Phase 2 Data for AG10 in TTR Amyloidosis Cardiomyopathy at the AHA 2018 Scientific Sessions in a Late-Breaking Featured Science Oral Presentation

SAN FRANCISCO, Oct. 05, 2018 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced that results of its Phase 2 clinical trial studying AG10 in subjects with symptomatic transthyretin (TTR) amyloidosis cardiomyopathy (ATTR-CM) will be announced in a late-breaking featured science oral presentation at this year’s American Heart Association (AHA) Scientific Sessions.

Daniel Judge, M.D., professor in the division of cardiology at the Medical University of South Carolina, will discuss the data in a presentation entitled “Safety, Tolerability and Transthyretin Stabilization by AG10: A Phase 2, Randomized, Double-blind, Placebo-controlled Clinical Trial in Patients with Transthyretin Amyloid Cardiomyopathy and NYHA Class II/III Heart Failure” as part of the New Trials in Cardiovascular Care oral session at 9:00 AM CT on November 10, 2018.

Eidos will host an investor conference call and webcast following the presentation to highlight details of the Phase 2 data. Details for the call and webcast will be announced at a later date.

About AG10

AG10 is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to amyloidosis, or ATTR. AG10 is currently being examined in an open-label extension of a Phase 2 clinical trial in patients with ATTR cardiomyopathy.

AG10 was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a “rescue mutation” because it has been shown to prevent ATTR in individuals carrying pathogenic, or disease-causing, mutations in the TTR gene. To our knowledge, AG10 is the only TTR stabilizer in development that has been observed to mimic the “super-stabilizing” properties of this rescue mutation.

About Eidos Therapeutics

Eidos Therapeutics is a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR). For more information, please visit www.eidostx.com.

Media Contact:
Carolyn Hawley
Canale Communications 619-849-5382
Carolyn@canalecomm.com
Investor Contact:
Alex Gray
Burns McClellan 212-213-0006
agray@burnsmc.com