By Patrick Crutcher
With the second quarter underway, we wanted to highlight some events in biotech that should be taking place in the coming months. Below is a list of seven companies with pending developments that investors in biotech should be aware of for the coming months. We expect that some of these catalysts will be a big boon for biotech investors.
Amarin Corp. (AMRN) expects top-line results from Phase 3 ANCHOR trial of AMR101 in patients with high triglyceride levels. At the recent 10th Annual Needham Healthcare Conference, Joseph Zakrzewski, Chairman and CEO of AMRN, reiterated that they expectd data in the early part of Q2 and reiterated no one at AMRN has seen the results(questions about insider selling). As of March 16th, they noted that they have completed the last patient visit for the Phase 3 ANCHOR trial. Management remarked that they are on-track to submit a New Drug Application submission for AMR101 in Q3-2011 using the existing MARINE trial data.
Recently, analysts at Jeffries gave some guidance about expectations for ANCHOR trial results that investors should keep in mind. Quoting the article, "Investors have been divided on expectations for both the primary endpoint of triglyceride lowering and the secondary endpoint of LDL neutrality, and concerns have been compounded by insider selling ahead of the data release. To meet these endpoints AMR-101 must show a 15% reduction in TG vs. placebo and a less than +6% difference in LDL at the upper bound of the 95% confidence interval."
Corcept Therapeutics Inc. (CORT) anticipates submitting a New Drug Application for the use of CORLUX in Cushing’s Syndrome to the FDA the week of April 11, 2011. They will be seeking a 6-month priority review for CORLUX. Corcept raised roughly $42 million in January, following very positive clinical data (primary and secondary endpoints) from their pivotal study of CORLUX in Cushing's Syndrome. Corcept plans on presenting detailed Phase 3 data at the Endocrine Society Annual Meeting, which takes place June 4-7th in Boston. Piper Jaffray notes that: "We continue to expect approval of Corlux in Cushing's syndrome, potentially a $500 million opportunity.” See our previous note on Phase 3 results and interview with Corcept.
There is an FDA Advisory Panel meeting on Thursday, April 28th from 8:00am - 12:00pm to discuss Vertex Pharmaceuticals (VRTX) hepatitis C virus (HCV) protease inhibitor, Telaprevir. This will be used to treat chronic HCV genotype 1 infections in combination with pegylated interferon-alfa and ribavirin in patients that are previously untreated or who have failed previous therapy. It cuts current treatment time down from 1 year to 6 months, roughly doubles standard of care cure rates and has done exceedingly well in those have failed previous treatment. An FDA decision date has been set for May 23rd, 2011 under the Prescription Drug User Fee Act. By in large, most are expecting Telaprevir to be a blockbuster drug, with potential peak annual sales of over $3 billion.
Optimer Pharmaceuticals (OPTR) recently received a positive FDA Advisory panel recommendation for DIFICID (fidaxomicin) for the treatment of Clostridium difficile infection (CDI). OPTR currently has a PDUFA date of May 30th(barring a Federal shutdown). Although the committee was split on the decision about the recurrence rate, management believes that by working with the FDA over labeling, there will be something in the label to reflect DIFICID's benefits for preventing relapse after 30 days. They just recently signed a 2-year co-promotion deal with Cubist Pharmaceuticals (CBST) to help launch fidaxomicin in the United States.
Onyx Pharmaceuticals Inc. (ONXX) still plans on submitting a New Drug Application for the use of carfilzomib in patients with relapsed multiple myeloma as early as mid-2011. Onyx has already initiated a rolling submission of a New Drug Application for potential accelerated approval. Onyx posted very compelling Phase 2b data back in December and was given Fast Track designation back in January. Onyx has currently been doing big business through revenues on sales of their liver and kidney cancer drug, Nexavar, which did global sales of $934 million for FY2010. This program is partnered with Bayer, whom they have 50-50 profit share and collaboration agreement with.
One potential caveat for Onyx: AVEO Pharmaceuticals (AVEO) is currently running a head-to-head pivotal Phase 3 trial (TIVO-1) comparing the efficacy and safety of tivozanib to sorafenib (Nexavar) in patients with clear cell kidney cancer who have undergone a prior nephrectomy. Their Phase 2 data were very compelling, but keep in mind the bar for significance is very high in a superiority study. The top-line results for progression-free survival are expected sometime in mid-2011. They recently signed a big partnership with Astella Pharma Inc. that netted them $125 million upfront with about $1.3 billion in potential milestone payments.