Outlook: Bullish Catalyst: American Academy of Neurology Meeting, Mid-March Isis Pharmaceuticals recently received approval for Kynamro, a drug to treat Homozygous Familial Hypercholesterolemia (HoFH); but it is the development of an early stage treatment for Spinal Muscular Atrophy (SMA) that is currently generating the most interest. It is the leading inherited cause of infant mortality […]
INFI – Infinity Pharmaceuticals Still Going Strong
Sentiment: Bullish Catalyst: ASCO, Early June 2013 Following a nice presentation at ASH 2012, Infinity Pharmaceuticals impressed once again at the annual JP Morgan Healthcare conference earlier this month. The company is developing the dual PI3K delta/gamma inhibitor IPI-145 in multiple hematological malignancies and inflammatory diseases, and HSP90 inhibitor Retaspimycin for lung cancer. Results were […]
JPM13, Biotech Showcase: Management Meetings
Notes From The Conference ImmunoCellular Therapeutics (NYSE MKT:IMUC) Met with Peter Ho, former scientist and analyst, now Director of Business Development at Immunocellular Therapeutics. According to Ho, the reason for previous CEO Singh’s departure was a difference in vision for the company between him and the board; he was aiming for a sale while board […]
RTRX – Retrophin- From Hedgefund to Biotech
Introduction Started in 2010 as an “extracurricular activity” by hedge fund manager Martin Shkreli, Retrophin was officially formed in February 2011 to focus on developing drugs for orphan indications. The current pipeline consists of a single compound in Phase II along with three preclinical projects. It went public in December 2012 through a reverse merger […]
3 Phase III trials set to read out in 2013 – ONTY, CLSN, NLNK
With Patrick Crutcher Some of the biotech sector’s most highly anticipated catalysts approach in early 2013. Nearly every issues has been debated upon between the first two companies, while the third is relatively unknown amongst most investors. Admittedly, it is very difficult to come to a clear consensus on the outcome of each study. They […]
2012 ASH Meeting – Myelofibrosis, Lymphoma, Myeloma
It’s going to be a busy few days at ASH, which begins December 8 in Atlanta. A huge amount of data will be presented; here is an overview of some interesting abstracts. YMI: YM Biosciences Title: Phase I/II Study of CYT387, a JAK1/JAK2 Inhibitor for the Treatment of Myelofibrosis YM Biosciences will present final safety […]
Rheumatoid Arthritis Treatments: The Decade Ahead
Rheumatoid arthritis sufferers have seen dramatic improvements in the standard of care over the last 25 years. Prior to the approval of methotrexate (MTX) in 1988, the best that doctors had to offer were NSAIDs and analgesics to manage pain and inflammation. As the first approved disease modifying drug, MTX revolutionized RA treatment and is […]
INCY, LLY – Baricitinib at ACR 2012
Incyte Corp and Eli Lilly participated in an investor meeting following the presentation of Phase II data for their jointly developed JAK inhibitor, baricitinib, at the American College of Rheumatology (ACR) on November 13. Baracitinib is a selective and highly potent JAK1/2 inhibitor entering Phase III studies in Rheumatoid Arthritis (RA). The companies described highly […]
FOLD – Phase II Amigal Co-administration Update
Amicus Therapeutics released updated data Thursday for its chaperone molecule Amigal (migalastat) for Fabry disease at the American Society of Human Genetics (ASHG) Annual Meeting. Results looked good, continuing to build on positive data presented earlier this year. This study looks at the ability of Amigal to increase the activity of Enzyme Replacement Therapies (ERTs) […]
CPRX – Catalyst Pharmaceutical Partners – Promising But Still Early
Catalyst Pharmaceutical Partners Inc. (CPRX) -NasdaqCM Catalyst: Phase IIb trial results Sentiment : Bullish Tiny Catalyst Pharmaceutical Partners is expected to disclose top-line results from its pivotal Phase IIb study of CPP-109 for the alleviation of cocaine addiction in mid-November. CPP-109 is a very promising drug in a field otherwise littered with failure. Should it […]
SRPT – Sarepta Presents 48-Week Data At World Muscle Society Congress
Sarepta Presents 48-Week Data At World Muscle Society Congress Long-term safety and efficacy results were presented this Saturday at the WMS Congress in Persh, Australia. We had already learned from a press release of top-line results October 3rd that results were positive: 50mg/kg treated patients remained stable- perhaps improved on the 6MWT- and treatment delayed […]
AASLD 2012 – An HCV Primer
It seems EASL has just ended, but AASLD is fast approaching, coming mid November. For HepC watchers, here is an overview of some companies that will be presenting at the Liver Meeting. Despite the tremendous progress made to-date, new advances keep it exciting. Gilead: Acknowledged leader in the race to bring an all-oral drug combination […]
VRTX – Vertex: Kalydeco Is The Key
Catalyst: North American Cystic Fibrosis Conference, October 11 Outlook: Neutral Ten abstracts from Vertex have been accepted at the North American Cystic Fibrosis Conference (NACFC). Investors will be keenly focused on the October 11 presentation titled, “The Investigational CFTR Corrector, VX-809 (Lumacaftor) Co-Administered with the Oral Potentiator Ivacaftor Improved CFTR and Lung Function in F508DEL […]
INFI – Infinity Pharmaceuticals – Is It Overvalued
Catalyst: ASH 2012, December 8-11 Sentiment: Bullish The bulk of Infinity’s value lies in the early stage asset, dual PI3K delta/gamma inhibitor IPI-145. Based on near-term clinical results, Infinity may be deemed vastly overvalued, or the next must-own biotech stock. The company is also developing an HSP90 inhibitor retaspamycin, but we ascribe nominal value to […]
INCY – Incyte: Taking The Long View
The pain has not stopped for INCY shareholders since the company offered disappointing revenue guidance in its 2Q conference call August 2. A less than stellar projection of $120 to $135 million in 2012 Jakafi sales did not sit well with investors. This was compounded by the company’s decision to switch mid-year from a sell-through accounting method to […]
SRPT – Sarepta – Approaching Major Catalyst
Sarepta’s release of positive interim data from its DMD drug on July 24 has lit a fire under the stock- and stoked animated discussion in the twitterverse. We revisit this story as a critical catalyst approaches. Due to the severity of the disease and the young age of those afflicted, investors uniformly cheer for eteplirsen, Sarepta’s […]
SGYP – Synergy Pharmaceuticals, Expecting Positive Data
Synergy is a different kind of company- that is for certain. It is the epitome of a virtual biotech: it’s staff of nine is charged with running a pivotal 880 patient Phase II/III study; initiating follow-on projects; and in-licensing new compounds. The company’s lead candidate is plecanatide, a peptide analog of the human hormone uroguanylin. […]
HALO – Halozyme – The Platform Still Stands
Halozyme shares cratered following a Complete Response Letter (CRL) received by Pharma partner Baxter for the potential blockbuster drug HyQ on August 1. The FDA had concerns over high levels of non-neutralizing antibodies patients developed to recombinant human hyaluronidase, or rHuPH20, Halozyme’s platform technology. HyQ is a combination of PH20 with Baxter’s Gammagard (basically a […]
Bristol’s HepC Drug Fails – Now Idenix Compound Is On Clinical Hold
Bristol’s nuc, INX189, crashed not long after the compound was acquired through a $2.5 billion buyout of Inhibitex as a result of heart failure in one patient. Now the FDA has called for a partial clinical hold on a similar compound made by Idenix out of caution. IDIX stock has dropped about 30% today. Toxicity […]
SRPT – Sarepta Therapeutics Develops Promising Treatment For DMD
Duchenne’s Muscular Dystrophy (DMD) is a genetic disorder that afflicts approximately 1 out of every 3,600 male infants. The inherited disease is caused by mutations in the gene for dystrophin- a muscle protein- resulting in muscle weakness at an early age that quickly worsens over time. Breathing difficulties and heart disease typically begin in the […]
