The Adcom is over and Eteplirsen was voted down. A final decision still remains for approval. Much was discussed in the Adcom, the key arguments were over the following: 1. Adequacy of dystrophin creation. 2. Efficacy of 201/202 Trial vs Historical Controls Both are intertwined. Adequacy of dystrophin creation: FDA had little faith in […]
GWPH: Is Epidiolex Nothing But Placebo?
GW Pharmaceuticals made the mistake of allowing expectations to exceed its abilities, forcing the company to announce a delay in the read-out of its pivotal Dravet study. Data originally anticipated for release at the end of 2015 has been pushed back a full quarter, leaving investors on their toes. This Dravet trial is the first of 2 studies […]
Sarepta: Is Eteplirsen Dead?
This was originally going to be titled “Ahead of the AdCom” but that no longers seems fitting. The highly anticipated Advisory Committee meeting originally scheduled for Friday January 22 was canceled due to blizzard conditions. Extremely negative reviews in Briefing documents [link] from the FDA are leading many to expect a denial for Eteplirsen’s drug […]
BLCM: Limited Opportunities In a Changing Environment
The driving interest in Bellicum revolves around its safety switch for use in hematologic stem cell transplants. It’s product, BPX-501, is a T-cell therapy containing a caspase-9 gene that can be activated by the compound rimiducid, leading to cell apoptosis. BPX-501 is initially being positioned as an adjunct T-cell therapy for allogeneic HSCT. Stem […]
LOXO: Staying On TRK
Loxo Oncology presented highly anticipated results from its ongoing Phase 1 study of TRK inhibitor LOXO-101 yesterday November 8, in a latebreaker at AACR-NCI-EORTC; they did not disappoint. Disclosure of a response from just one patient with diagnosed TRK fusion mutation in a July publication of Cancer Discovery set much of this excitement in motion. […]
SRPT: Better The Devil You Know
The Bull/Bear tussle in Sarepta has been ongoing for years now. Long term investors have ridden a rollercoaster as excitement has inevitably been followed by disappointment. Its Exon 51 skipping drug eteplirsen is set for an Advisory Committee meeting January 22, 2016 with the PDUFA date tentatively set for February 26. A war of words […]
Global Blood Therapeutics: Targeting Sickle Cell Disease
Global Blood Therapeutics (GBT) is a recent graduate of Third Rock Ventures, a highly regarded firm with a list of innovative portfolio companies including Bluebird Bio, Foundation Medicine, Agios, and Sage Therapeutics. The company’s most advanced program is a compound in Phase 1 for the treatment of patients with Sickle Cell Disease. Sickle cell disease […]
Raptor Pharma: Of Cystinosis and Huntingtons
Raptor Pharmaceuticals is a small Novato, CA. company that markets Procysbi for the treatment of nephropathic cystinosis. It has active clinical trials in Huntington’s Disease and Leigh Syndrome. A recent acquisition has provided entry into the Cystic Fibrosis field. Procysbi- also known as RP103- took a different route to market from that of most newly […]
STARTRK-1: Lost In Space [RXDX]
This is a brief analysis of recent entrectinib data presented by Ignyta. Entrectinib is a multi-kinase inhibitor with potent activity against TrkA/B/C, ROS1, and ALK. It was licensed from Nerviano Medical Sciences in November 2013, whereupon Ignyta took over development of the compound. Entrectinib is currently being studied in two Phase 1 studies- one in […]
INCY: Predicting Success in Pancreatic Cancer
Incyte Pharmaceuticals’ lead drug Jakafi (Ruxolitinib) is approved for the treatment of the bone marrow disorders Myelofibrosis and Polycythemia Vera. It is expected to reach blockbuster sales despite these relatively niche indications. The company has ambitions to expand Jakafi into solid tumors with Pancreatic cancer leading the way. Top line data from a Phase 2 […]
Loxo Oncology: One to Watch
Loxo Oncology (LOXO) is a biotech company built around the idea that cancer is best treated with highly targeted drugs paired to companion diagnostics. CEO Josh Bilenker, a partner at Aisling Capital, founded the company in 2013. Research and Development is outsourced, allowing about a dozen employees to manage company operations. Loxo acquired essentially all […]
Trevena Scores In Pain Study
Trevena Inc. (TRVN) presented positive Phase 2b results Monday August 31 from its TRV130 pain study. The primary endpoint of superior pain reduction compared to placebo was met, as well as superiority to morphine in the secondary endpoint of lower rates of nausea, vomiting, and hypoventilation. The results were quite good, and together with the previous […]
ESPR Quick Note
Esperion shares have fallen over 8% today as The Medicines Co and Alnylam released early data on their RNAi candidate, ALN-PCSsc, targeting PCSK9. The drug was shown to efficiently and safely reduce LCL-C when given subcutaneously. It was shown to maintain LCL-C suppression 140 days after a single 1.5 ml injection. The long duration of […]
Quick Note on GWPH
Embargoed Data was released yesterday in an announcement from AAN regarding GW Pharmaceuticals’ Epidiolex study: https://www.aan.com/PressRoom/Home/PressRelease/1364 This paragraph summarizes the Key Takeaway: “For the 137 people who completed the 12-week study, the number of seizures decreased by an average of 54 percent from the beginning of the study to the end. Among the 23 people […]
CYNAF is OFF the Mark
Cynapsus is developing a sublingual film formulation of apomorphine for the treatment of acute “Off” episodes in patients with Parkinson’s Disease resulting from long term use of Levodopa. While Levodopa is considered the single best treatment for Parkinson’s, long term use of the medicine leads to motor fluctuations as its efficacy declines. Patients then begin […]
Quick Take on Marinus Pharmaceuticals
Marinus Pharmaceuticals is developing the allopregnanolone analog Ganaxolone as a treatment for epilepsy and other seizure disorders. Its most advanced program is a multinational P3 trial of patients with refractory focal onset seizures. The drug is also being tested in patients with Fragile X syndrome and in girls with epilepsy due to PCDH19 mutation. The […]
Pfenex: Biosimilar Enthusiasm Is High
San Diego’s Pfenex Inc. is riding the market’s enthusiasm for biosimilars, molecules that although not considered identical, can be automatically substituted for biologic drugs. Previously considered immune to generic competition, recent regulatory changes have cleared the path to entry for copycats of so-called large molecule compounds. A looming patent cliff in 2020 puts about $100 […]
Quick Note on GWPH
GWPH has been on one huge rollercoaster. After climbing back from $61 in mid-October to $80 in late November, it has given up 15% in just the last 3 trading days, falling back to $68 as the rest of biotech surges following their morning Q3 conference call. Perhaps investors had been expecting more detailed data […]
What to Look For at ASH 2014
The year is nearly over, though it feels like scientific conferences never end. December 6th marks the last big Cancer meeting of the year- then we turn the page to 2015. We’ve put together a list of over a dozen companies presenting at ASH, along with data we believe investors will be looking for: AGIO […]
[Free Content] TGTX in Pictures
TG Therapeutics has two programs, the CD20 targeting antibody TG-1101, and PI3K delta inhibitor TGR-1202. The focus of this post is on TGR-1202. Earlier coverage can be found here [link]. TG has been hesitant in giving out the structure of TGR-1202. It’s out now, so here it is below along with Idelalisib and IPI-145 […]
