Below is a sample of entries from the
<font> BioMedReports.com FDA Calendar </font> , which includes a database of over 200 entries. The companies outlined below have expected FDA new drug product decisions or clinical trial results expected over the next three months of summer. The list also includes some
<font> Extreme Trades </font> of companies with market caps below $250 million that have pending clinical trial results or FDA decisions that could have a major stock price impact.
Rigel Pharma (NASDAQ:RIGL): On 5/5/09, RIGL reported its quarterly financial results and stated that the Company expects to deliver top-line results in July 2009 from its TASKi2 and TASKi3 Phase 2b clinical trials, which add over 670 additional patients studied with lead compound R788 (given by oral administration) in rheumatoid arthritis (many of the treatments for this condition are given by injection which is not a popular route of administration) to the 189 patients studied from the Company’s Phase 2a trial results reported in December 2007 (check out a two-year stock price chart to see the major upside impact of these results).
On 6/1/09, RIGL announced that its oral Syk inhibitor, R788, is also being evaluated in a Phase 2 clinical trial funded, designed and implemented by the National Cancer Institute (NCI), part of the U.S. National Institutes of Health. This open-label, single arm clinical trial will include patients with advanced colorectal, thyroid, non-small cell lung, hepatocellular, head and neck, or renal cell cancers who have failed to respond to at least one line of therapy. In addition, the company has an ongoing Phase 2 clinical trial of R788 in patients with peripheral T-cell lymphoma and reported favorable results from a Phase 2 clinical trial of R788 in the treatment of patients with certain B-cell lymphomas in June 2008.
Aeterna Zentaris (NASDAQ:AEZS): On 6/15/09, AEZS reported that patient follow-up in the open-label safety study (study 041) of its Phase 3 program in benign prostatic hyperplasia (BPH) with its lead endocrinology compound, cetrorelix pamoate, is scheduled to be completed at the end of the week. Therefore, data analysis and reporting will be brought forward from the scheduled fourth quarter into the third quarter of 2009, and will follow the disclosure of results from the first double-blind placebo controlled efficacy study (study 033).
Cetrorelix is currently in three Phase 3 trials involving more than 1,600 patients with symptomatic BPH in Canada, the United States and Europe. First efficacy results are expected during 3Q09 with a NDA filing targeted in 2010. AEZS has also announced recently that patients completing two years of therapy with cetrorelix in the first efficacy study (study 033), will be eligible to continue with the cetrorelix treatment, according to treatment regimen of the ongoing Phase 3 study, until the end of 2011. Patients entering this extension study sponsored by the Company's partner Sanofi-Aventis (NYSE:SNY), will be followed-up for safety, International Prostate Symptom Score (IPSS) and quality of life during the extended treatment, providing follow-up data on cetrorelix for up to 5 years.
Insmed (NASDAQ:INSM): On 5/13/09, INSM reported its quarterly operating results, including an update on the clinical development of IPLEX, for which the Company continues to expect preliminary Phase 2 results in the treatment of Myotonic Muscular Dystrophy (MMD) during 2Q09. The FDA will also allow Insmed to provide access to IPLEX for investigational use in patients with Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's disease), and the Company will conduct a controlled clinical trial under an Investigational New Drug (IND) application after finalizing the study protocol with the FDA.
Jazz Pharma (NASDAQ:JAZZ): On 6/15/09, JAZZ announced that sodium oxybate (JZP-6) demonstrated statistically significant and clinically meaningful improvement in pain and the core symptoms associated with fibromyalgia, according to Phase 3 data presented last week. JAZZ has also completed its second Phase 3 clinical trial of JZP-6 and expects to announce top-line results from this study around mid-2009. Assuming positive results for the second study, the company anticipates submitting a NDA for sodium oxybate for the treatment of fibromyalgia to the FDA by the end of 2009. UCB SA (EBR:UCB) (PINK:UCBJF) anticipates filing in the EU shortly after. UCBJF.PK has the exclusive marketing and distribution rights to sodium oxybate for fibromyalgia in Europe and some other countries outside North America and will manage registrations accordingly.
Human Genome Sciences (NASDAQ:HGSI): On 6/11/09, HGSI announced Phase 2 continuation data which demonstrated sustained improvement in disease activity and patient response rate through four years of BENLYSTA therapy in combination with standard of care in patients with serologically active systemic lupus erythematosus (SLE). The frequency of disease flares as measured by the SELENA SLEDAI flare index and by BILAG A or B organ domain scores decreased over four years of BENLYSTA therapy and there was no increase in overall adverse events, serious adverse events, malignancies or serious infections over time.
HGSI and GlaxoSmithKline (NYSE:GSK) have selected BENLYSTA as the brand name for belimumab (formerly known as LymphoStat-B). HGS and GSK expect to report the first Phase 3 data for BENLYSTA in July 2009 from the BLISS-52 trial, with results from BLISS-76 anticipated in November 2009. BLISS-52 and BLISS-76 are the largest clinical trials ever conducted in lupus patients. BENLYSTA is being developed by HGSI and GSK under a co-development and commercialization agreement entered into in August 2006.
Takeda Pharma (TYO:4502) (PINK:TKPHY): Pending alogliptin (SYR-322) NDA for the proposed treatment of Type 2 Diabetes. Alogliptin is a dipeptidyl peptidase IV (DPP-4) inhibitor being reviewed as an adjunct to diet and exercise for the treatment of Type 2 diabetes. Pharmaceutical Product Development (NASDAQ:PPDI) receives $25 million if alogliptin receives FDA approval. Although the alogliptin NDA was filed prior to issuance of FDA's December 2008 guidance on new Type 2 diabetes treatments, the FDA announced that it will apply these guidelines when reviewing the alogliptin NDA. Additionally, the FDA does not believe that the amount of existing alogliptin clinical data is sufficient to meet certain statistical requirements in the new guidance. The agency is open to discussions regarding the design of additional cardiovascular (CV) studies with alogliptin.
Alogliptin's PDUFA action date remains unchanged at 6/26/09. In December, 2008 the FDA issued "Guidance for Industry: Diabetes Mellitus -- Evaluating Cardiovascular Risk in New Antidiabetic Therapies to Treat Type 2 Diabetes". In October 2008, Takeda received notification from the FDA that it was unable to complete its review of the alogliptin NDA by the original PDUFA date of 10/27/08 due to internal resource constraints. The FDA did not raise any issues with the data in the alogliptin NDA at that time. In December 2007, Takeda submitted its NDA for alogliptin to the FDA.
AMAG Pharma (NASDAQ:AMAG): On 4/30/09, AMAG reported that the FDA was satisfied with observations noted during a recent inspection of the Company's manufacturing facility. AMAG is awaiting a decision by the agency for its iron replacement therapy, Feraheme (ferumoxytol injection), to treat iron deficiency anemia in patients with chronic kidney disease (CKD). AMAG also noted that a re-inspection of its manufacturing facility will not be required as a pre-requisite to FDA approval of Feraheme. AMAG has previously issued guidance on labeling discussions, which are likely to include a broad label that includes both dialysis and non-dialysis patients with CKD. On 5/12/09, AMAG announced the FDA accepted the Company’s resubmission of its Feraheme™ (ferumoxytol injection) New Drug Application in response to the December 2008 Complete Response letter as a complete, Class 1 response with a PDUFA date of 6/29/09.
King Pharma (NYSE:KG) and Acura Pharma (NASDAQ:ACUR): Acurox (oxycodone + niacin) NDA (priority review). Acurox is an immediate release opioid analgesic, has a proposed indication for relief of moderate-to-severe pain and is designed to deter common methods of misuse and abuse. The expected PDUFA action date for a FDA decision is 6/30/09.
Spectrum Pharma (NASDAQ:SPPI): Zevalin (Ibritumomab Tiuxetan) (a radio-immunotherapeutic that specifically targets and kills cancer cells) sBLA to expand FDA-approved label as a first-line consolidation therapy (following a response to first-line treatments) for non-Hodgkin's lymphoma (NHL). On 2/23/09, a three-month PDUFA action date delay to 7/2/09 was announced as SPPI submitted additional data to the FDA, which was classified as a major amendment by the agency.
Labopharm (NASDAQ:DDSS): DDSS has a pending NDA for a rapid-onset formulation of trazodone (DDS-04A) filed through the 505(b)(2) pathway as a new formulation of an existing drug for the proposed treatment of major depressive disorder with an estimated PDUFA action date of 7/18/09 for a possible FDA decision.
United Therapeutics (NASDAQ:UTHR): UTHR announced on 4/28/09 that the FDA has extended the PDUFA action date for the Company’s pending Tyvaso (inhaled treprostinil) NDA. The new PDUFA date is 7/30/09 and UTHR expected the delay (which was announced in a PR in mid-March) due to the submission of additional clinical trial data to the FDA, which was considered a major amendment by the agency. The additional data was related to human factors testing to validate the instructions for using the Optineb nebulizer device which delivers the medication.
Auxilium Pharma (NASDAQ:AUXL): On 4/28/09, the FDA accepted for priority review (six-month) Auxilium's BLA for Xiaflex (clostridial collagenase for injection) for the treatment of Dupuytren's contracture (this is a progressive condition that affects the connective tissue that lies beneath the skin in the palm, resulting in collagen deposits which impair normal hand function). AUXL filed the BLA on 2/27/09 and included data from 1,082 subjects and over 2,600 injections. The PDUFA action date is 8/28/09 for the Xiaflex BLA. If approved by the FDA, AUXL plans to launch Xiaflex in the U.S. within 60 days during 2H09.
On 2/3/09, BioSpecifics Technologies (NASDAQ:BSTC) received $6.375 million of the $75 million upfront payment paid to AUXL by Pfizer (PFE) and will receive 8.5% of the $410 million in potential additional milestone payments that may be made by PFE to AUXL. Of these additional milestone payments, $150 million are tied to regulatory milestones and $260 million are based on sales milestones. In addition, BSTC will continue to receive milestone and royalty payments from AUXL in accordance with its original agreement for Xiaflex.
ISTA Pharma (NASDAQ:ISTA): On 6/1/09, ISTA announced the Dermatologic and Ophthalmic Drugs Advisory Committee will review ISTA's New Drug Application (NDA) for Bepreve(TM) (bepotastine ophthalmic solution) on June 26, 2009. On 11/13/08, ISTA announced it had filed its NDA with the FDA for Bepreve as an eye drop treatment for ocular itching associated with allergic conjunctivitis. As bepotastine is a new chemical entity in the U.S., ISTA expected an Advisory Committee would be convened for Bepreve. ISTA continues to expect a standard review of ten months and has been given a PDUFA decision date of 9/12/09 for the FDA to complete its review of the pending NDA.
Theravance (NASDAQ:THRX): On 4/24/09, the FDA accepted as complete for review Theravance's response to the Agency’s February 2009 Complete Response Letter (CRL), which outlined requirements for approval of telavancin for the treatment of complicated skin and skin structure infections (cSSSI). Telavancin is a novel, bactericidal, once-daily injectable investigational antibiotic studied in the treatment of cSSSI and hospital-acquired pneumonia (HAP) caused by Gram-positive bacteria, including resistant pathogens such as methicillin-resistant Staphylococcus aureus (MRSA). The FDA assigned a PDUFA action date of 9/16/09 for approval of telavancin in the treatment of cSSSI.
Allos Therapeutics (NASDAQ:ALTH): ALTH received a priority (six-month) review designation on 5/26/09 with a PDUFA action date of 9/24/09. PTCL comprises a biologically diverse group of hematologic malignancies that typically has a worse prognosis than other types of lymphoma and is less responsive to traditional chemotherapy regimens. There are currently no agents approved by the FDA for the treatment of patients with PTCL.
Disclosure: No positions.
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