Sunday, October 19, 2014

Amicus Therapeutics (FOLD) Announces Additional Positive Phase 3 Data Late Sunday Night

Amicus (FOLD) announced additional positive phase 3 results from its Phase 3 trial in Fabry disease using its lead compound Migalastat late Sunday night. I've provided an insert of the release below and expect shares to move up Monday morning due to the additional positive data. Across All Subgroups, Patients Treated with Migalastat Compare Favorably to Natural History on Kidney Function (eGFR) Additional GL-3 Data Further Validate Assay for Identifying Patients with Amenable Mutations CRANBURY, N.J., and SAN DIEGO, Oct. 19, 2014 (GLOBE NEWSWIRE) -- Amicus Therapeutics (FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced additional positive data from a Phase 3 study (Study 011) of the oral small molecule chaperone migalastat HCl ("migalastat") in Fabry disease patients with amenable mutations. In a poster at the American Society of Human Genetics (ASHG) Annual Meeting, Daniel G. Bichet, M.D., M.Sc., Professor, Department of Physiology, University of Montreal, presented results from patients in Study 011 including those who continued on migalastat in an open-label extension (Study 041). Assessment of kidney function by various measures of glomerular filtration rate (GFR) for patients receiving migalastat in Study 011 for at least 18 months and continuing migalastat treatment in Study 041 showed continued stability of kidney function for an average of 32 months. Decline in kidney function is a key cause of morbidity and mortality in patients with Fabry disease. Measured (iohexol) GFR (mGFR) showed stability over 18-24 months in Study 011 but was not collected in Study 041; mGFR was previously reported with topline Study 011 results. Mean Annualized Change in GFR (ml/min/m2/yr) (SEM) Over an Average of 32 Months with Migalastat in 011 and 041 Estimated GFR (eGFR) (CKD-EPI) (n=41) -0.20 (0.60) eGFR (MDRD) (n=41) +0.63 (0.08) Stratifying patients for gender and baseline proteinuria demonstrated that patients treated with migalastat experienced less decline in kidney function than untreated patients from a previously published natural history study1. Dr. Daniel Bichet, Full Professor and Section Head, Renal Function & Transport Physiology, University of Montreal, said, "Baseline proteinuria levels are among the most predictive indicators of disease prognosis and kidney function decline in Fabry patients. The data presented today show that when comparing patients with similar levels of proteinuria, patients treated with migalastat are more stable in their kidney function versus untreated patients. These results are very encouraging for migalastat as a treatment for Fabry patients with amenable mutations." Data from a subgroup analysis comparing the change in GL-3 substrate levels between amenable patients and non-amenable patients based on the GLP HEK cell assay provided additional validation of the sensitivity of the GLP HEK assay for identifying patients who will respond to migalastat monotherapy. Overall, patients with amenable mutations had declining levels of GL-3 when treated for six months with migalastat. In contrast, patients with non-amenable mutations had no change or increasing levels of GL-3 after six months of migalastat treatment. "We are pleased to present these additional Phase 3 results in a scientific forum. With 32 months of data, Fabry patients treated with migalastat exhibit long-term stability in their kidney function. These results contrast with the decline in kidney function reported in natural history studies. Decline in kidney function is one of the primary causes of morbidity and mortality in Fabry patients," stated Dr. Jay A. Barth, Chief Medical Officer of Amicus Therapeutics, Inc. "The additional data on substrate reduction show that we can accurately identify patients who may benefit from migalastat. We look forward to meeting with regulatory agencies starting this quarter as we work to make migalastat available for all amenable Fabry patients as quickly as possible."

Thursday, October 9, 2014

Targacept (TRGT) provides update on partnership

Targacept (TRGT) filed an 8K today which stated that their partnership with AstraZeneca was terminated including their Alzheimer's compound AZD1446. This is pretty shitty news for the company as the only glimmer of hope after their recent two phase 2 drug failures was this partnership. The company still has a ton of cash but no real value in the pipeline so expect them to waste the money on an in-license opportunity (Teva just announced they are shedding around 14 development programs under their recently announced restructuring so that could be an opportunity). I currently own 10,000 shares of Targacept which isn't good with this announcement.

 I really like this book: The Biotech Investor's Bible

Tuesday, October 7, 2014

Trading Opportunity - Oxigene (OXGN)

Oxigene announced that will present Phase 2 data for its lead compound Fosbretabulin to treat Ovarian Cancer on November 9th at the International Gynecologic Cancer Society meeting. The company also noted that "additional data are expected to be included in the conference presentation" which tells me that overall survival may be announced at the meeting. The company already reported PFS improvement of 31.5% from the trial but a statistical improvement of overall survival would be a major catalyst for the company as any pivotal trial would likely require overall survival as the primary endpoint. I've provided a copy of the abstract for the meeting below which includes an overall conclusion from the sponsors. Abstract: RANDOMIZED PHASE 2 EVALUATION OF BEVACIZUMAB VERSUS BEVACIZUMAB/FOSBRETABULIN IN RECURRENT OVARIAN, TUBAL OR PERITONEAL CARCINOMA: A GYNECOLOGIC ONCOLOGY GROUP STUDY Aims The Vascular disrupting agent (VDA) fosbretabulin tromethamine selectively targets pre-existing tumor vasculature causing vascular shutdown leading to cell death and necrosis. Anti-angiogenesis agents like bevacizumab, a humanized anti-VEGF monoclonal antibody, might prevent revascularization after/during VDA treatment. Methods Patients with recurrent/persistent epithelial ovarian, tubal, or peritoneal carcinoma; measurable or detectable disease; and < 3 prior regimens were randomized to bevacizumab (15 mg/kg IV q 3weeks) or bevacizumab (15 mg/kg) + fosbretabulin (60 mg/m2) IV every 3 weeks until disease progression or toxicity. Randomization was stratified by disease status (measurable vs. non-measurable), prior bevacizumab, and platinum-free interval. The primary endpoint was progression-free survival (PFS). The study was designed with 80% power at a 10% level of significance to detect a hazard ratio (HR) reduction of 37.5%. Results The study enrolled 107 patients. Median PFS was 4.8 and 7.3 months for bevacizumab and bevacizumab+ fosbretabulin, respectively (HR = 0.685; 90% 2-sided CI=0.47 ~1.00). The proportion responding to bevacizumab was 28.2% (90% CI 16.7 ~ 42.3%) among 39 patients with measurable disease and 35.7% (90% CI 23.5 ~ 49.5%) among 42 patients treated with the combination. Adverse events (> grade 2) were more common in the combination particularly hypertension (35% versus 16%). There was one grade 3 thromboembolic event with the combination. One intestinal perforation in the bevacizumab arm was observed. Conclusion Based on the PFS and tolerability of these two anti-vascular therapies, further evaluation is warranted for this chemotherapy-free regimen. Fosbretabulin in combination with bevacizumab may double the risk of hypertension.

Thursday, October 2, 2014

Lilly (LLY) gives up on Lupus Drug providing less competition for Immunomedics (IMMU) and Anthera (ANTH)

Lilly (LLY) terminated the development of Tabalumab which was being studied for potential treatment of systemic lupus erythematosis (SLE) after the drug did not show efficiency in two phase 3 trials.

Immunomedics (IMMU) and Anthera (ANTH) have drugs in phase 3 trials focused on SLE so this is good news on one hand as it's less competition but bad news on the other as it highlights how difficult this disease is to treat and the prospects of successful trial results of their compounds.  Immunomedics is expected to report top-line results of its compound in Q1 2015.

I own shares of both companies as I'm willing to make the gamble as the upside is significant for both companies.

Wednesday, October 1, 2014

Anthera Pharmaceuticals (ANTH) provides update on partner discussions for lead compound

Anthera (ANTH) announced after the bell that they are in partnership discussions for its lead compound Blisibimod.  Blisibimod is a potential treatment for Lupus and IgA nephropathy and the company said the discussions were specifically related to partnerships for the compound outside of the United States.  This is a very positive development as a partnership with a reputable partner would show that due diligence was done on both the prospects of the compound and the credibility of the leadership team and company.  Anthera has a mere market cap of $40 million with a Phase 3 drug to treat an indication that desperately needs improved options - Lupus.  This tells me one thing - no one believes in the compound or the company.  Firming up a solid partner would help alleviate these concerns and could send the stock much higher.

I currently own 6,000 shares of Anthera but probably won't add to his position until this story plays out.