Tue, May 31, 9:10 AM
Tue, May 31, 6:01 AM
Fri, May 27, 4:24 PM
- The European Commission approves Strimvelis, an ex-vivo stem cell gene therapy for the treatment of an ultra-rare inherited disorder called Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID). Children born with the homozygous disease do not have a fully functioning immune system and are unable to fight off common infections. It affects ~15 European children each year.
- Strimvelis is only administered once and does not rely on a third party donor. The patient's own bone marrow cells are removed and a vector is used to insert a normal copy of the ADA gene into the cells, which are then reintroduced into the patient via IV infusion. In order to improve the engraftment of the gene-modified cells into the bone marrow, patients are pretreated with low-dose chemo.
- The data supporting the application was collected from 18 children with ADA-SCID. All participants in the pivotal study (n=12) who received Strimvelis were alive three years post treatment. All the children (n=18) who contributed to the data package are alive today (median follow up of ~seven years). The complete data analysis was recently published in the journal BLOOD.
- Strimvelis, developed by GlaxoSmithKline (GSK -0.8%) and Italian firms Fondazioine Telethon and Ospedale San Raffaele, is first corrective gene therapy for children to gain regulatory clearance anywhere in the world. It is the second gene therapy approved in the EU, following uniQure's (NASDAQ:QURE) Glybera (alipogene tiparvovec), approved in November 2012 for the treatment of lipoprotein lipase deficiency.
Mon, May 16, 12:21 PM
- Novo Nordisk (NVO +1.1%) submits its Biologics License Application (BLA) to the FDA seeking approval of its long-acting factor IX, nonacog beta pegol, for the treatment of patients with hemophilia B. The glycopegylated recombinant factor IX has a 5x longer half-life than standard factor IX products which enables once-weekly dosing.
- Related tickers: (BIIB +1.3%)(DMTX +8%)(QURE +2.9%)(SGMO -3.3%)(BAX +0.4%)
Fri, May 13, 7:36 AM
- The European Commission approves Biogen (NASDAQ:BIIB) and collaboration partner Swedish Orphan Biovitrum AB's (OTC:BIOVF) Alprolix (Coagulation Factor IX [Recombinant], Fc Fusion Protein) for the treatment of hemophilia B. It is administered every 7-10 days so it provides prolonged protection against bleeding episodes as well as on-demand treatment.
- The data supporting the marketing application was generated in two Phase 3 trials, B-LONG, in adults and adolescents, and Kids B-LONG, in children under age 12.
- Swedish Orphan has final development and commercialization rights in Europe, North Africa, Russia and most of the Middle East. Biogen has commercialization rights in North America and the rest of the world ex-Swedish Orphan territory.
- Alprolix in now approved in the EU, U.S., Canada, Japan, Australia, New Zealand and certain other countries.
- Previously: Biogen's hemophilia B med successful in Phase 3 trial in children (Feb. 27, 2015)
- Related tickers: (NYSE:NVO)(NASDAQ:DMTX)(NASDAQ:QURE)(NASDAQ:SGMO)(NYSE:BAX)
Wed, May 11, 12:03 PM
- The European Commission approves CSL Behring's Orphan Drug-tagged IDELVION [Coagulation Factor IX (Recombinant), Albumin Fusion Protein] for the treatment and prophylaxis of bleeding in patients of all ages with hemophilia B (factor IX deficiency). The labeling includes routine prophylaxis to prevent/reduce the frequency of bleeding episodes, on-demand control and perioperative management of bleeding. Market launch will commence in the next few months. Orphan Drug status means that IDELVION has a 10-year period of market exclusivity in the EU for the indication.
- IDELVION's principal value proposition is 14-day dosing. This is achieved via the maintaining of high levels of factor IX activity, above 5% over 14 days at 75 IU/kg, via the fusion with recombinant albumin.
- The FDA approved IDELVION in early March.
- Related tickers: (BIIB -0.8%)(NVO -0.1%)(DMTX -0.8%)(QURE -1.2%)(SGMO -0.2%)(BAX +1.9%)
Tue, May 10, 4:27 PM
- UniQure (NASDAQ:QURE) updates its loan agreement with Hercules Technology Growth Capital, increasing the total commitment to $40M, $20M which is currently outstanding. The facility isn't cheap, though. The interest rate is 8.25% per annum, but there is a backend fee of 4.85% and facility fee of 0.75%.
- The loan has an interest-only period of 18 months (from May 6, 2016) that can be extended to 24 months contingent on the company raising $30M in upfront corporate payments and/or proceeds from equity financing and to 36 months contingent on uniQure raising a cumulative $50M from equity financing.
- The loan facility matures on March 1, 2020.
Wed, May 4, 11:22 AM
- Some investors were expecting big things out of uniQure (QURE -11.4%) when the European Commission approved its gene therapy, Glybera (alipogene tiparvovec), in November 2012 for the treatment of the ultra-rare condition known as lipoprotein lipase deficiency. Patients with the inherited disorder lack an enzyme required to process fat. It affects only about 200 Europeans so the company priced the therapy at $1M per regimen, making it the most expensive drug in the world and an expected growth driver for the company.
- Since approval, however, Glybera has been commercially used in but one patient. Reimbursement has only been established in Germany and even there patient access is daunting. A recent example from a Berlin physician is informative. She had to complete a thesis-size application for regulators and personally call the CEO of an insurer to ask him to pay the $1M price tag.
- The marketing investment required to ramp Glybera was apparently too daunting for uniQure. It dropped plans for U.S. approval and sold the European rights to Italian drug maker Chiesi Farmaceutici, a small outfit with scant experience selling such a complex therapy.
- QURE has lost ~67% of its value since peaking at ~$36 in late September 2015.
- Previously: UniQure sets price record with Glybera (Nov. 26, 2014)
Mon, Apr. 4, 6:28 AM
Mon, Apr. 4, 5:51 AM
Mon, Mar. 7, 7:05 AM
- The FDA approves CSL Behring's IDELVION [Coagulation Factor IX (Recombinant), Albumin Fusion Protein] for the routine prophylaxis of hemophilia B in children and adults to prevent or reduce the frequency of bleeding episodes, on-demand control and prevention of bleeding episodes and the perioperative (around the time of surgery) management of bleeding.
- IDELVION's principal value proposition is 14-day dosing. This is achieved via the maintaining of high levels of factor activity, above 5% over 14 days at 75 IU/kg.
- IDELVION will be available later this month.
- Related tickers: (NASDAQ:BIIB)(NYSE:NVO)(NASDAQ:DMTX)(NASDAQ:QURE)(NASDAQ:SGMO)(NYSE:BAX)
Fri, Jan. 8, 10:15 AM
- Novo Nordisk (NVO -0.4%) submits its Marketing Authorization Application (MAA) to the European Medicines Agency seeking clearance for the use of nonacog beta pegol, a long-acting recombinant factor IX, for the treatment of hemophilia B. Compared to standard factor IX products, nanacog beta pegol has a 5x longer half-life which enables once-weekly administration.
- The company intends to file a BLA with the FDA no later than mid-year.
- Hemophilia B-related tickers: (DMTX -0.1%)(QURE +5.8%)(SGMO +1.2%)(BIIB +0.4%)(BXLT +0.5%)(PFE)
Thu, Jan. 7, 9:15 AM
- Gainers: INVT +35%. DVAX +27%. QURE +14%. HMY +9%.
- Losers: TLOG -75%. EPZM -20%. GALE -19%. FINL -17%. SUNE -14%. AKBA -11%. RLYP -8%. FRO -8%. OTIC -8%. CIG -7%. SYN -7%. ATNM -7%. JKS -7%. KBH -7%. SAGE -7%. CEMP -7%. DNR -6%. ATML -6%. WLL -6%. SDRL -6%. ADMS -6%. BHP -6%. BBL -5%. AEZS -5%. VALE -5%. GGB -5%.
Thu, Jan. 7, 7:52 AM
- Preliminary results from the low-dose cohort in a Phase 1/2 clinical trial assessing uniQure's (NASDAQ:QURE) AAV5/FIX gene therapy, AMT-060, in patients with moderately severe to severe hemophilia B showed a significant clinical benefit in two participants who had completed at least 12 weeks of follow up and an initial treatment benefit in two more who had not completed the full follow up period (out of three).
- All five subjects had documented Factor IX (FIX) levels less than 1 - 2% and required chronic prophylactic treatment with recombinant FIX (rFIX) at the time of enrollment (results from an earlier study performed at St. Jude Children's Hospital showed that a durable FIX expression of ~5% of normal produced long-term clinical benefits). AMT-060 was administered through a peripheral vein in one treatment session lasting ~30 minutes. There are two treatment arms: low dose and high dose. To date, there have been no patient screening failures due to pre-existing neutralizing antibodies against AAV5 and no patients have developed inhibitory FIX antibodies.
- The two participants who completed at least 12 weeks of follow up showed FIX expression levels of 5.5% and 4.5%, respectively. Three additional subjects had been dosed but had not achieved the full 12 weeks of follow up. Two of the three, though, met a secondary endpoint of discontinuing prophylactic rFIX based on their FIX expression levels.
- The company intends to present a more complete analysis of the data at a scientific conference in Q2. Assuming Data Monitoring Committee approval, enrollment in the high dose cohort will commence this quarter.
- Management will host a conference call this morning at 8:30 am ET to discuss the data.
Wed, Jan. 6, 5:23 PM
- UniQure (NASDAQ:QURE) will announce top-line results from its Phase 1/2 gene therapy trial in hemophilia B tomorrow morning at 6 am ET. It will host a conference call at 8:30 am ET to discuss the data.
- The company needs something to goose its stock price. Shares are down almost 60% since September when it reported results from a Phase 1/2 study of AMT-110 in patients with Sanfilippo B syndrome.
- Previously: Four Sanfilippo B patients respond well to treatment with uniQure's AMT-110 gene therapy; shares up 18% premarket (Sept. 21, 2015)
Dec. 18, 2015, 9:15 AM
uniQure NV engages in the discovery, development, and commercialization of gene therapies. Its core gene therapies include AMT-060 for the treatment of hemophilia B; preclinical S100A1 therapeutic for the treatment of congestive heart failure; and Glybera a gene therapy that is designed to... More
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