Pending Clinical Trial Results: Ten Extreme Trades

by: Mike Havrilla

As a follow-up to my recent article on nine extreme FDA trades of pending new product decisions among small and micro-cap companies, I have examined the Clinical Trial Calendar database to find companies with market caps below $250M which have pending Phase 2 or 3 clinical trial results expected before the end of 2009.

Below is a sample of 10 stocks meeting this criteria from the database of 111 entries included in the Clinical Trial Calendar, which is in addition to the 120 entries in the FDA Calendar database as of 5/13/09.

Amicus Therapeutics (NASDAQ:FOLD): Market Cap = $174M, Net Cash = $108M. FOLD expects to release Phase 2 clinical trial results during late 3Q09 for Plicera in the treatment of Gaucher disease. The Company also plans to announce the final Phase 3 clinical trial protocol and begin Phase 3 clinical development for Amigal (migalastat) in the treatment of Fabry disease during 2Q09.
Avanir Pharma (NASDAQ:AVNR): AVNR completed targeted enrollment on 3/16/09 of patients into the STAR trial, a pivotal Phase 3 trial of Zenvia (dextromethorphan/quin...) in patients exhibiting signs and symptoms of pseudobulbar affect. The randomized, multi-center, international STAR trial is designed to compare the effects of Zenvia 30/10 mg, Zenvia 20/10 mg and placebo on involuntary crying/laughing episodes rates. AVNR expects to release topline data no later than end of 3Q09 and plans to submit a complete response to the FDA approvable letter during 1H10.
Arena Pharma (NASDAQ:ARNA): On 3/30/09, ARNA announced Phase 3 clinical trial results for its weight loss drug lorcaserin. While the results partly met FDA benchmarks, they still fell shy of a proposed FDA guideline that calls for at least a 5% point difference in weight loss between the test drug and placebo. The average weight loss for lorcaserin patients was 5.8% (12.7 pounds) versus 2.2% (4.7 pounds) for patients receiving a placebo for a difference of 3.6%, which is shy of the FDA's 5% standard. Arena will announce additional Phase 3 data for lorcaserin in September and still expects to file for FDA approval by year-end based on positive safety data and meeting the study objectives for weight loss.
Orexigen Therapeutics (NASDAQ:OREX): OREX has three pending Phase 3 clinical trials for its experimental weight loss drug Contrave, with results expected during 3Q09.
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 5/7/09, JAZZ reported its quarterly operating results and provided a clinical development update on JZP-6, which is the Company's sodium oxybate product currently in Phase 3 development for the treatment of fibromyalgia. JAZZ has completed its second Phase 3 pivotal trial for JZP-6 and expects to announce the results in mid-2009. JAZZ also provided guidance on submitting a NDA for JZP-6 by the end of 2009 seeking FDA approval in the treatment of fibromyalgia.
Clinical Data (NASDAQ:CLDA): CLDA has a pending second Phase 3 trial for vilazodone, and the Company anticipates reporting results from this second study by the end of June. In addition, CLDA expects to submit a New Drug Application (NDA) for vilazodone by the end of 2009 for the treatment of major depressive disorder.
Poniard Pharma (NASDAQ:PARD): On 5/5/09, PARD announced that independent Data Monitoring Committee (DMC) recommended that the SPEAR (Study of Picoplatin Efficacy After Relapse) trial continue. The international, pivotal Phase 3 trial is evaluating the efficacy and safety of picoplatin in patients with small cell lung cancer (SCLC) who were refractory to or progressed within six months of first-line therapy. Based on patient enrollment data, the Company believes that overall survival data will be available from the SPEAR trial during 2H09.
Rockwell Medical Technologies (NASDAQ:RMTI): RMTI is developing a water soluble iron replacement therapy for dialysis patients called SFP for short (Soluble Ferric Pyrophosphate). On 4/29/09, RMTI announced the completion of patient enrollment in the Phase 2b study of SFP, which is a six-month, dose-ranging study in about 130 hemodialysis patients to determine the safety parameters and optimal SFP concentration to maintain normal levels of iron and hemoglobin. The Phase 2b trial should be completed by the end of September and data should be released about 60 days later, which should occur in late November or early December.
Genta (OTC:GNTA): On 5/12/09, Genta reported its quarterly operating results and provided an update on its lead experimental cancer drug Genasense (oblimersen sodium). AGENDA is a Phase 3, randomized, double-blind trial in 315 patients with advanced melanoma which is designed to confirm certain safety and efficacy results from a previous randomized trial of Genasense combined with dacarbazine in patients who have not previously received chemotherapy. AGENDA employs a biomarker to define those patients who derived maximum clinical benefit during the preceding study. These patients are characterized by low-normal levels of LDH, a tumor-derived enzyme that is readily detected in blood.
Key upcoming milestones for Genasense in the Phase 3 AGENDA trial include (1) a final safety and futility analysis by the independent Data Monitoring Committee is expected in May 2009 and (2) Progression-free survival (PFS) results are expected Q4 2009 if the trial proceeds to completion pending the safety and futility analysis in May. Genta included the following disclaimer regarding its funding and liquidity – Absent additional funding, the Company currently projects that it will run out of funds in June 2009. The terms of the 2009 Notes enable those note-holders, at their option, to purchase additional notes with similar terms. The Company does not have any additional financing in place.

Disclosure: No positions.