Eidos Therapeutics Presents Data from its Phase 1 Clinical Trial of AG10 at the 22nd Annual Scientific Meeting of the Heart Failure Society of America

SAN FRANCISCO, Sept. 17, 2018 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX) announced the presentation of results from its Phase 1 clinical trial of AG10 during a poster session at the 22nd Annual Scientific Meeting of the Heart Failure Society of America (HFSA). The poster, entitled “AG10, A Novel, Potent, and Selective Transthyretin Stabilizer, Is Well-Tolerated at Doses Resulting in Target Therapeutic Blood Levels, and Demonstrates Clinical Proof-of-Concept in Healthy Volunteers,” was presented on Saturday, September 15, and is accessible through the science section of the company’s website.

In this initial Phase 1 study in healthy adult volunteers, AG10 was well-tolerated with no safety signals of potential clinical concern identified. At the highest tested dose, AG10 achieved 100% transthyretin (TTR) stabilization at peak concentration and over 95% TTR stabilization on average at steady state. Serum TTR concentrations, an in vivo indicator of TTR stability, were increased to a greater degree in AG10-treated patients than placebo-treated patients from baseline to Day 12.

“We believe that maximizing the level of TTR stabilization will lead to optimal clinical benefit for TTR Amyloidosis (ATTR) patients. This initial human trial demonstrated that AG10 stabilized TTR in established ex vivo assays and increased circulating TTR concentrations,” noted Jonathan C. Fox, M.D., Ph.D., president and chief medical officer of Eidos. “Given that below normal levels of serum TTR are associated with poorer prognosis in ATTR patients, and stabilization of TTR by protective mutations or small molecule stabilizers increase serum TTR concentrations, we believe that these data provide clinical proof of concept. We are rapidly moving forward with additional studies of AG10 and have already completed enrollment in our Phase 2 trial in patients with symptomatic ATTR cardiomyopathy (ATTR-CM).”

TTR normally circulates in the blood as a four-part molecule, or tetramer. In ATTR, the tetramer is destabilized by inherited mutation or age-related factors and dissociates into individual monomers, which can aggregate and are deposited as amyloid fibrils in various tissues. AG10, Eidos’ lead product candidate, is designed to target ATTR at its source by stabilizing tetrameric TTR in the blood. Eidos is pursuing AG10 for the treatment of 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 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 transthyretin amyloidosis, or ATTR. AG10 is currently being tested in a Phase 2 clinical trial in patients with symptomatic ATTR cardiomyopathy. Top-line results from this trial are expected to be reported in the fourth quarter of 2018.

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.

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 top-line data therefrom, the initiation of Phase 3 clinical trials, 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 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 the final

prospectus for our initial public offering filed with the SEC on June 21, 2018, and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2018, which are 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

Eidos Therapeutics Announces Publication of AG10 Molecular Design in Journal of Medicinal Chemistry

SAN FRANCISCO, Aug. 23, 2018 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX) today announced publication of the design and preclinical characterization of its lead product candidate, AG10, in the Journal of Medicinal Chemistry.

AG10 is designed to prevent progression of transthyretin (TTR) amyloidosis (ATTR) by stabilizing tetrameric TTR in the blood. AG10 is currently being evaluated in a Phase 2 study in patients with symptomatic ATTR cardiomyopathy (NCT03458130).

“Stabilizing transthyretin has been validated as an approach for treating ATTR based on the correlation of genetic mutations with disease progression and the success of previous clinical trials in the disease. A comparison of various TTR stabilizers and their potency based on structural evidence has not been previously conducted,” said Mamoun Alhamadsheh, PhD, co-founder of Eidos and associate professor at the University of the Pacific (Stockton, CA). “We believe our results provide strong evidence that AG10’s potentially superior stabilizing activity is driven by its unique ability to mimic the disease-protective T119M mutation and its selectivity for binding TTR in the plasma.”

The published paper demonstrates that AG10’s binding to TTR is driven by molecular interactions that mimic the structure of the T119M TTR variant that is known to protect carriers from developing ATTR and leads to prolonged lifespan in otherwise healthy individuals. Using in vitro and ex vivo assays, the data indicated that:

  • AG10 demonstrated more potent activity than other clinical candidates in binding and stabilizing the TTR tetramer at clinically relevant concentrations,
  • the binding of AG10 to TTR was not significantly affected by the presence of additional plasma proteins such as albumin, and
  • AG10 exhibited a predictable relationship between pharmacokinetics and pharmacodynamics in animal studies.

“This newly published paper describes the preclinical support for AG10 and further validates AG10’s mechanism to potently and selectively stabilize TTR,” said Uma Sinha, PhD, chief scientific officer of Eidos. “As a result, AG10 has the potential to be a disease-modifying therapy for ATTR, which we are evaluating in our ongoing Phase 2 study in symptomatic ATTR cardiomyopathy patients.”

About AG10

AG10 is an 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. Top-line results from this trial are expected to be reported by the end of 2018.

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.

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 statements about the clinical and therapeutic potential of AG10, the completion of our ongoing Phase 2 clinical trial of AG10 in patients with symptomatic ATTR cardiomyopathy and availability of top-line data therefrom, 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 and any commercialization of any of our product candidates, our dependence on the success of our current 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

Eidos Therapeutics Announces Closing of Initial Public Offering and Exercise in Full of the Underwriters’ Option to Purchase Additional Shares

SAN FRANCISCO, June 22, 2018 /PRNewswire/ — Eidos Therapeutics, Inc. (Nasdaq: EIDX), a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR), today announced the closing of its initial public offering of 7,187,500 shares of common stock, including the exercise in full of the underwriters’ option to purchase 937,500 additional shares of common stock, at a public offering price of $17.00 per share. The aggregate gross proceeds to Eidos from the offering were approximately

$122.2 million, before deducting underwriting discounts and other offering expenses. All of the shares in the offering were offered by Eidos. Eidos’ common stock is listed on The NASDAQ Global Select Market under the ticker symbol “EIDX.”

J.P. Morgan Securities LLC and BofA Merrill Lynch acted as joint book-running managers for the offering. Barclays Capital Inc. also participated as a joint book-running manager.

The shares were offered by Eidos pursuant to a registration statement that was declared effective by the Securities and Exchange Commission (“SEC”) on June 19, 2018. A prospectus relating to and describing the terms of the offering has been filed with the SEC and is available on the SEC’s website at www.sec.gov.

This offering was made only by means of a prospectus. Copies of the final prospectus relating to this offering can be obtained from (1) J.P. Morgan Securities LLC, Attention: c/o Broadridge Financial Solutions, Attn: Prospectus Department, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (866) 803-9204, or by emailing prospectus-eq_fi@jpmchase.com; (2) BofA Merrill Lynch, NC1-004-03-43, 200 North College Street, 3rd Floor, Charlotte, NC 28255-0001, Attention: Prospectus Department, or by emailing dg.prospectus_requests@baml.com; and (3) Barclays Capital Inc., c/o Broadridge Financial Solutions, Attn: Prospectus Department, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (888) 603-5847, or by emailing barclaysprospectus@broadridge.com.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

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).

Eidos Therapeutics Announces Pricing of Initial Public Offering

SAN FRANCISCO, June 19, 2018 /PRNewswire/ — Eidos Therapeutics, Inc. (Nasdaq: EIDX), a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR), today announced the pricing of its initial public offering of 6,250,000 shares of common stock at a public offering price of $17.00 per share. All of the shares are being offered by Eidos. The shares are expected to begin trading on the Nasdaq Global Select Market on June 20, 2018 under the ticker symbol “EIDX.” The gross proceeds from the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Eidos, are expected to be approximately $106.3 million. The offering is expected to close on June 22, 2018, subject to the satisfaction of customary closing conditions. In addition, Eidos has granted the underwriters a 30-day option to purchase up to an additional 937,500 shares of common stock at the initial public offering price.

J.P. Morgan Securities LLC and BofA Merrill Lynch are acting as joint book-running managers for the offering. Barclays Capital Inc. is also participating as a joint book-running manager.

A registration statement relating to these securities was declared effective by the Securities and Exchange Commission on June 19, 2018. The offering will be made only by means of a prospectus, copies of which may be obtained from J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (866) 803-9204, or by emailing prospectus-eq_fi@jpmchase.com; BofA Merrill Lynch, NC1-004-03-43, 200 North College Street, 3rd Floor, Charlotte, NC 28255-0001, Attention: Prospectus Department, or by emailing dg.prospectus_requests@baml.com; or Barclays Capital Inc., c/o Broadridge Financial Solutions, Attn: Prospectus Department, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (888) 603-5847 or by emailing barclaysprospectus@broadridge.com.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

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).

NeuroVive Out-Licenses Targeted LHON Therapy to BridgeBio Pharma’s New Subsidiary Fortify Therapeutics

LUND, Sweden and PALO ALTO, Calif., June 18, 2018 /PRNewswire/ — NeuroVive Pharmaceutical AB and BridgeBio Pharma today jointly announced that BridgeBio has entered into an exclusive licensing agreement for a subset of succinate prodrug chemistry under NeuroVive’s NVP015 program. BridgeBio also announced that it has launched a subsidiary company Fortify Therapeutics to further develop this chemistry for local treatment of Leber’s Hereditary Optic Neuropathy (LHON), with an initial financial commitment of $20 million USD. NeuroVive’s NVP015 program for other mitochondrial disorders will continue without any changes in focus or timelines.

LHON is caused by mitochondrial DNA mutations in subunits of NADH dehydrogenase (complex I), leading to reduced oxidative phosphorylation and energy production in retinal cells. The disease predominantly affects young adults, and results in sudden onset of progressive and severe vision loss. The licensed succinate prodrugs have the potential to overcome the disease by bypassing the dysfunctional metabolic pathway, providing an alternate source of energy to the retinal cells.

“As a targeted treatment for a genetic disease, the LHON program is a clear fit with the BridgeBio model,” said Neil Kumar, Ph.D., CEO of BridgeBio. “We have been impressed with the ability of these compounds to rescue specific genetic mitochondrial deficiencies, and we have assembled a team of international experts to further develop a subset of the NVP015 chemistry to address this devastating disease.”

Fortify Therapeutics will develop selected lead compounds derived from NeuroVive’s novel NVP015 succinate prodrug program into drug candidates for the localized treatment of LHON. These compounds have been selected because they have properties that make them suitable for delivery to the eye.

The licensing agreement for this particular subset of the NVP015 program has a total deal value of approximately $60 million USD, which includes limited initial funding for research, and later milestone payments and a single digit royalty stream, that are dependent on successful development and market approval.

“The agreement with BridgeBio is important to both NeuroVive and our innovative NVP015 program, as it validates the quality of the program, our business development model and potential in a variety of mitochondrial disorders,” commented NeuroVive CEO Erik Kinnman, M.D., Ph.D. “We will work closely with BridgeBio to further develop this chemistry subset and make the LHON program successful. It is important to note that our intentions for the NVP015 program are unchanged, and we are progressing towards experimental proof-of-principle during 2018.”

This information is information that NeuroVive Pharmaceutical AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out below, at 08:30 a.m. CET on 18 June 2018.

About LHON
Leber’s Hereditary Optic Neuropathy (LHON) is a disease caused by mitochondrial DNA mutations in subunits of NADH dehydrogenase (complex I), a component of the electron transport chain. This results in dysfunctional oxidative phosphorylation and ATP production, leading to degeneration of the retinal ganglion cells and loss of central vision. LHON most commonly affects males in their second or third decade of life. The prevalence of LHON in Europe is between 1:30,000-1:50,000.

About NVP015
One of the most common causes of mitochondrial diseases relates to Complex I dysfunction, i.e. when energy conversion in the first of the five protein complexes in the mitochondrion that are essential for effective energy conversion does not function normally. This is apparent in disorders including Leigh’s Syndrome and MELAS, both of which are very serious diseases with symptoms such as muscle weakness, epileptic fits and other severe neurological manifestations. The NVP015 project is based on a NeuroVive innovation in which the body’s own energy substrate, succinate, is made available in the cell via a prodrug technology. A prodrug is an inactive drug that is activated first when it enters the body by the transformation of its chemical structure. Results from the NVP015 project were published in the prestigious Nature Communications journal in August 2016.

About BridgeBio Pharma
BridgeBio is a privately held 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, based in Palo Alto, CA, has built a robust portfolio of nineteen transformative assets, each housed in its own subsidiary, ranging from pre-clinical to late stage development in multiple therapeutic areas including oncology, cardiology, neurology, dermatology and endocrinology. The company’s focus on scientific excellence and rapid execution aims to translate today’s discoveries into tomorrow’s medicines.

About NeuroVive
NeuroVive Pharmaceutical AB is a leader in mitochondrial medicine, with one project in clinical phase II development for the prevention of moderate to severe traumatic brain injury (NeuroSTAT®) and one project in clinical phase I (KL1333) for genetic mitochondrial diseases. The R&D portfolio consists of several late stage research programs in areas ranging from genetic mitochondrial disorders to cancer and metabolic diseases such as NASH. The company’s strategy is to advance drugs for rare diseases through clinical development and into the market. The strategy for projects within larger indications outside the core focus area is out-licensing in the preclinical phase.