GW Pharma’s Tuesday morning R&D meeting looked pretty good. Efficacy data were released for 58 patients with full 12-weeks of epidiolex treatment- 40 of whom have been treated for 16-weeks. The total safety database consisted of 151 patients who have received any drug. Data from this larger pool of 58 patients- double that from the […]
Sarepta at World Muscle Society: Follow-up
The only new data provided by Sarepta were various measures of lung function, including, including %predicted MIP, MEP, and FVC; all of which demonstrated stabilization through 144 weeks for the 12 boys in the treated population. Individual patient data were once again provided, this time at the 144 week time point. As the trend has […]
SRPT and SAGE: Quick Notes
Nice 36-month Natural history study (or “Pane Study”) in Duchenne published essentially confirming what’s pretty much already known- that boys age 7 and over decline more rapidly than those under 7. They had previously reported 24 month results. This study and the Eteplirsen study are largely comparable. Average age of the older boys subgroup are […]
GW Pharmaceuticals: Quick Update
Investors are waiting impatiently for GW Pharma’s December R&D day. The most sought-after information is updates on the Epidiolex Expanded Access Program in Dravet Syndrome. A previous analysis involved 27 patients; there are now 100 boys being treated as of the third quarter. Even if a significant number of patients are excluded as data immatures, […]
[Free Report] JNJ Aquires Alios: What This Means For Achillion
Johnson & Johnson announced today it will acquire privately held Alios BioPharma for $1.75 Billion in cash, gaining rights to the mid-stage treatment for infants with respiratory syncytial virus (RSV), AL-8176, and early stage nuc AL-335 for HCV. “We are excited that this acquisition will enable us to explore treatment options for a number of […]
ECYT Fails to Meet OS Endpoint; Hope Rests on Tubulysin
Endocyte presented much anticipated results from its Phase II TARGET trial of vintafolide in non-small cell lung cancer (NSCLC) at the European Society of Medical Oncology (ESMO) Congress. The study compared vintafolide to vintafolide plus docetaxel, to docetaxel alone as second line treatment in folate-receptor positive patients with NSCLC. As a reminder, Vintafolide is a […]
[Free Report] Gilead Files NDA in Japan for HCV, Plans US, EU Filing for HIV
Gilead announced today it has submitted a New Drug Application (NDA) with Japan’s Pharmaceutical and Medical Devices Agency (PMDA) for approval of a fixed dose combination of NS5A inhibitor ledipasvir and nucleotide analog polymerase inhibitor sofosbuvir for the treatment of patients with genotype 1 HCV. The NDA is supported by a Japanese Phase 3 study […]
[Free Report] Wellcome Grant to Fast-track Ebola Drug Trials
The Wellcome Trust announced it has awarded 3.2 million pounds ($5.25 million) in grant funding to a consortium led by Dr. Peter Horby in a bid to fast-track multiple clinical trials for experimental Ebola drugs in Africa. Included in this consortium are Mapp Pharmaceuticals, Sarepta, and Tekmira. Mapp and Tekmira made headlines recently when their […]
[Free Report] Tekmira Authorized to Treat Ebola; Amgen Seeks Approval for BiTE
Shares of Tekmira are up over 15% midday on news the FDA has authorized the company to provide the drug TKM-Ebola for treatment of subjects with confirmed or suspected Ebola virus infections under expanded access protocols. Tekmira has long been the subject of speculation over approval of its drug for the treatment of Ebola. The […]
[Free Report] Flexion Receives Clinical Hold; Merck KGaA Looks to Partner Immuno-Onc Drug
Flexion Therapeutics announced the FDA informed the company yesterday that a clinical hold has been placed on patient enrollment and dosing in an ongoing Phase IIb trial evaluating FX006 in patients with osteoarthritis (OA) of the knee. Flexion has not yet received written notice of the hold, but verbal communication from the FDA indicates it […]
[Free Report] Endo Makes Bid For Auxilium; Gilead Fails Pancreatic Cancer Trial
Endo International made an unsolicited proposal yesterday to acquire all the outstanding shares of Auxilium Pharmaceuticals for $28.10 per share in a mixed cash and stock transaction valuing the company at $2.2 billion. Endo intends on funding the deal with existing cash as well as debt financing. Endo expects the addition of Auxilium to be […]
[Free Report] SIGA Files for Bankruptcy; NeuroDerm Files for IPO
SIGA Technologies filed for Chapter 11 bankruptcy protection Tuesday as it seeks time to appeal a court order favoring PharmAthene. In an earlier ruling, the Delaware Court of Chancery found SIGA had failed to execute its licence agreement with PharmAthene relating to the smallpox drug Tecovirimar. SIGA believes the lump sum payment and damages it […]
[Free Content] Avanir Scores Positive Alzheimer’s Data; NPS Gets Nod From Panel
Avanir announced positive results from its Phase II trial evaluating the safety of AVP-923 in a 10-week study of 220 alzheimer’s patients. Treatment with AVP-923 resulted in a statistically significant reduction in agitation and caregiver burden. Complete data will be presented at the American Neurological Association’s (ANA) 2014 Meeting , October 12-14. The company plans […]
[Free Report] Merck KGaA Ends Cancer Vaccine Trials; RTRX Takes Heat
Following the failure of tecemotide (formerly known as Stimuvax) in a Phase I/II study in Japanese patients, Merck has decided to discontinue all clinical development of the drug, including the Phase III START2 and INSPIRE non-small cell lung cancer trials. Tecemotide is a MUC1 antibody-specific immunotherapy licensed from biotech company Oncothyreon. It had previously failed […]
GWPH: Full Throttle Ahead
GW Pharmaceuticals looks to be quite busy in the next few months as a number of trials are initiated. Rapid development along with promising initial results for epilepsy drug Epidiolex have sent shares soaring. In merely a year, the company has directed investor attention away from slow sales of its most advanced drug Sativex to […]
Quick Take on GERN Clinical Hold
Geron announced this morning the FDA had placed a clinical hold on its lead drug Imetelstat. While the agency cited only trials in ET, PV, and MM, the company also expects a delay in its key Phase II MF study- originally set to begin mid-year. Shares plunged from $4.40 to as low as $1.39, but […]
High Risk, High Reward With GW Therapeutics (GWPH)
In just a year’s time, GW has morphed from a company focused on the marketing of an approved product to one dedicated to the development of an early stage orphan drug candidate. A majority of the company’s valuation is now derived from this Phase I anti-epilepsy treatment. Crucial data from an ongoing Investigator Sponsored Trial […]
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 […]
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 […]
