Trillium Therapeutics Inc. (TRIL) – Nasdaq Trillium Therapeutics (TRIL) presented at BloomBurton-2016 Healthcare Investor Conference in Toronto on May 3rd, the same as last year the speaker was TRIL’s Chief Scientific Officer (CSO) Dr. Bob Uger, he took the stand in Hall A (the largest room at the conference) that was full with investors, press […]
Eteplirsen: Reasonable Evidence of Efficacy
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 […]
Catatonic Economics
***”Economists have forecast 9 out of the last 5 recessions. *** One contribution I make to the CRG team is my background in economics. I have a pervasive interest in macroeconomics and am close to getting my Masters in Economics. I have worked for an economic indicator at the US Census Bureau, and work as […]
SRPT – YEAR 4 DATA ANALYSIS
YEAR 4 DATA ANALYSIS Review of Eteplirsen to treat DMD By: @boscaptn, et al. Compiled From SAREPTA Public Data For Week 216 (Year 4) of Study 201/202 for Exon 51 Disclaimer: NO FINANCIAL ADVICE – The Information in this report is provided for education and informational purposes only, without any express or […]
Dave-Trading – GILD
Gilead – GILD *** David Sobek is a disciplined value investor. Using models, research, and fundamental data, David devises a value for biotech stocks, buys when significantly undervalued, and sells when significantly overvalued.*** Dave LOVES writing about GILD. Their combined Hep C franchise is by all metrics the most successful launch ever, so it is […]
Biotech Inflection Point – Watch List-1
Following my last technical analysis “XBI – A Look At The Near and Long Term In The Biotech Sector” I will start adding names to our watch list with some beaten down tickers, we can find many deep value biotech stocks in the recent pullback, some are trading at or very close to their cash […]
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 […]
XBI – A Look At The Near and Long Term In The Biotech Sector
SPDR S&P Biotech ETF (XBI)–NYSE We will maintain our focus on the XBI as the biotech barometer, many are waiting for the XBI to outperform the IBB again and lead the way to a real recovery, to give us the sign that the technical correction is done and we are back to a […]
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 […]
SRPT Breakout- A Quick Look At The Chart
Sarepta Therapeutics, Inc. (SRPT) – Nasdaq After three years of a roller coaster, Sarepta getting closer to the the real deal, started with the NDA acceptance by the FDA on August 25th and a PDUFA date set on Feb 26th, 2016, but still waiting for the final date of the FDA Advisory Committee (AdCom) that […]
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 […]
BioBlast Pharma: an under the radar ultra-rare disease company
Bio Blast Pharma Ltd. (ORPN) – Nasdaq BioBlast Pharma (ORPN) is a clinical-stage biotech company, focused on treatments for rare and ultra-rare diseases, especially genetic and metabolic diseases. The company has three diversified platforms currently address five different diseases. The platforms include Cabaletta, which is a platform for diseases characterized by mutated proteins; BBrm platform is a […]
