BridgeBio Pharma Closes $299.2 Million Financing Round to Support Its Efforts to Target Genetic Disease at the Source

PALO ALTO, Calif.–(BUSINESS WIRE)–BridgeBio Pharma, a clinical-stage biopharmaceutical company focused on genetic diseases, today announced a new financing round of $299.2 million. The round was co-led by existing investors KKR and Viking Global Investors. Other existing investors participating included Perceptive Advisors, AIG, Aisling Capital, Cormorant Capital, and Hercules Capital; and they were joined by new investors Sequoia Capital, and a blue-chip long-term investor. The financing will be used to support BridgeBio Pharma’s existing drug research and development programs and expand its efforts to rapidly develop medicines for patients with unmet needs.

“We are privileged to be working with investors who believe in our goal of creating medicines for patients with genetic disease. We are aware that many of these patients lack effective treatment options, and we take our mission to help them seriously,” said Neil Kumar, Ph.D, co-founder and chief executive officer of BridgeBio Pharma. “The path from promising early-stage science to a drug that makes a difference for patients requires a long-term vision and steady commitment. We are fortunate to have our investors’ support as we develop these treatments.”

Genetic diseases are conditions that derive directly from mutations in their patients’ DNA. These mutations can be either inherited or spontaneous, and the diseases stemming from them include both Mendelian diseases, which tend to affect pediatric patients, and cancers. While there are more than 7,000 genetic diseases affecting 25 to 30 million Americans in aggregate, fewer than 500 drugs are approved for these conditions, leaving a significant number of patients without any therapeutic options.

BridgeBio Pharma was formed in 2015 by a team of drug research and development veterans from both the biotech industry and academia. The company seeks to translate novel scientific discoveries from universities, academic medical centers, and pharmaceutical research groups into genetically-targeted therapeutics that address the fundamental causes of disease. Its portfolio of more than 15 assets includes several in the pre-clinical stages of development, as well as four programs in or approaching pivotal trials.

Each of these drug assets is housed in its own subsidiary company, with access to centralized resources and capabilities courtesy of a novel corporate structure developed in conjunction with Dr. Andrew Lo of MIT’s Sloan School of Management. BridgeBio employs a lean, capital-efficient model that harnesses a central research and development platform to simultaneously operate multiple programs that fit the company’s stringent science criteria. Using this model, personnel and funding can be efficiently redistributed amongst the assets on an as-needed basis. The portfolio assets span therapeutic areas including genetic dermatology, oncology, cardiology, neurology, endocrinology, renal disease, and ophthalmology. Some of the specific indications targeted include transthyretin amyloidosis (ATTR-CM and ATTR-PN), pantothenate kinase-associated neurodegeneration (PKAN), Gorlin syndrome and frequent basal cell carcinomas, dystrophic epidermolysis bullosa (DEB), Darier and Hailey-Hailey diseases, Netherton syndrome, venous malformations, Canavan disease, Leber’s hereditary optic neuropathy, molybdenum cofactor deficiency Type A, achondroplasia, and FGFR, SHP-2, and K-RAS-driven cancers.

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 portfolio of more than 15 transformative drugs ranging from pre-clinical to late stage development in multiple therapeutic areas including genetic dermatology, oncology, cardiology, neurology, endocrinology, renal disease, and ophthalmology. 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.

Contacts

Ariana Romio
aromio@theoutcastagency.com

QED Therapeutics Announces a Collaboration with Foundation Medicine to Develop Companion Diagnostics for Infigratinib

SAN FRANCISCO, Dec. 10, 2018 /PRNewswire/ — QED Therapeutics today announced that it has entered into an agreement with Foundation Medicine to develop a companion diagnostic for infigratinib, an FGFR1-3 selective tyrosine kinase inhibitor, in patients with cholangiocarcinoma. The companion diagnostic, which will include detection of activating FGFR2 fusions, is expected to be incorporated into FoundationOne®CDx, Foundation Medicine’s FDA-approved comprehensive genomic profiling (CGP) assay for all solid tumors that includes multiple companion diagnostics.

“While targeted therapies have transformed the treatment landscape for multiple cancers, such as lung cancer, cholangiocarcinoma has not yet benefitted from these advances,” noted Susan Moran, M.D., M.S.C.E., chief medical officer of QED Therapeutics. “Given infigratinib’s demonstrated promise in FGFR2 fusion-driven cholangiocarcinoma, a companion diagnostic is critical to help physicians reliably identify which patients might be candidates for treatment.”

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 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.

Eidos Therapeutics Appoints William Lis to Board of Directors

SAN FRANCISCO, Dec. 06, 2018 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), a clinical stage biopharmaceutical company focused on addressing the large unmet need in transthyretin (TTR) amyloidosis (ATTR), today announced the appointment of William Lis, to Eidos’ Board of Directors, effective December 4, 2018. Mr. Lis has more than 25 years of experience in the biopharmaceutical industry, including serving as chief executive officer of Portola Pharmaceuticals, Inc.

“Bill is an accomplished biopharmaceutical executive with a proven track record in building and leading organizations, and has significant expertise in successfully developing new therapies in the field of cardiology,” said Neil Kumar, PhD., chief executive officer of Eidos. “We are pleased to welcome Bill to our Board of Directors. Bill’s extensive experience will serve us well as we continue to advance the clinical development of AG10, a potentially disease-modifying therapy for the treatment of ATTR.”

“With the Phase 3 study of AG10 expected to start in 2019, this is an important time for Eidos,” said Mr. Lis. “I look forward to leveraging my experience in building biopharmaceutical organizations and developing novel therapies to help Eidos achieve its mission of developing a potentially best-in-class treatment for ATTR patients.”

William Lis has more than 25 years of biopharmaceutical experience. He served as Chief Executive Officer and a director of Portola Pharmaceuticals, Inc. from 2010 until 2018 after serving as Chief Operating Officer and Chief Business Officer in 2009 and 2008, respectively. Under his leadership, Portola successfully grew from a discovery stage company to a fully integrated R&D and commercial organization, and independently advanced: Andexxa® and Bevyxxa®, which received FDA Breakthrough and Fast Track designation, respectively, from discovery and early stage development through commercial launch, and cerdulatinib, a novel immunology and oncology compound, from discovery through Phase 2 clinical development. He led private and public financings including an initial public offering in 2013; multiple corporate partnerships; academic clinical development collaborations; and milestones that resulted in the growth of the company’s valuation from $250 million to $2.7 billion during his tenure. Prior to Portola, Mr. Lis held executive and management positions at Scios, Inc. (a Johnson & Johnson company) from 2003 to 2008 where he last served as Vice President of Commercial Operations and Business Development, having led efforts for the in-licensing, and then the strategic development and

pre-commercial launch for Xarelto®. He also held positions of increasing responsibility at Millennium Pharmaceuticals, Inc. (previously COR Therapeutics, Inc.) from 1998 to 2003 in commercial and medical affairs for INTEGRILIN® and early stage compounds, Earlier in his career, he was involved in the U.S. commercial launch of several products with multiple pharmaceutical companies, including Lovenox® and Rilutek® while at Rhone- Poulenc Rorer. Mr. Lis serves as independent director for Zai Lab Limited (ZLAB, Nasdaq) and served as a member of the Biotechnology Innovation Organization (BIO) Board of Directors for the Emerging Companies Section in 2015 and 2016. Mr. Lis holds a B.S. from the University of Maryland.

Additionally, Eidos also announced that Hoyoung Huh, MD, PhD, who has served as a member of Eidos’s Board of Directors since March 2016, has stepped down from his role as a director of Eidos, effective as of December 4, 2018. Dr. Huh will continue as a consultant to Eidos through his leadership at BridgeBio Pharma LLC, the parent company and majority shareholder of Eidos.

“We are grateful for Hoyoung’s service to Eidos,” stated Dr. Kumar. “His leadership and guidance have been instrumental in the progress we have achieved at Eidos.”

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. In a Phase 2 clinical trial in ATTR-CM patients, 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. AG10 is currently being studied in an open-label extension of a Phase 2 clinical trial in patients with ATTR cardiomyopathy. Subject to discussions with regulatory agencies, Eidos plans to initiate Phase 3 pivotal studies of AG10 in the first half of 2019.

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, including the initiation of Phase 3 pivotal studies for 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 AG10 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

LEO Pharma and PellePharm Announce $760 Million Collaboration to Advance Innovative Therapies for Rare Skin Diseases

SAN FRANCISCO & BALLERUP, Denmark–(BUSINESS WIRE)– Danish dermatology specialists LEO Pharma and California rare disease pioneers PellePharm today announced a strategic development and commercialization collaboration to address unmet medical needs across various skin diseases with no approved treatments, advancing innovation and access to potential therapies for patients with life-altering conditions, such as Gorlin Syndrome and High Frequency Basal Cell Carcinoma (BCC), two distinct and rare forms of skin cancer.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20181120005227/en/

“We are very excited about the partnership with PellePharm, who are pioneers in Gorlin Syndrome and experts in rare skin diseases. Supporting our ambitious 2025 strategy, it marks LEO Pharma’s entrance in rare skin diseases and it offers a unique opportunity to bring the first treatment forward to people suffering from a very severe skin disease for which there currently are no approved therapies,” said Thorsten Thormann, vice president of research at LEO Pharma.

Under the terms of the agreement, LEO Pharma has initially committed $70 million comprised of equity financing and financial R&D support to fund the global Phase 3 trial for patidegib topical gel 2% for the prevention and treatment of Gorlin Syndrome, with LEO Pharma securing an option to acquire all shares in PellePharm. PellePharm and its stockholders could receive up to an additional $690 million including merger consideration, and regulatory and commercial milestone payments. In addition, PellePharm stockholders are eligible to receive a double-digit royalty after achieving certain commercial milestones.

“Our company is founded on the commitment to targeting rare dermatologic diseases at the source and bringing new groundbreaking treatments forward to patients as efficiently and effectively as possible. As a global leader in medical dermatology, LEO Pharma is a great fit as both a development and commercialization partner. This collaboration puts us on track to commence our pivotal Phase 3 Gorlin Syndrome trial in early 2019. Then after the potential merger, we look forward to working with LEO Pharma to address other rare skin diseases with unmet needs,” said Sanuj Ravindran, president and chief executive officer of PellePharm.

The agreement establishes a joint development committee with PellePharm maintaining responsibility for global development and LEO Pharma supporting in an advising role. Both companies will jointly drive commercialization planning, and Anders Kronborg, chief financial officer of LEO Pharma, will join PellePharm’s board of directors.

”Gorlin Syndrome Group welcomes the news that PellePharm and LEO Pharma are collaborating in developing patidegib topical as a treatment for BCCs. Data from PellePharm’s Phase 2 study suggest patidegib is effective in treating BCCs, with minimal side effects. We hope that by working with LEO, these benefits can be confirmed in a Phase 3 study. An effective topical medication, which can prevent and/or treat BCCs, will avert the need for the painful, disruptive and disfiguring surgeries we currently undergo,” said Sally Webster, chairperson and Matthew Helbert, trustee of Gorlin Syndrome Group U.K.

Currently, there are no FDA-approved therapies for Gorlin Syndrome, and the standard of care for this rare disease is surgery. Patients with this lifelong, severe disease can have as many as 30 surgeries per year beginning in their mid-teens.

PellePharm’s 2% topical formulation of patidegib aims to be the first approved therapy for the prevention of Gorlin Syndrome, also known as Basal Cell Carcinoma Nevus Syndrome (BCCNS), and has shown early promise in Phase 2 clinical trials treating patients with Gorlin Syndrome and Sporadic BCC in both the U.S. and the U.K.

“For more than 18 years, our organization has been dedicated to supporting, educating, and seeking much-needed treatments for people living with Gorlin Syndrome (BCCNS), and yet people with this devastating disease still have no FDA-approved therapies,” said Jean Pickford, executive director of the BCCNS Alliance. “With the Phase 3 study of patidegib beginning recruitment in early 2019, we continue to be encouraged by PellePharm’s dedication to our community, now in partnership with LEO Pharma.”

PellePharm was advised by Rothschild & Co. on this transaction.

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. The topical formulation of patidegib was developed to provide the efficacy previously demonstrated by oral patidegib in Phase 1 trials without the adverse systemic side effects. The 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 and High Frequency BCC. PellePharm has received both Orphan Drug Designation and Breakthrough Therapy Designation for patidegib topical gel in Gorlin Syndrome from the FDA.

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, often on the face.

With no FDA-approved drugs available for Gorlin Syndrome, 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 (BCC)

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 LEO Pharma

LEO Pharma helps people achieve healthy skin. By offering care solutions to patients in more than 130 countries globally, LEO Pharma supports people in managing their skin conditions. Founded in 1908 and owned by the LEO Foundation, the healthcare company headquartered in Denmark has devoted decades of research and development to delivering products and solutions to people with skin conditions. In 2017, LEO Pharma employed around 5,200 people worldwide and had sales of 1.4 billion euros. For more information, go to: www.leo-pharma.com / www.linkedin.com/company/leo-pharma

About PellePharm

Founded by world leaders in hedgehog pathway signaling, PellePharm, a BridgeBio Company, is committed to targeting rare, genetic dermatological diseases, including Gorlin Syndrome and High Frequency Basal Cell Carcinoma (BCC), at their source. PellePharm’s mission is to improve the quality of life for those suffering from Gorlin Syndrome and High Frequency BCC 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.

About BridgeBio Pharma

BridgeBio is a clinical-stage biotech company developing novel, genetically targeted therapies to improve the lives of patients. The BridgeBio approach combines a traditional focus on drug development with a unique corporate model, allowing rapid translation of early stage science into medicines that treat disease at its source. Founded in 2015 by a team of industry veterans, the company has built a robust portfolio of seventeen 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.

Contacts

LEO Pharma Media Contact:
Henrik Kyndlev
Leo Pharma
+45 3140 6180
henrik.kyndlev@leo-pharma.com
PellePharm Media Contact:
Lauren Barbiero
W2O Group
(310) 774-3425
lbarbiero@w2ogroup.com

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