As Cytokinetics confirmed in its Q4 2013 conference call, it will report top-line data from the BENEFIT-ALS study in the second quarter of this year, precisely, at the American Academy of Neurology annual meeting in Philadelphia April 26-May 3. If the company can’t have the whole data ready for that meeting, they’ll present a […]
Prospects for Prana's PBT2
On the wildly speculative front, Prana Biotechnology (PRAN) is a developer of metal protein attenuating compounds(MPAC) for neurodegenerative disorders. Shares are up over 350% in the past year as the company has neared readouts from 2 Phase 2 studies of their lead product, PBT2. The first is a small, 12-month study in 40 patients with […]
Upcoming Phase 3 trials
With a fresh start to 2014 and the biotech market still very strong, we wanted to examine some upcoming Phase 3 results that will be in focus in the first quarter. Expectations for some of these trials varies widely. We have a blend of small biotech to big pharma. Company: Merck (MRK) and Amgen (AMGN) […]
GERN- A Speculative Trade
To be clear, I’m not a big fan of Geron. However, upcoming events provide an interesting, although somewhat risky trading opportunity. The telomerase inhibitor imetelstat has demonstrated activity in the myeloproliferative disorders essential thrombosis and myelofibrosis. Geron is now planning a company sponsored P2 in MF set to begin around June; data may become available […]
A look at Innate Pharma
Immuno-oncology was one of the hottest themes of 2013 and its been almost entirely a big pharma story. We saw Bristol-Myers Squibb, Merck and Roche reveal more data and future plans for their PD-1 and PD-L1 inhibitors. It’s going to be the hottest theme for awhile, so we want to highlight one company that has […]
GenVec: hearing a comeback
On the verge of shutting down just a few months ago, Genvec (NASDAQ: GNVC) has recently had a comeback that might last for a few more years if its partnered hearing loss program yields good results in the clinic. As some background, in early 2010, Genvec was running higher in hopes that its gene therapy […]
Quick Take On NBIX
I have not followed this story before today, but I’ll throw my hat into the ring anyways It looks like Phase 2b results turned out positive in this second study due to some key differences. The first being a dose titration, lower average AIM score, and central raters. I think this bodes well for eventual […]
Analysis of TGR-1202, a PI3K Delta Inhibitor From TGTX
This is a quick take on the TGTX efficacy data presented at ASH this past weekend. TG Therapeutics reported PK data as well as results from the higher 800 mg cohort. It appears this step up from 400 mg has finally resulted in some activity and the company continues to dose escalate; an 1800 mg […]
SRPT – FDA Says No To Sarepta AA
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 […]
TSRO – A look at Tesaro
Following additional information released at ASCO 2013 and their recent quarterly updated, we wanted to take a moment to talk about Tesaro (TSRO). Since their IPO, Tesaro’s stock is up roughly 186% at a fully diluted marketcap of $1.22 billion (@ $35 per share) based on roughly 35 million shares outstanding. Tesaro’s pipeline consists of […]
ASH 2013 Preview on ALL/CML/MDS/AML
This year’s annual meeting of the American Society of Hematology (ASH13) is highly anticipated for a number of reasons. The first series in our preview covered what to expect in multiple myeloma. Today’s preview will focus on some of the moves going in acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), chronic myeloid leukemia (CML), […]
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 […]
ASH 2013 Preview on Lymphoid Malignancies
This year’s annual meeting of the American Society of Hematology(ASH13) is highly anticipated for a number of reasons. Pharmacyclic’s Ibrutinib could be viewed as one of the several catalysts sparking the biotech sector’s run the past 2 years. They are now within months of receiving approval for 2 indications where they could benefit patient lives […]
ASH 2013 Preview on rare blood disorders
This year’s annual meeting of the American Society of Hematology (ASH13) is highly anticipated for a number of reasons. Orphan diseases has been a key element in the bull market in biotech over the past 2-3 years. Consider that the market for hemophilia A treatments in 2011 was over $5 billion alone. Today’s preview will […]
ASH 2013 Preview on Multiple Myeloma
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 […]
CLDX – A look at CDX-1127
Celldex Therapeutics (CLDX) has one of the most anticipated data releases amongst small biotech companies this fall. They are scheduled to present data from their Phase 1 dose-escalation trial of a CDX-1127 (CD27, a T-cell stimulator) at the annual meeting of the Society for ImmunoTherapy of Cancer(SITC), November 7-10th. We will also likely see some […]
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 […]
Datasets in focus at IASLC this weekend
This weekend is the 15th annual World Conference on Lung Cancer, sponsored by tge International Assoc. for The Study Of Lung Cancer (IASLC). It takes place Sunday October 27th through Wednesday 30th (Site). Abstracts can be found here. In particular, we want to speak briefly about 7 presentations. PD1 inhibitors Bristol-Myers (BMY), Merck (MRK) and Roche (RHHBY) […]
Biocryst up on hope(hype) for HAE prophylaxis
In a previous post, Jason Chew provided some background and analysis on Biocryst (BCRX) BCX4161, an oral drug candidate for the treatment of hereditary angioedema (HAE). Biocryst’s stock is up 300%+ since July of this year on the back’s of Phase 1 trial of BCX4161 in healthy volunteers. With that said, we do question some […]
MDVN – Confusion over PREVAIL data explained in a single graph
On Tuesday, the market was rather confused on what to make of the PREVAIL news. To be clear, this wasn’t entirely the market’s fault: Medivation released a poorly worded press release that did not convey the whole story. A little extra detail would have gone a long way. From the press release, we know that […]
