PTC Therapeutics
Oct 12, 2017

Spinal Muscular Atrophy Program Advances into Pivotal Study in SMA Patients

- Concluded dose-finding part of SUNFISH study -
- Milestone triggers $20 million payment to PTC from Roche -

SOUTH PLAINFIELD, N.J., Oct. 12, 2017 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) transitioned into the pivotal part of the study evaluating the efficacy and safety of RG7916 in pediatric and adult Type 2/3 SMA patients. RG7916 is an oral survival motor neuron 2 (SMN2) splicing modifier. The study, named SUNFISH, consists of two parts, an exploratory dose finding part (Part 1) for 12-weeks which has been concluded - and a confirmatory part (Part 2) for 24-months. The initiation of Part 2 triggers a $20 million milestone payment to PTC from Roche. SMA is a rare genetic disorder that results in neuromuscular disability and is the leading genetic cause of mortality in infants and young children.

"We are excited to move RG7916 into the pivotal part of the SUNFISH trial," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "RG7916 resulted in a substantial increase in SMN protein production in SMA patients. We believe that a major advantage of RG7916 is that it is an oral drug that distributes throughout the body. This is important because the SMN protein is critical both in the CNS and peripheral tissues."

The confirmatory part of SUNFISH is a randomized, double-blinded, placebo-controlled study. It will be conducted in approximately 168 Type 2 and Type 3 SMA patients for up to 24 months, followed by an open-label extension. The primary objective of the study is to evaluate the efficacy of RG7916 compared to placebo after 12 months of treatment.

The U.S. Food and Drug Administration granted orphan drug designation to RG7916 for the treatment of patients with SMA earlier this year. RG7916 directly targets the underlying molecular deficiency of SMA by modulating SMN2 splicing to increase expression of full-length SMN2 mRNA from the SMN2 gene. An interim analysis from the first part of SUNFISH of the five cohorts treated with RG7916 for 28 days or longer demonstrated an exposure-dependent increase in SMN protein. SMA is characterized by reduced levels of SMN protein, motor neuron loss, and muscle atrophy. To date, RG7916 remains well-tolerated in patients at all doses and there have been no drug-related safety findings leading to withdrawal.

The SMA program was initially developed by PTC Therapeutics in partnership with the SMA Foundation in 2006 to accelerate the development of a treatment for SMA. In November 2011, Roche gained an exclusive worldwide license to the PTC/SMA Foundation SMN2 alternative splicing program. The development of these compounds is being executed by Roche and overseen by a joint steering committee with members from PTC, Roche, and the SMA Foundation.

About PTC Therapeutics, Inc.
PTC is a global biopharmaceutical company focused on the discovery, development, and commercialization of novel medicines using our expertise in RNA biology. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders and oncology. PTC has discovered all of its compounds currently under development using its proprietary technologies. Since its founding nearly 20 years ago, PTC's mission has focused on developing treatments to fundamentally change the lives of patients living with rare genetic disorders. The company was founded in 1998 and is headquartered in South Plainfield, New Jersey. For more information on the company, please visit our website

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Forward Looking Statements:
All statements, other than those of historical fact, contained in this press release, are forward-looking statements, including statements regarding: any advancement of the joint development program in SMA with PTC, Roche, and SMAF, in particular as related to the timing of enrollment, completion and evaluation of the Phase 2 clinical studies of RG7916 in SMA patients and the period during which the results of the studies will become available; the clinical utility and potential advantages of RG7916, including its potential to impact every aspect of the disease; the timing and outcome of PTC's regulatory strategy and process; PTC's strategy, future expectations, plans and prospects, future operations, future financial position, future revenues or projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "potential," "will," "promise," "expect," "plan," "target," "anticipate," "believe," "estimate," "intend," "may," "project," "possible," "would," "could," "should," "continue," and similar expressions.

PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the initiation, enrollment, conduct and availability of data from either the SUNFISH or FIREFISH studies and the outcome of such studies; events during, or as a result of, these studies that could delay or prevent further development of RG7916, including future actions or activities under the SMA joint development program; our expectations for regulatory approvals; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products, including with respect to PTC's joint development program in SMA with Roche and the SMAF. There are no guarantees that any product candidate under the joint development program will receive regulatory approval in any territory or prove to be commercially successful.

The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

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