On Tuesday morning, Sarepta informed investors that in what appeared to be a change from its previous stance, the FDA was not supportive of the company’s planned Accelerated Approval filing for the DMD drug eteplirsen. Even more damaging, the agency not only called into question the legitimacy of dystrophin as a biomarker, but cast doubt […]
Quick take on Geron’s Imetelstat abstract
Today we finally got a glimpse at the ongoing investigator sponsored study of Geron (GERN) imetelstat in myelofibrosis. We have previously discussed this program here. The abstract can be found here. An oral presentation is scheduled for Monday, December 9th at 4:45 PM. The study was carried out by Dr. Tefferi at the Mayo Clinic. […]
ASH 2013 Preview on Myelofibrosis
This year’s annual meeting of the American Society of Hematology (ASH13) is highly anticipated for a number of reasons. With the general market for the healthcare/biotech sector at all-time highs on the backs of Pharmacyclic’s Ibrutinib (stock is up 1000% since ASH11), Celgene’s Revlimid (~67% of their $6.3B 2013 revenue) and Amgen’s $10.6B deal for […]
Ariad- Where Do They Go From Here
Following the FDA’s clinical hold, then discontinuation of the Phase III trial in front line CML for Ariad’s only marketed drug, Iclusig, the most pressing issue comes down to are finances. Ariad is a big spender, burning through $69 million in the second quarter of this year with $83 million in operating expenses. It will […]
GERN – Quick Take on Geron
Geron shares have run up substantially in recent days on speculation of positive results for its telomerase inhibitor imetelstat in an investigator sponsored study for the treatment of myelofibrosis. During the August 8th Q2 conference call, management noted the second 11 patient cohort had completed enrollment and met the pre-specified criteria of at least 2 […]
SRPT – Sarepta Quick Commentary
It Has Everything And Nothing To Do With Dystrophin Investors have been intensely focused on Sarepta CEO Chris Garabedian’s July 24 “Regulatory Update”, parsing each sentence as if it were one of Bernanke’s speeches. The call went generally as expected: following discussions with the FDA, Sarepta announced plans around an NDA filing based on Phase […]
BCRX – BioCryst – Entering The HAE Market
BioCryst announced on Monday July 22 the successful completion of a Phase I study on the safety and PK of BCX4161, a candidate for the treatment of Hereditary angioedema (HAE). HAE is a genetic disorder resulting from the loss or dysfunction of complement C1 Inhibitor (C1INH). Among the functions performed by C1INH is regulation of […]
STML – Follow up on Stemline
In what is becoming increasingly rare these last few months, Stemline made its public debut this January with a drug on track for late stage studies. The company focuses on cancer stem cells, with the clinical candidates SL-401 and SL-701. Today, we will delve into the former, a drug targeted to the IL-3 receptor. Read […]
INFI – Infinity Pharma, Staying The Course
This has been one heck of a roller-coaster ride. The stock took 8 months to triple in price, and just 2 to give it all back. Shares began to swoon prior to data presentations at ASCO and ICML. We had expected positive results to soothe the market, but this was not the case. Investors focused […]
SGYP – Cautiously Optimistic On Synergy Pharmaceuticals
We revisit Synergy Pharmaceuticals as the company prepares to initiate a Phase III Chronic Idiopathic Constipation (CIC) study of lead drug plecanatide later this year. Top-line results from the Phase II/III trial were released January 2 early this year, followed by complete data reported at the Digestive Disease Week convention in May. As we had […]
INFI – Infinity Pharmaceuticals: Down But Not Out
Less than two months ago on April 2, INFI hit a multi-year high of $50.26. Since then, the stock tumbled more than 50% in a relentless fall driven by high trading volume. Shares recently recovered some and now sit at about $25. Many have argued the stock was overpriced in early April when it carried […]
GILD – Gilead – Building An Oncology Powerhouse
Having solidified its position as the leading provider of HIV medicines, Gilead turned toward a strategy of diversification, first with pulmonary and cardiovascular disease, then HCV. The first two were mostly busts, but a strong focus on Hepatitis C has paid dividends for stakeholders. Now the company is building on its momentum to create an […]
ASCO2013 Take Three: PD1 antibodies will have significant presence
This is our third piece in a series of articles on developments expected at ASCO 2013[1,2]. Currently, we think the majority of the exciting presentations at ASCO 2013 will be from clinical candidates at big pharma. In particular, anti-programmed death-1 monoclonal antibodies(anti-PD-1) like Bristol Myers’ Nivolumab(BMS-936558), Merck’s Lambrolizumab (MK-3475), GSK’s AMP-224, Teva/CureTech’s CT-011 and Roche/Genentech’s […]
GERN – Geron: Time To Turn Out The Lights
Once a high-flying stem cell company, Menlo Park based Geron has fallen on hard times. Year-to-date, shares are off some 25% even as most biotech indices have soared over 20%. The company is in the midst of a restructuring, reducing its workforce from 64 to 44; this is down from 174 employees in November 2011 […]
POZN – Hurdles Lay Ahead For Pozen
We have been actively following Pozen for the last couple years and believe now is a good time to pick up coverage once again. The company submitted an NDA for PA32540/PA8140 only a month ago, a full year after announcing positive top-line results from the second of two Phase III trials. An indication will be […]
ACHN – Sell Achillion, Buy Idenix
A lot has happened since we last discussed HCV stocks back in October ahead of AASLD. One thing that remains unchanged is Gilead’s dominant lead in the development of next-generation HCV drugs. Beating expectations, Gilead is set to submit its regulatory filing of an all-oral combo consisting of sofosbuvir and GS-5885 by mid-2014 with potential […]
CRIS – Curis Pipeline May Propel Shares
Curis is best known for its hedgehog (Hh) pathway inhibitor Erivedge, which has been licensed to Genentech (Roche). Erivedge was approved for the treatment of inoperable basal cell carcinoma (BCC) in January 2012. The launch has been slow; sales reached only $30.6 million last year. Partnered early, Curis receives only 5% royalties, escalating to high […]
ASTX – Astex Is One To Watch
Astex Pharmaceuticals is a promising small cap biotech company with both a marketed drug and pipeline products waiting in the wings. The Dublin, CA based company generates royalty revenue from sales of hypomethylating agent Dacogen (decitabine) for the treatment of AML and MDS. In recent years, the growing revenue stream has helped offset research spending […]
SGMO – Sangamo Getting Ahead Of Itself
SGMO: Sangamo Getting Ahead Of Itself Sangamo BioSciences has received a great deal of attention in recent weeks with the unveiling of a broad orphan drug therapy initiative at JPMorgan. This has overshadowed what appears to be weak data in its leading HIV drug candidate SB-728-T. Background: SB-728-T is an autologous zinc finger nuclease (ZFN) […]
ARIA – Ariad: Overvalued
Investors have high expectations for Ariad’s recently approved drug Iclusig. The tyrosine kinase inhibitor performed well in a pivotal Phase II of resistant/intolerant CML and Ph+ ALL patients. The successful PACE trial allowed for accelerated approval in mid-December 2012, with the launch beginning a few weeks later. Briefly, there are currently five approved targeted therapies […]
