Ultragenyx announced topline “positive” safety and efficacy data from the second dose cohort and positive longer-term data from the first dose cohort of the ongoing Phase 1/2 study of DTX301, an investigational adeno-associated virus, or AAV, gene therapy for the treatment of ornithine transcarbamylase, or OTC deficiency. The 52-week study is designed to enroll patients with late-onset disease who are clinically stable and on a stable dose of alternate pathway medication. All three patients in the second dose cohort received a single dose. As of the September 12, data cutoff date, patient 1 in Cohort 2 had been followed for 24 weeks, patient 2 for 21 weeks, and patient 3 for 18 weeks. The first patient in Cohort 2 demonstrated normalization of ureagenesis to 104% at week 24. The patient had an initial peak effect at Week 6 and then a decline at Week 12 that was associated with initiation of a tapering course of steroids to manage a mild asymptomatic elevation in alanine aminotransferase, or ALT, levels. After discontinuation of steroids, the rate of ureagenesis normalized at Week 20 and remained normal at Week 24, at which time a second course of steroids had been initiated due to mild asymptomatic ALT elevations, which were controlled. The second and third patients in Cohort 2 did not show a clinically meaningful change in rate of ureagenesis at 12 weeks and did not have ALT elevations nor require steroid treatment. The Data Monitoring Committee, or DMC, has completed its review of Week 12 data from Cohort 2 and recommended that Ultragenyx proceed to the third dose cohort of the study. In Cohort 3, three patients will be enrolled and will each receive a single dose. The first patient is expected to be enrolled before the end of 2018, and data from the Cohort 3 are expected in mid-2019.
Thursday, September 27, 2018
Alexion acquisition bodes well for Argenx, says Nomura Instinet
Nomura Instinet analyst Christopher Marai believes Alexion Pharmaceuticals’ (ALXN) acquisition of Syntimmune highlights potential demand for FcRn therapies by acquirers and bodes well for Argenx (ARGX). The analyst keeps a Buy rating on Argenx with a $161 price target.
Quest Diagnostics acquires PhenoPath
Quest Diagnostics announced that it has acquired PhenoPath, a national provider of specialized anatomic pathology and related services. Based in Seattle, PhenoPath provides AP, molecular oncology and other services that complement and extend those of the company’s Advanced Diagnostics portfolio of services. These include services, such as genetic sequencing and genomic tumor analysis, which require higher-order expertise and skill. The PhenoPath business will operate as part of AmeriPath, a wholly owned business of Quest Diagnostics. The leading provider of anatomic pathology services, AmeriPath provides high-end pathology services to many ambulatory health and surgery centers, imaging centers, clinics and physician offices, and has on-site community pathologists in more than 170 hospitals across the country. Additional terms of the transaction were not disclosed.
Alnylam weakness on safety concerns a buying opportunity, says Piper Jaffray
Piper Jaffray analyst Edward Tenthoff attributes the initial weakness today in shares of Alnylam Pharmaceuticals to safety concerns from the interim Phase III givosiran data in acute hepatic porphyrias. Safety was consistent with prior Phase I data showing no additional deaths or cases of pancreatitis, five serious adverse events were due to underlying disease and one discontinuation for liver transaminase elevation resolved, Tenthoff tells investors in a research note. The analyst says he’s a buyer of Alnylam due to the unmet medical need in acute hepatic porphyrias. Tenthoff reiterates an Overweight rating on Alnylam with a $160 price target. The stock is down 1%, or $1.12, to $90.21 in early trading.
GW Pharmaceuticals up as DEA moves cannabis-derived med to low risk
Shares of GW Pharmaceuticals jumped in late morning trading after the company said Epidiolex will be now classified as a Schedule V controlled substance, the lowest ranked class, instead of a Schedule I drug, which had been widely expected. DEA RESCHEDULES EPIDIOLEX: GW Pharmaceuticals announced this morning, along with its U.S. subsidiary Greenwich Biosciences, that the U.S. Drug Enforcement Administration has rescheduled the company’s Epidiolex oral solution. Instead of a Schedule I drug, the DEA will classify the cannabis-derived drug as a Schedule V controlled substance, the lowest restriction classification. WHAT’S NOTABLE: Epidiolex was approved in June by the U.S. Food and Drug Administration to treat patients two years and older with Dravet Syndrome and Lennox-Gastaut Syndrome, two rare forms of epilepsy that emerge during childhood. Epidiolex is derived from cannabidiol, or CBD, a molecule contained in the marijuana plant. Marijuana is considered a Schedule I drug, which are drugs seen to have high abuse potential, no medical use and severe safety concerns. Schedule V drugs, meanwhile, including some cough medicines containing codeine, have a currently accepted medical use in treatment. EXECUTIVE COMMENTARY: In a statement, GW Pharmaceuticals CEO Justin Gover said he is “pleased” that the DEA has placed Epidiolex in the lowest restriction schedule, “because it will help ensure that patients with LGS and Dravet syndrome, two of the most debilitating forms of epilepsy, can access this important new treatment option through their physicians.” Gover said the company will “work hard” to make Epidiolex available within the next six weeks. ANALYST COMMENTARY: Cantor Fitzgerald analyst Elemer Piros said the DEA’s rescheduling of Epidiolex is the “best possible outcome” for GW Pharmaceuticals, as the company has now checked off the last box to prepare for U.S launch of the drug within the next six weeks. The analyst reiterated an Overweight rating and $211 price target.
Mirati Therapeutics weakness a buying opportunity, says Cowen
Cowen analyst Chris said he sees the pullback in Mirati Therapeutics shares as an attractive buying opportunity and reiterates his Outperform rating on the stock. There was never a reason to expect that Mirati’s data was under consideration for a late-breaking abstract presentation at ESMO, Sibutani tells investors. He expects management to share their plans for the sitravatinib + nivolumab pivotal study program at the ESMO meeting after data is presented on Monday, October 22. The analyst maintains an Outperform rating on Mirati shares.
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