Today - Thursday, November 27, 2014
- Britain's National Institute for Health and Care Excellence (NICE), the advisory group for the National Health Service, recommends the commission of Alexion Pharmaceuticals' (NASDAQ:ALXN) Soliris (eculizumab) for the treatment of patients with atypical hemolytic uremic syndrome (aHUS), an ultra-rare life-threatening disorder characterized by renal failure and the formation of blood clots in the small blood vessels throughout the body (thrombotic microangiopathy or TMA). In the UK, the total number of potential patients, typically children five years old or less, is only ~25.
- 79% of patients with aHUS die within three years of diagnosis despite plasma exchange or plasma infusion. The majority of patients with aHUS who undergo a kidney transplant experience subsequent systemic TMA and face a 90% transplant failure rate.
- Soliris first achieved regulatory clearance in 2007 in the U.S. and EU for the treatment of another ultra-rare life-threatening blood disorder called paroxysmal nocturnal hemoglobinuria (PNH). It was approved for aHUS in the U.S. and EU in 2011. Soliris is also cleared for sale for both indications in Japan and selected other countries.
- A full regimen of Soliris costs ~$540,000, the world's most expensive drug until UniQure's Glybera (alipogene tiparvovec) leapfrogged it.
- NICE recommended funding for Soliris for aHUS in September.
- Previously: NICE recommends Soliris for aHUS
- Previously: UniQure sets price record with Glybera
- An experimental Ebola vaccine from GlaxoSmithKline (NYSE:GSK) produced an immune response in all 20 healthy volunteers who received it, and did not cause any serious side effects, Reuters reports quoting the New England Journal of Medicine.
- The early-stage clinical trial, which began on Sept. 2, is primarily aimed at assessing how safe the vaccine is and will monitor the volunteers for 48 weeks.
Wednesday, November 26, 2014
6:38 PM| 1 Comment
4:45 PM| Comment!
- Affymax (OTCPK:AFFY) declares a special cash distribution of $0.05 per share to shareholders of record December 2, 2014. Also, The BOD appoints Jonathan M. Couchman as a Class I director and President & CEO of the firm. The remaining members of the board resign.
- The company will continue its evaluation of strategic alternatives.
- Previously: The end for Affymax
4:07 PM| Comment!
- The Federal Trade Commission approves the merger between Medtronic (MDT +1%) and Covidien plc (COV +1.2%) with the proviso that Covidien divest its drug-coated catheter business before completing the transaction. Covidien agreed to sell the business for $30M to Spectranetics (SPNC +1%) several weeks ago.
- Previously: Medtronic buying Covidien for $42.9B
- The Federal Trade Commission approves GlaxoSmithKline's (GSK +0.4%) proposed acquisition of Novartis' (NVS +0.6%) vaccines business and the proposed creation of a consumer healthcare joint venture between the two firms.
- As a condition of the positive vote, Novartis will divest its private label nicotine replacement therapy transdermal patch, Habitrol, in the U.S. to Dr. Reddy's Laboratories SA (RDY +1.5%).
- Dutch biotech UniQure (QURE +22.6%) catapults into uncharted territory with the pricing of its gene therapy drug Glybera (alipogene tiparvovec). It is set to debut in Germany at €1.1M ($1.4M).
- Glybera treats an ultra-rare disease called lipoprotein lipase deficiency (LPLD), a condition that clogs the blood with fat. The European Commission approved it in November 2012 but the launch was delayed because the company needed to collect six-year follow up data on its benefits.
- UniQure's commercial partner, Italian firm Chiesi, has filed a pricing dossier with German regulators, who will issue an assessment of the drug's benefits by April 2015. Chiesi seeks a retail price of €53,000 per vial. Under German rules, the launch price will be valid for 12 months.
- At this price, the full regimen cost will be €1.1M for a typical LPLD patient weighing 62.5 kg, who will require 42 injections from 21 vials. The number of eligible patients is tiny, only 150 - 200 across Europe.
- Former #1 Soliris (eculizumab), made by Alexion Pharmaceuticals (ALXN +0.7%) is now a distant second at ~$540,000.
- ETFs: IBB, BIB, BIS
- The FDA issues a Complete Response Letter (CRL) to Bristol-Myers Squibb (BMY +0.2%) in response to its NDA for HCV drug daclatasvir in combination with other agents.
- The company's initial daclatasvir NDA focused on its use in combination with asunaprevir. Considering BMY's withdrawal of asunaprevir in October, the FDA requests additional data for daclatasvir in combination with other antiviral agents for the treatment of HCV infection. BMY is in discussions with the agency about the scope of these data.
- Previously: Bristol-Myers pulls hep C NDA
- Previously: Bristol-Myers presents data on HCV Trio regimen
- Guided Therapeutics (OTCQB:GTHP -4.3%) prices its offering of 16.8M shares of common stock and warrants to purchase 0.5 shares of common stock at $0.225 per full share. Both are offered at a combined price of $0.225. Definitive agreements with purchasers are already in place.
- Net proceeds will fund the FDA approval activities for LuViva, to repay certain bridge loans, working capital and operations.
- Nebraska Medical Center has received four Hemopurifiers from Aethlon Medical (OTCQB:AEMD +17.4%) to stockpile for future Ebola treatments. The company has also completed onsite training for clinical personnel related to the implementation of Hemopurifier therapy within the dialysis infrastructure currently located at the center.
- Previously: Aethlon Medical up on successful Ebola treatment
- The Drugs for Neglected Diseases initiative ((DNDi))and Medecins Sans Frontieres take exception to the $125M that Gilead Sciences (NASDAQ:GILD) paid to Knight Therapeutics (OTC:KHTRF) for its priority review voucher (PRV) that it received from the FDA in connection to its approval of Impavido (miltefosine) for leishmaniasis. The humanitarian groups cry foul over Knight's windfall because the R&D for miltefosine was largely conducted by a division of the World Health Organization (WHO/TDR - Special Programme for Research and Training in Tropical Diseases) in the mid-1990s using private and public funding. Since then, DNDi and partners have invested money in clinical studies of the drug.
- The groups are asking Knight and commercialization partner Endo Health Solutions to disclose the drug's manufacturing cost and to price it accordingly with minimal profit margin in order to make it affordable for the countries that need it. They argue that "the PRV mechanism, which aims to stimulate or at least reward drug development for neglected diseases, currently contains no access provisions and fails to ensure that only entities that invested in R&D are awarded the vouchers."
8:44 AM| 4 Comments
- In a letter to shareholders, Pershing Square's Bill Ackman says recent developments at Herbalife (NYSE:HLF) reinforce the fund's short thesis.
- Herbalife missed Q3 earnings consensus, reduced guidance and took an impairment charge on the currency exchange rate assumption pertaining to its business in Venezuela.
- Previously: Herbalife chief boosts stake in company
- Harvard Apparatus Regenerative (NASDAQ:HART) will no longer be providing its HART-Trachea product for future transplant procedures performed at Krasnodar, Russia as part of the ongoing airway transplant studies there which began in 2012. The company provides no reasons for its action.
- It will continue its preclinical work in the U.S. and EU to prepare for the initiation of clinical trials. The HART-Trachea is designed to replace or repair a severely damaged trachea due to physical trauma or cancer. Its scaffold technology has been in six surgeries to date, provided under compassionate use exemptions.
- Britain's National Institute for Health and Care Excellence (NICE) issues final guidance for Biotie Therapies' (OTCPK:BIOZF) Selincro (nalmefene) for reducing alcohol consumption for people with alcohol dependence. The National Health Service has three months to implement the recommendation and establish funding.
- Up to 600,000 people in England and Wales may be eligible for treatment.
- Selincro, a dual-acting opioid modulator, is the first therapy approved in Europe for reducing alcohol consumption in alcohol dependent individuals. Biotie licensed the global rights to the product to Lundbeck A/S. It received Marketing Authorization in February 2013 and has launched Selincro in over 20 European markets.
- Under the terms of the license agreement, Biotie is eligible to receive up to €89M (upfront fee, milestones and sales-based royalties). To date, it has received €22M in milestones from Lundbeck.
7:24 AM| Comment!