Effective March 1, 2016 Håkan Wickholm has succeeded Unni Hjelmaas as CEO in Lytix Biopharma.
Mr. Wickholm’s appointment reflects the new phase in Lytix Biopharma’s development with increasing focus on Business Development (Press release, Lytix Biopharma, MAR 1, 2016, View Source [SID:1234509331]). Håkan Wickholm will continue as head of Business Development.

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Board Chair Gert W. Munthe says:

"The entire Board is delighted that Håkan Wickholm now takes on this role as the clinical program is starting to show substantial immune responses in patients treated with LTX-315. This lays the foundation for successful business development activities. At the same time the Board would like to express their thanks to Unni Hjelmaas for her hard work and dedication to Lytix Biopharma in her four years as CEO."

Håkan Wickholm says:

"I am very excited by this opportunity to take on the role of CEO at this stage, when important immune response data develops, allowing us to intensify business development activities and further clinical development. ’’

On March 1, 2018 FLAG Therapeutics Inc. reported that the U.S. Food and Drug Administration’s Office of Orphan Products Development and the European Medicines Agency (EMA) have both granted Orphan Drug Designation to FLAG-003 for the treatment of glioma (Press release, Flag Therapeutics, MAR 1, 2016, View Source [SID1234525570]). Gliomas (including Glioblastoma) are the most aggressive forms of brain cancer and carries a very poor prognosis for survival and is one of the deadliest forms of cancer. Two and 5-year survival rates are 27% and 10%, with the median progression-free survival (PFS) being only 6.9 months.

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Orphan status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases – those which affect fewer than 200,000 Americans annually. Orphan drug designation entitles FLAG Therapeutics to 7 years marketing exclusivity following product launch in the United States (10 years marketing exclusivity in the EU) and enables the company to apply for research funding, tax credits, a waiver from FDA (PDUFA) user fees, FDA assistance in clinical trial design, and access to the central authorization procedure within the European Union.

FLAG-003 for the treatment of Glioma

FLAG-003 is a small molecule which exerts both cytotoxic and cytostatic activity due to two distinct and well characterized mechanisms of action. It possesses cytotoxic anti-tubulin activity by binding to the colchicine site of tubulin causing microtubule depolymerization. It also possesses anti-angiogenic activity through binding and inhibition of RTK receptor tyrosine kinase (RTKs) activity. The anti-tubulin and anti-angiogenic activities of FLAG-003 have translated into potent antitumor and anti-vascular effects in vivo with significantly better inhibitory activity on GBM tumor growth and vascularization than the currently approved chemotherapy, temozolomide (TMZ).

On February 29, 2016 OPKO Health, Inc. (NYSE:OPK), a multinational biopharmaceutical and diagnostics company, reported financial and operating results for the three months and year ended December 31, 2015 (Filing, Q4, Opko Health, 2015, FEB 29, 2016, View Source [SID:1234509286]).

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Business Highlights

Clinical Utility Study Demonstrates 4Kscore Test Reduces Unnecessary Prostate Biopsies While Improving Risk Prediction for Aggressive Prostate Cancer: The results of a 611 patient peer-reviewed study, "The 4Kscore Test Reduces Prostate Biopsy Rates in Community and Academic Urology Practices", written by Badrinath Konety, MD, et al. (Reviews in Urology, January 2016), indicated that the 4Kscore test led to 65% fewer prostate biopsies being performed among participating patients and influenced approximately 89% of decisions about performing a prostate biopsy. A higher 4Kscore test result was significantly associated with a greater likelihood of having a prostate biopsy.

4Kscore Blood Test to Identify Risk of Aggressive Prostate Cancer Assigned a Level 1 CPT Code: The American Medical Association (AMA) Current Procedural Terminology (CPT) Editorial Panel granted a Category I CPT code which will be effective in January 2017 for OPKO’s 4Kscore Test, the only blood test that accurately identifies an individual patient’s risk for aggressive prostate cancer. A Category I CPT code is a designation reserved for established diagnostic tests, and will provide broader access to the 4Kscore Test to urologists and their patients across the United States.

Rayaldee PDUFA Date Remains on Track for March 29, 2016: In late 2014, OPKO announced successful top-line results from both pivotal Phase 3 trials with Rayaldee. These trials were identical randomized, double-blind, placebo-controlled, multisite studies intended to establish the safety and efficacy of Rayaldee as a new treatment for secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency.

Topline Phase 3 Results for hGH-CTP in Adults Expected 2H 2016; Pediatric Phase 3 Initiation Anticipated in 2H 2016: The Phase 3 trial in adults is designed to evaluate the safety and efficacy of hGH-CTP with a primary endpoint of superiority compared with placebo in decreasing fat mass in adults with GHD. The trial is a randomized, double-blind, placebo-controlled, multicenter, global study in adults with growth hormone deficiency (GHD). The study is divided into two treatment periods: a 26-week, double blind, placebo-controlled period, followed by a 26-week, open-label extension. A Phase 3 trial in pediatric patients is anticipated to commence in 2H 2016.

First Patient Dosed in Phase 2a Clinical Trial of Long Acting Factor VIIa for the Treatment of Hemophilia: In February 2016, the first patient was dosed in OPKO’s Phase 2a clinical trial of its long acting Factor VIIa, a dose escalation study to determine safety and explore efficacy endpoints of OPKO’s long acting form of coagulation Factor VIIa (Factor VIIa-CTP) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX. The study will enroll 24 patients in the United States.

Clinical Studies for Long Acting Oxyntomodulin for Obesity and Diabetes Expected to Begin: OPKO expects to commence studies for its long acting subcutaneous oxyntomodulin for diabetes and obesity in Q1 2016.

VARUBITM (Rolapitant) was Approved by the FDA on September 2, 2015 and Commercial Launch Commenced in November 2015: OPKO’s partner, TESARO received FDA approval of oral VARUBI, a neurokinin-1 (NK-1) receptor antagonist, in combination with other antiemetic agents in adults for the prevention of delayed nausea and vomiting associated with initial and repeat courses of emetogenic chemotherapy. TESARO commenced commercial sales in the U.S. in November 2015 and expects to submit its Marketing Authorization Application to the European Medicines Agency for oral VARUBI in Q2. VARUBI has been included in the NCCN Guidelines as a recommended option in combination with other antiemetic agents for patients receiving both high emetic risk intravenous chemotherapy (HEC) and moderate emetic risk intravenous chemotherapy (MEC). Category 1, the highest level category of evidence and consensus, was granted to VARUBI for both HEC and MEC chemotherapy. OPKO is eligible to receive up to $95 million in additional milestones and tiered, double-digit royalties.

Varubi (Rolapitant) IV Formulation NDA Submission. TESARO expects to submit NDA for IV formulation of Varubi in Q1.

"We made great progress during 2015," stated Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO. "We have recently begun to expand the marketing and sales of our 4Kscore Test utilizing the commercial infrastructure of Bio-Reference, with very promising results. As we obtain broad reimbursement for the 4Kscore Test, we believe patients, providers and insurers will realize the benefits of this test in 2016 and beyond. We are now preparing for our launch of Rayaldee for treatment of SHPT in patients with stage 3 or 4 CKD later this year. Our commercial partner, TESARO, launched VARUBI late in 2015 and the launch is progressing as expected. Our collaboration with Pfizer is on track and we expect to have topline data for our ongoing Phase 3 clinical trial for hGH-CTP in adults later this year and expect to initiate the pivotal clinical trial for the pediatric indication later this year. We continue to pursue other development programs as well and, earlier this month, initiated a clinical study utilizing our CTP platform technology for our long-acting Factor VIIa project. We expect to begin a Phase 1 clinical trial for long acting oxyntomodulin next month," Dr. Frost continued.

Financial Highlights

Consolidated revenues increased to $276.2 million from $25.5 million for the three months ended December 31, 2015 compared to the 2014 period, and increased to $491.7 million from $91.1 million for the year ended December 31, 2015 as compared to 2014. The 2015 periods include revenue from Bio-Reference and EirGen beginning with their acquisitions in August and May 2015, respectively. Revenue for the three months and year ended December 31, 2015 also includes $17.7 million and $65.5 million, respectively, from OPKO’s collaboration with Pfizer. The 2015 periods also include a $15.0 million milestone payment from TESARO related to the launch of VARUBI in November 2015.

Net income for the three months ended December 31, 2015 was $1.6 million compared with a net loss of $53.0 million for the 2014 period and net losses for the year ended December 31, 2015 decreased to $30.0 million compared to $171.7 million for the 2014 period. In addition to the revenue related items, the 2015 three month and year periods include significant non-recurring and non-cash activities, including:

• $26.5 million and $113.7 million of income tax benefit, respectively, reflecting the release of valuation allowances against all of OPKO’s U.S.-based deferred tax assets as a result of the Bio-Reference acquisition in 2015;
• $15.9 million gain related to the deconsolidation of OPKO’s previously consolidated variable interest entity, SciVac, in the year ended December 31, 2015 as SciVac completed an initial public offering by merger with Levon Resources Ltd. in July 2015;
• $25.9 million of non-recurring operating expense related to the repayment of a grant to the Office of the Chief Scientist in Israel related to the Pfizer transaction in the nine month period of 2015; and,
• Other income and (expense) of ($15.9) million and ($39.5) million primarily related to the change in fair value of derivative instruments as well as a ($7.3) million temporary impairment charge of an available for sale investment in the three months and year ended December 31, 2015, respectively, compared with ($20.9) million and ($25.2) million in the 2014 periods. The change in fair value is principally related to an embedded derivative in our January 2013 convertible senior notes due in 2033.
Cash, cash equivalents and marketable securities were $193.6 million as of December 31, 2015.
• This reflects receipt of Pfizer upfront payments of $295.0 million, partially offset by a $94.7 million cash payment for the acquisition of EirGen (net of EirGen’s cash on hand) and a one-time $25.9 million payment to the Office of the Chief Scientist in Israel related to the Pfizer transaction and cashed used by operations, principally related to our research and development expense.

On FEBRUARY 29, 2016 BioMotiv, a drug development accelerator associated with The Harrington Project, and Rutgers, the State University of New Jersey, reported the formation of a new biotechnology startup, Z53 Therapeutics. Z53 Therapeutics aims to develop novel anti-cancer drugs that target tumors with p53 mutations (Press release, Z53 Therapeutics, FEB 29, 2016, View Source [SID:1234509511]).

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Z53 Therapeutics is based on intellectual property exclusively licensed from Rutgers University and SUNY Upstate Medical University in Syracuse, N.Y., which was developed by the laboratory of scientific founder Darren Carpizo, M.D., Ph.D., Surgical Oncologist at The Rutgers Cancer Institute of New Jersey. The work was done in collaboration with the synthetic chemistry laboratories of Rutgers Translational Sciences, headed by S. David Kimball, Ph.D., Associate Vice President Research Translation and Commercialization, and the biochemistry and molecular biology laboratory of Stewart Loh, Ph.D., at SUNY Upstate.

"I am very excited to collaborate with BioMotiv to advance our research from our initial discoveries and mechanistic work to the identification of compounds that can be used in the clinic to target a wide spectrum of tumors with specific p53 mutations", said Dr. Carpizo.

The p53 protein has been called the "the guardian of the human genome" for its central role in suppressing tumor formation. The gene encoding p53 is the most commonly mutated gene in human cancer with a large number of mutations resulting in a defect in the protein’s structure due to an impairment in the ability of the protein to bind zinc (so-called zinc deficient mutant p53). Dr. Carpizo and colleagues discovered that small molecules could restore the normal structure and function to zinc deficient mutant p53. Dr. Carpizo, along with Dr. Loh, went on to elucidate the mechanism of action of these compounds to be a restoration of zinc binding in mutant p53 which they have named "zinc metallochaperones".

Zinc metallochaperones represent a novel class of anti-cancer drugs. "The pharmacologic restoration of structure and function of a mutant protein by the delivery of a metal ion is unprecedented in drug development," said Dr. Carpizo. Together with Drs. Loh and Kimball, the research team has identified novel zinc metallochaperones which will be developed by Z53 Therapeutics. Dr. Carpizo goes on to say, "Targeting mutant p53 with small molecule compounds has been one of the holy grails of drug development, so it is very exciting to be a forefront of this field of research."

Christopher Molloy, Ph.D., Senior Vice President for Research and Economic Development at Rutgers, said, "The rapid progress in identifying a biologically active lead chemical series provides validation for Rutgers’ investment in pre-clinical translational research, which is being led by Dr. Kimball. We are pleased that this agreement provides additional resources to allow Dr. Carpizo and our translational sciences team to advance the research on these promising discoveries."

"The discoveries made by Dr. Carpizo have great promise," said Baiju R. Shah, Chief Executive Officer of BioMotiv. "We are looking forward to partnering with him in developing those discoveries in Z53 Therapeutics."

Dr. Carpizo is a Harrington Scholar-Innovator whose work has been supported by the Harrington Discovery Institute of University Hospitals in Cleveland. His laboratory is also funded by the National Cancer Institute, Sidney Kimmel Cancer Foundation, and the Breast Cancer Research Foundation.

# # #

About BioMotiv
BioMotiv is the mission-driven accelerator associated with The Harrington Project for Discovery & Development, a $250 million national initiative for advancing medicine centered at University Hospitals in Cleveland. The focus is to accelerate breakthrough discoveries from research institutions into therapeutics for patients through an innovative model that efficiently aligns capital and collaborations. The company leverages an experienced team and advisory board to select, fund, and actively manage and advance a portfolio of drug development programs.
Learn more at www.biomotiv.com.

About Rutgers University
Rutgers, The State University of New Jersey, is a leading national research university. Established in 1766 and celebrating a milestone 250th anniversary in 2016, the university is the eighth oldest higher education institution in the United States. More than 67,000 students and 22,000 faculty and staff learn, work, and serve the public at Rutgers locations across New Jersey and around the world. Rutgers University–New Brunswick is the only public institution in New Jersey represented in the prestigious Association of American Universities. Rutgers is a member of the Big Ten Conference and its academic counterpart, the Committee on Institutional Cooperation, a consortium of 15 world-class research universities. Rutgers is among the top 30 universities nationally for total R&D funding and last year achieved an 18.3 percent increase in overall funding for research and sponsored programs over the previous year, from $517.6 million in fiscal year 2014 up to $612.5 million in fiscal year 2015. The Office of Research and Economic Development is a central point for industry to access Rutgers and offers a website designed for the business community, www.businessportal.rutgers.edu.

About SUNY Upstate
SUNY Upstate Medical University in Syracuse, NY, is the only academic medical center in Central New York and the region’s largest employer with 9,460 employees. Affiliated with the State University of New York and anchored by its four colleges—Medicine, Nursing, Health Professions and Graduate Studies (biomedical sciences), Upstate’s mission is to improve the health of the community through education, biomedical research and health care. As a biomedical research enterprise, Upstate focuses on the most prevalent human diseases, including cancer, diabetes, heart disease, nervous system disorders, vision, and infectious diseases. The quest for treatments and cures is built upon expertise in structural, molecular and systems biology. The Upstate University Health System serves 1.8 million people, often the most seriously ill and injured, and includes Upstate University Hospital; Upstate University Hospital at Community Campus; Upstate Golisano Children’s Hospital, and numerous satellite sites across Upstate New York. Research Administration at Upstate, which includes the Offices of Technology Transfer, Sponsored Programs and Clinical Trials, assists companies interested in licensing new technologies or in working with Upstate researchers or clinicians (View Source).

On February 29, 2016 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the fourth quarter and full year ending December 31, 2015 (Filing, Q4/Annual, PTC Therapeutics, 2015, FEB 29, 2016, View Source [SID:1234509287]).

"We are shocked and disappointed to have received a Refuse to File (RTF) letter from the FDA regarding our NDA for Translarna, and we are engaging in dialogue with the FDA to determine a path forward," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "Despite this recent setback, we achieved a number of key milestones in 2015 as we continue to build PTC into a global, commercial biotechnology company. Translarna had a landmark first year launch in nonsense mutation Duchenne muscular dystrophy and we are currently under regulatory review in the EU to remove the condition to the existing marketing authorization as well as expand our label to include nonsense mutation cystic fibrosis. Our Phase 3 ACT CF clinical trial is fully enrolled and our clinical stage SMA and cancer stem cell assets continue to progress. I am proud of what we accomplished in 2015. Resilience has been one of our key values since I founded PTC in 1998 and our team continues to work diligently to bring Translarna to patients globally."

Key Fourth Quarter, Full Year 2015 and other Corporate Highlights:

· Refuse to File letter received from the FDA regarding Translarna for nonsense mutation Duchenne muscular dystrophy (nmDMD). The letter from the U.S. Food and Drug Administration (FDA) received on February 22, 2016 stated that the new drug application (NDA) for Translarna was not sufficiently complete to permit a substantive review. Specifically, PTC was notified in the letter that, in the view of the FDA, both the Phase 2b and ACT DMD trials were negative and do not provide substantial evidence of effectiveness. The FDA also characterized certain of the company’s adjustments to the ACT DMD study as post hoc and therefore not supportive of effectiveness. In addition, the FDA noted that the NDA did not contain adequate information regarding the abuse potential of Translarna, a requirement for new molecules that cross the blood-brain barrier. PTC is engaging in dialogue with the FDA to discuss and clarify the matters set forth in the letter and to determine the best path forward.

· Successful first year Translarna launch with 2015 revenues of $33.7M. PTC has established a strong global commercial footprint launching the first approved therapy in Duchenne muscular dystrophy (DMD), with sales generated in 23 countries including most recently Argentina, the Czech Republic, Hungary, Portugal and Singapore. PTC is targeting to expand access to Translarna to over 35 countries by the end of 2016. Market access discussions regarding funding on a country-by-country basis are ongoing. In the UK, PTC has had constructive discussions with the National Health Services (NHS) England regarding a managed access agreement for Translarna with a decision from the National Institute for Health and Care Excellence (NICE) expected in the coming months. In Germany, PTC has had multiple discussions with the German Federal Association of the Statutory Health Insurances (GKV-SV) over the last several months to come to agreement on pricing and reimbursement. Recently, these discussions transitioned into an arbitration process, which did not lead to an acceptable agreement. As a result, PTC expects to delist Translarna from the German pharmacy ordering system. Under these circumstances, patients and healthcare professionals may be able to

access Translarna through a reimbursed importation pathway possible under German law, thus minimizing any access issues for existing and new German patients.

· Actively pursuing regulatory approvals for Translarna in DMD globally. In early January 2016, PTC submitted the ACT DMD Phase 3 results to the European Medicines Agency (EMA) in fulfillment of the principal condition of the EMA marketing authorization for the treatment of nmDMD in ambulatory patients aged five and over. The submission to the EMA, called a type II variation, seeks to remove the condition to the existing marketing authorization. PTC anticipates the Committee for Medicinal Products for Human Use (CHMP) will issue its recommendation regarding this request in mid-2016. Translarna also received regulatory approvals in both Israel and South Korea in 2015. In September 2015, Health Canada initiated an expedited review of Translarna for potential approval for nmDMD in the first half of 2016.

· ACT CF Phase 3 clinical trial on track for completion by year-end 2016 with top-line results expected early 2017. In November 2015, PTC announced that it had completed enrollment for ACT CF, the company’s second Phase 3 clinical trial of Translarna for patients with nonsense mutation cystic fibrosis (nmCF). ACT CF is a 48-week placebo-controlled Phase 3 clinical trial designed to evaluate the effect of Translarna in patients six years of age or older with nmCF not receiving chronic inhaled aminoglycosides. During the third quarter, PTC submitted a variation to its marketing authorization requesting EMA approval of Translarna for the treatment of nmCF based on the company’s previous Phase 3 study. PTC anticipates the CHMP will issue its recommendation regarding this submission in mid-2016.

· As part of 10 by ‘20 strategy, four additional indications in development for Translarna. Given its mechanism of action, Translarna has the potential to address numerous genetic disorders caused by a nonsense mutation to address significant unmet need across a spectrum of many rare diseases. In addition to its advanced DMD and CF programs, PTC is pursuing indications in mucopolysaccharidosis type I (MPS I), aniridia, and two genetically defined epilepsy disorders, Dravet syndrome and CDKL5. PTC’s goal is to investigate Translarna’s activity in a minimum of ten indications beyond DMD

and CF by 2020 in order to deliver on its commitment to patients and maximize the potential of Translarna as both a product and a pipeline.

· Internally developed pipeline continues to progress. In January 2016, clinical development of the spinal muscular atrophy (SMA) program, a collaboration with Roche and the SMA Foundation, resumed with a second compound, RG7916, beginning a Phase 1 study in healthy volunteers. In addition, PTC’s cancer stem cell program in oncology continues to advance with Phase 1 data expected in 2016. PTC’s discovery group is focused on the advancement of novel programs for rare and neglected disorders including next generation nonsense read-through, Huntington’s disease and familial dysautonomia.

· Maintained strong balance sheet with approximately $339 million in cash and cash equivalents. PTC completed a successful $150 million offering of 3.00% convertible senior notes due 2022 in August 2015, raising net proceeds of approximately $145 million. PTC finished 2015 with approximately $339 million in cash and cash equivalents.

Upcoming Events:

PTC will participate in the following upcoming conferences:

· Cowen and Company’s 36th Annual Health Care Conference on March 8 at 11:20 a.m. (ET) in Boston, MA
· Barclay’s Global Healthcare Conference on March 15 at 1:35 p.m. (ET) in Miami, FL
· Deutsche Bank’s 41st Annual Healthcare Conference on May 4-5 in Boston, MA

The presentations will be webcast live on the Events and Presentations page under the investor relations section of PTC’s website at www.ptcbio.com and will be archived for two weeks following the presentation. PTC’s current investor presentation is available at the same website location.

Fourth Quarter and Full Year 2015 Financial Highlights:

· Translarna net product sales were $12.7 million for the fourth quarter of 2015, representing 30% sequential growth versus $9.8 million in the third quarter of 2015. For the full year 2015, Translarna generated $33.7 million in net product sales compared to $0.7 million in the prior year.

· Total revenues for the fourth quarter of 2015 were $12.7 million compared to $12.7 million in the same period of 2014. Total revenues for 2015 were $36.8 million compared to $25.2 million for the same period of 2014. The change in total revenue was a result of the expanded commercial launch of Translarna during 2015, which received marketing authorization from the EMA in August 2014, offset by lower grant revenue.

· Non-GAAP R&D expenses were $31.4 million for the fourth quarter of 2015, excluding $3.7 million in non-cash, stock-based compensation expense, compared to $23.7 million for the fourth quarter of 2014, excluding $3.2 million in non-cash, stock-based compensation expense. GAAP R&D expenses were $35.0 million for the fourth quarter of 2015 compared to $26.9 million for the fourth quarter of 2014. For the full year 2015, non-GAAP R&D expenses were $105.7 million, excluding $16.1 million in non-cash, stock-based compensation expense, compared to $70.1 million for 2014, excluding $9.7 million in non-cash, stock-based compensation expense. For the full year 2015, GAAP R&D expenses were $121.8 million compared to $79.8 million in the prior year period. The increase in R&D expense for the fourth quarter and year ended December 31, 2015, as compared to the prior year periods was primarily due to expansion of our clinical development activities including late-stage studies and extension programs in both Duchenne muscular dystrophy and cystic fibrosis.

· Non-GAAP SG&A expenses were $21.7 million for the fourth quarter of 2015, excluding $4.2 million in non-cash, stock-based compensation expense, compared to $14.5 million for the fourth quarter of 2014, excluding $3.5 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $25.9 million for the fourth quarter of 2015 compared to $18.0 million for the fourth quarter of 2014. For the full year 2015,

non-GAAP SG&A expenses were $64.2 million, excluding $17.8 million in non-cash, stock-based compensation expense, compared to $35.2 million for 2014, excluding $9.6 million in non-cash, stock-based compensation expense. GAAP full-year 2015 SG&A expenses were $82.1 million compared to $44.8 million in 2014. The increase in SG&A expense for the fourth quarter and year ended December 31, 2015, as compared to the prior year periods primarily resulted from additional costs associated with commercial activities in support of the launch of Translarna for DMD.

· Net loss for the fourth quarter of 2015 was $50.9 million compared to a net loss of $27.3 million for the same period in 2014. Net loss for the full year 2015 was $170.4 million compared to $93.8 million for the same period in 2014.

· Cash, cash equivalents, and marketable securities totaled approximately $339 million at December 31, 2015 compared to approximately $315 million at December 31, 2014. This increase includes net proceeds of approximately $145 million from a $150 million convertible debt offering completed in the third quarter of 2015.

· Shares issued and outstanding as of December 31, 2015 were 34.3 million, which includes 0.3 million shares of unvested restricted stock.

2016 Guidance:

· Total ex-U.S. Translarna nmDMD revenues for 2016 are anticipated to be between $65 and $85 million. This guidance assumes current exchange rates and the continued commercial expansion for Translarna in nmDMD outside of the U.S.

· Operating expenses for 2016 are currently under review as a result of the Refuse to File letter recently received from the FDA.

Non-GAAP Financial Measures

In this press release, PTC’s financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results exclude stock-based compensation expense. These results are provided as a complement to results reported in GAAP because management believes these non-GAAP financial measures are the best indication of the company’s business.

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