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.
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Sep. 21, 2015, 7:45 AM
- Top-line results from a Phase 1/2 clinical trial in four patients with Sanfilippo B syndrome show that a single administration of uniQure's (NASDAQ:QURE) novel gene therapy, AMT-110, had a positive effect on restoring the activity of the defective protein that causes the condition. Three months after each patient received one two-hour infusion of AMT-110 directly into the brain, the catalytical activity of the NaGLU protein increased in the cerebrospinal fluid from 0% to 14-17%. All experienced a persistent effect at 12 months as defined by incremental cognitive development. The data were presented at the European Society of Gene and Cell Therapy and Finnish Society of Gene Therapy Collaborative Congress in Helsinki, Finland.
- The results validate the effective transmission of the NAGLU gene with the AAV5 viral vector and validate the company's insect cell-based manufacturing platform. The company intends to advance the program to the pivotal stage of development (data will support a regulatory filing).
- Sanfilippo B is a rare inherited lysosomal storage disorder that results in serious brain degeneration in children. The generally fatal disease is caused by a defect in the alpha-N-acetylglucosaminidase (NAGLU) gene which leads to the accumulation of partially degraded heparin sulfate. Children with the condition usually live only ~15 years. There are no commercially available treatments for the disorder.
- Shares are up 18% premarket on increased volume.
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Apr. 6, 2015, 8:12 AM
- Bristol-Myers Squibb (NYSE:BMY) and uniQure N.V. (NASDAQ:QURE) ink an agreement that provides BMY with exclusive access to uniQure's gene therapy technology platform for multiple targets in cardiovascular diseases. The collaboration includes the latter's proprietary gene therapy program for congestive heart failure that is designed to restore the heart's ability to synthesize S100A1, a calcium sensor and master regulator of heart function. The companies may collaborate on as many as 10 targets.
- Bristol-Myers will lead development and regulatory activities across all programs, including paying all R&D costs, and will be responsible for the commercialization of all products while uniQure will lead discovery efforts and will be responsible for manufacturing clinical and commercial supplies using its vector technologies and insect-cell-based manufacturing platform.
- Under the terms of the agreement, BMY will make near-term payments of ~$100M, including an upfront fee of $50M, $15M for the selection of three collaboration targets in addition to S100A1, and a 4.9% equity investment in uniQure at $33.84 per share (~$32M). BMY will acquire an additional 5% of uniQure before the end of 2015 at a 10% premium and will be granted two warrants to acquire up to another 10% of uniQure based on the addition of additional collaboration targets. uniQure will be eligible to receive research, development and regulatory milestones of up to $254M for S100A1 and up to $217M for each other gene therapy product candidate plus sales-based milestones and single-to double-digit royalties. A separate supply contract covers manufacturing by uniQure.
- The partnership will become effective in Q2 contingent on customary closing conditions including clearance under the Hart-Scott-Rodino Antitrust Improvements Act.
- QURE is up 42% premarket on average volume.
Jan. 14, 2015, 12:43 PM
Jan. 5, 2015, 12:48 PM
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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