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5/12/06 — PTC Therapeutics Inititates Phase 1A Clinical Trial of PTC299
4/21/06 — PTC Therapeutics Announces Promotion of Cláudia Hirawat
4/4/06 — PTC Therapeutics Announces Interim Phase 2 Results of PTC124 in Cystic Fibrosis
3/31/06 — PTC Therapeutics Files for Initial Public Offering
3/30/06 — PTC Therapeutics Receives Fast Track Designation for PTC124 in the Treatment of Duchenne Muscular Dystrophy
3/20/06 — Schering-Plough and PTC Therapeutics Announce Collaboration for Development of PTC's Preclinical Hepatitis C Compounds
PTC THERAPEUTICS INITIATES PHASE 1A CLINICAL TRIAL OF PTC299
SOUTH PLAINFIELD, N.J., May 12, 2006 — PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery
and development of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes,
today announced that it had commenced a Phase 1a clinical trial of the company's product candidate PTC299 in healthy
volunteers. The trial is being conducted in Belgium. PTC299 was discovered through PTC's proprietary Gene Expression
Modulation by Small-molecules (GEMS) technology.
This Phase 1a trial is a single-site, randomized, double-blind, placebo-controlled escalating single-dose safety and
pharmacokinetic study in healthy volunteers between the ages of 18 and 55. The primary objective of this trial is
to determine a dose range for PTC299 that is well tolerated, achieves pharmacologically active plasma concentrations
and is appropriate for use in a subsequent Phase 1b multiple-dose study. PTC expects to complete this trial in the
second quarter of 2006. If this trial is successful, PTC plans to initiate further clinical trials of PTC299 in solid
tumor cancer patients.
ABOUT PTC THERAPEUTICS, INC.
PTC is a biopharmaceutical company focused on the discovery and development of orally administered, proprietary
small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes
regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC
has assembled proprietary technologies and extensive knowledge of post-transcriptional control processes that it
applies in its drug discovery and development activities. PTC's current pipeline of clinical and preclinical
product candidates addresses multiple indications, including genetic disorders, oncology, and infectious diseases.
ABOUT PTC299
PTC299 is a novel, orally administered small-molecule compound designed to inhibit the production of the protein vascular
endothelial growth factor, or VEGF, in tumors. PTC has designed PTC299 to inhibit VEGF production in tumors by targeting
the post-transcriptional control processes that regulate VEGF formation. Because PTC299 inhibits VEGF production, its
action occurs at a different point in the VEGF pathway than other therapies that target the binding of VEGF to its
receptors on the surface of blood vessel cells.
FOR MORE INFORMATION:
Jane Baj
PTC Therapeutics, Inc.
(908) 222-7000, x167
jbaj@ptcbio.com
Sheryl Seapy
Pure Communications
(949) 608-0841
sheryl@purecommunicationsinc.com
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PTC THERAPEUTICS ANNOUNCES PROMOTION OF CLÁUDIA HIRAWAT
SOUTH PLAINFIELD, N.J., April 21, 2006 — PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the
discovery, development, and commercialization of orally administered, proprietary small-molecule drugs that target post-transcriptional
control processes, today announced the promotion of Cláudia Hirawat to Senior Vice President of Corporate Development.
"I am extremely pleased to announce this promotion. Cláudia has made significant contributions to our company, providing leadership
across multiple areas including leading our efforts in establishing our business collaborations," commented Stuart Peltz, Ph.D.,
President and CEO of PTC Therapeutics, Inc.
Ms. Hirawat was most recently Vice President of Corporate Development at PTC. Her responsibilities focused on business development, public
relations and advocacy group relations. In her new role as Senior Vice President of Corporate Development, Ms. Hirawat will focus on
business development, advocacy group relations and commercial development.
Ms. Hirawat joined PTC in 2000 as the company's seventh employee. Prior to PTC, Ms. Hirawat was a Vice President at LedbetterStevens, a
management consulting and senior-level retained search firm in New York focused exclusively in the biopharmaceutical area.
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PTC THERAPEUTICS ANNOUNCES INTERIM PHASE 2 RESULTS OF PTC124 IN CYSTIC FIBROSIS
SOUTH PLAINFIELD, N.J., April 4, 2006 — PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and
commercialization of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes, today announced
interim results from Phase 2 clinical trials of PTC124 in patients with cystic fibrosis (CF) due to a nonsense mutation. These interim data
suggest that PTC124 may have pharmacological activity that addresses the underlying cause of CF in these patients.
PTC124 is an orally delivered investigational product candidate that PTC is developing for the treatment of genetic disorders due to nonsense
mutations. Nonsense mutations are alterations in the genetic code that prematurely halt the translation process, producing a shortened, non-functional
protein. Phase 2 clinical trials are ongoing in the two initial indications being pursued, CF and Duchenne muscular dystrophy (DMD) caused by nonsense mutations.
PTC is conducting two comparable Phase 2 clinical trials of PTC124 in CF, one at the Hadassah University Hospital in Jerusalem, Israel, and
the other at four sites in the US (University of Alabama at Birmingham Hospital and Clinics, AL; Johns Hopkins Hospital, Baltimore, MD;
Rainbow Babies' and Children's Hospital, Cleveland, OH; and Denver Children's Hospital, Denver, CO). In each study, patients receive two
sequential two-week courses of treatment, first at a lower and then at a higher PTC124 dose level.
The primary endpoint of these trials is the change in the CFTR chloride channel activity (also known as chloride conductance). Chloride
conductance is evaluated using a standardized nasal transepithelial potential difference (TEPD) procedure. Cystic fibrosis patients
lack sufficient CFTR protein and therefore have an abnormal TEPD chloride conductance.
Fifteen patients have completed two cycles of treatment and data from these patients were available for inclusion in the interim analysis. Of
these patients, three were from the U.S. trial and 12 were from the Israeli trial. All patients had a nonsense mutation and multiple signs
and symptoms of CF, with most patients having lung dysfunction, chronic bacterial infection of the lungs, pancreatic insufficiency, and low body weights.
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PTC THERAPEUTICS FILES FOR INITIAL PUBLIC OFFERING
SOUTH PLAINFIELD, N.J., March 31, 2006 — PTC Therapeutics, Inc. today announced that the company has filed with the Securities
and Exchange Commission a registration statement relating to the proposed initial public offering of shares of its common stock. All
shares of the common stock to be sold in the offering will be sold by PTC Therapeutics, Inc.
Morgan Stanley & Co. Incorporated will act as sole book-runner and lead manager, J.P. Morgan Securities Inc. will act as co-lead manager and
Pacific Growth Equities, LLC will act as co-manager in the offering.
A registration statement relating to these securities has been filed with the Securities and Exchange Commission but has not yet become
effective. These securities may not be sold nor may offers to buy be accepted prior to the time the registration statement becomes
effective. When available, a preliminary prospectus may be obtained from Morgan Stanley & Co. Incorporated, by emailing
prospectus@morganstanley.com, or by contacting the prospectus department at Morgan Stanley & Co. Incorporated, 1585 Broadway,
New York, New York 10036, by telephone at 212-761-6775.
ABOUT PTC THERAPEUTICS, INC.
PTC is a biopharmaceutical company focused on the discovery, development, and commercialization of orally administered, proprietary
small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the
rate and timing of protein production and are of central importance to proper cellular function. PTC has assembled proprietary
technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and
development activities. PTC's current pipeline of clinical and preclinical product candidates addresses multiple indications,
including genetic disorders, oncology, and infectious diseases.
CONTACT:
Jane Baj
PTC Therapeutics, Inc.
(908) 222-7000, x167
jbaj@ptcbio.com
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PTC THERAPEUTICS RECEIVES FAST TRACK DESIGNATION FOR PTC124 IN THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
SOUTH PLAINFIELD, N.J., March 30, 2006 — PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization
of small-molecule drugs that target post-transcriptional control processes, today announced that the company has been granted Fast Track designation from the
United States Food and Drug Administration (FDA) for the development of PTC124 for the treatment of Duchenne muscular dystrophy (DMD) due to a nonsense mutation
in the dystrophin gene. In December 2004, PTC124 was granted Orphan Drug designation by the FDA for the treatment of DMD. PTC124 is currently in Phase 2 clinical
trials in DMD and cystic fibrosis (CF) in cases in which a nonsense mutation is the cause of the disease. PTC expects to complete these Phase 2 clinical trials in the second half of 2006.
The Fast Track program is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious
or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Development programs receiving Fast
Track designations typically receive FDA priority review (6-month vs. standard 10-month review).
"Fast Track designation in DMD is an additional important element in the development of PTC124," said Stuart W. Peltz, Ph.D., President and CEO of PTC. "DMD is
an unmet medical need where only palliative options are currently available. We hope PTC124 will represent a therapeutic option for patients with DMD due to a nonsense mutation."
ABOUT PTC THERAPEUTICS, INC.
PTC is a biopharmaceutical company focused on the discovery, development, and commercialization of orally administered, proprietary small-molecule drugs
that target post-transcriptional control processes. Post-transcriptional control processes are of central importance to the regulation of the rate and
timing of cellular protein production. PTC has assembled proprietary technologies and extensive knowledge of post-transcriptional control processes that
it applies in its drug discovery and development activities. PTC's current pipeline of clinical and preclinical product candidates addresses multiple
indications, including genetic disorders, oncology, and infectious diseases.
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SCHERING-PLOUGH AND PTC THERAPEUTICS ANNOUNCE COLLABORATION FOR DEVELOPMENT OF PTC'S PRECLINICAL HEPATITIS C COMPOUNDS
KENILWORTH, N.J., and SOUTH PLAINFIELD, N.J., March 20, 2006 — Schering-Plough Corporation (NYSE: SGP) and PTC Therapeutics, Inc. (PTC) today announced that they have entered
into an exclusive collaboration and licensing agreement for the development of PTC's preclinical compounds for the oral treatment of hepatitis C virus (HCV) infection and other
viral diseases. PTC's small molecules are designed to inhibit the HCV Internal Ribosome Entry Site (IRES) mediated production of viral proteins. The IRES is highly conserved
among all HCV genotypes and is required for the expression of all viral proteins involved in replication of the hepatitis C virus.
PTC identified the compounds in its HCV program through the company's proprietary Gene Expression Modulation by Small-molecules (GEMS) technology.
"The goal of this alliance is to develop new oral therapies to improve treatment for patients with hepatitis C, one of the most serious and common blood borne
infections in the world," said Catherine D. Strader, Ph.D., executive vice president, discovery research, Schering-Plough Research Institute. "Schering-Plough
is continuing its leadership in hepatitis C through internal research programs and strategic collaborations with innovative companies such as PTC, with a focus
on developing targeted viral inhibitors," she said.
"We are delighted to enter into this collaboration with Schering-Plough, as our teams share a commitment to develop new HCV therapies that will benefit patients," said Stuart Peltz,
president & CEO of PTC Therapeutics. "Schering-Plough's expertise in the research and development of antiviral therapies and its global hepatitis franchise affords
PTC the opportunity to realize the full potential of our IRES inhibitors. This collaboration provides additional validation for the GEMS technology and nicely complements
our strategy as we advance internally the development of our clinical programs."
Under the terms of the agreement, PTC and Schering-Plough will conduct a joint research program, and Schering-Plough will be
responsible for development and commercialization efforts worldwide. Schering-Plough will make an upfront payment to PTC of $12
million and provide funding for PTC's research efforts. Additionally, PTC can earn milestone payments if specific development,
regulatory and commercial goals are achieved. Total payments to PTC could exceed $200 million. Schering-Plough will receive
exclusive worldwide commercialization rights for any approved products and pay PTC royalties on worldwide net sales.
PTC's proprietary Gene Expression Modulation by Small-molecules (GEMS) technology exploits the regulatory mechanisms found in the untranslated regions of messenger RNA
for the identification of small molecule drugs that can treat diseases by selectively increasing or decreasing the expression of key proteins.
Chronic hepatitis C is estimated to affect more than 10 million people in major world markets, including in Canada, Europe and the United States. It is a leading
cause of chronic liver disease and one of the most common reasons for liver transplant.
PTC Therapeutics is a privately-held biopharmaceutical company focused on the discovery, development and commercialization of small-molecule drugs targeting post-transcriptional
control mechanisms. Post-transcriptional control processes are the sequence of events in the cell that ultimately regulate the rate and timing of all protein production. PTC
discovers and develops small-molecule drugs that alter these processes by selectively modulating how RNA is used to produce proteins. This approach enables PTC to advance
its drug discovery programs rapidly from targets to preclinical and clinical drug candidates, building a pipeline across multiple therapeutic areas including genetic
disorders, oncology and infectious diseases.
Schering-Plough is a global science-based health care company with leading prescription, consumer and animal health products. Through internal research and collaborations
with partners, Schering-Plough discovers, develops, manufactures and markets advanced drug therapies to meet important medical needs. Schering-Plough's vision is to
earn the trust of the physicians, patients and customers served by its more than 32,000 people around the world. The company is based in Kenilworth, N.J., and
its Web site is www.schering-plough.com.
SCHERING-PLOUGH DISCLOSURE NOTICE: The information in this press release includes certain "forward-looking statements" within the meaning of the Securities
Litigation Reform Act of 1995, including statements relating to the company's strategy and the potential development of and market for drugs that treat
hepatitis C. Forward-looking statements relate to expectations or forecasts of future events. Schering-Plough does not assume the obligation to update any
forward-looking statement. Many factors could cause actual results to differ materially from Schering-Plough's forward-looking statements, including market
forces, economic factors, product availability, patent and other intellectual property protection, current and future branded, generic or over-the-counter
competition, the regulatory process, and any developments following regulatory approval, among other uncertainties. For further details about these and other
factors that may impact the forward-looking statements, see Schering-Plough's Securities and Exchange Commission filings, including Item 1A. Risk Factors in the Company's 2005 10-K.
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