Nightstar Therapeutics initiated with a Buy at UBS. UBS analyst Carter Gould initiated Nightstar Therapeutics with a Buy rating and $21 price target.
Friday, January 4, 2019
KalVista expects KVD900 Phase 2 HAE trial data in 2019
KalVista Pharmaceuticals provided a development update on its oral plasma kallikrein inhibitor portfolio. “In December we filed with regulatory authorities to begin our Phase 2 study of KVD900 as a potential oral acute treatment for hereditary angioedema, or HAE. As previously announced, this enlarged study is expected to provide data in late 2019. We continue to be excited by the potential for KVD900 to provide a safe, oral on-demand option for HAE patients to more conveniently and effectively manage their disease,” said CEO Andrew Crockett. “We are also pleased to announce that we made the regulatory filings for our next oral plasma kallikrein inhibitor, KVD824, and expect to begin dosing that first-in-human trial soon. We expect to provide a further update on KVD824 around mid-year.” Following the necessary regulatory approvals, the Phase 2 trial evaluating KVD900 as an on-demand treatment for HAE attacks will begin dosing in approximately 50 patients at over 10 European clinical sites. The study will recruit type 1 and 2 HAE patients who have had three attacks in 90 days prior to enrollment. During the first part of this two-part study patients will receive a single 600 mg dose of KVD900 to explore pharmacokinetic and pharmacodynamic properties. All patients will then enter part two of the study, which is a crossover investigation in which the efficacy of KVD900 will be assessed versus placebo across two attacks. Patients experiencing an attack will take a single dose of 600 mg of KVD900 or placebo within one hour of the start of the attack. The second attack will be dosed with the other treatment. For all attacks, symptom severity will be monitored and additional data points will be collected for at least 24 hours. Patients will use their normal, on-demand treatment as required.
Biogen, C4T to develop potential treatments for neurological conditions
Biogen and C4 Therapeutics, or C4T, announced that they have entered into a strategic collaboration to investigate the use of C4T’s novel protein degradation platform to discover and develop potential new treatments for neurological conditions, such as Alzheimer’s disease and Parkinson’s disease. Under the agreement, C4T will provide expertise and research services in targeted protein degradation and Biogen will provide neuroscience expertise and drug development capabilities. Biogen and C4T will research potential targets together and Biogen will advance candidates for development and potential commercialization. Biogen will pay C4T up to a total of $415M in upfront and potential future milestone payments plus potential future royalties. Biogen expects to record a research and development expense of $15M-$25M in Q4.
Biogen, Skyhawk in collaboration for neurological disease treatment
Biogen, Skyhawk enter strategic collaboration for neurological disease treatment Biogen and Therapeutics announced a strategic collaboration in which the companies will leverage Skyhawk’s SkySTAR technology platform with the goal of discovering innovative small molecule treatments for patients with neurological diseases. Biogen will have the option to license therapies resulting from the collaboration and will be responsible for their development and potential commercialization. The agreement grants Biogen an exclusive license to worldwide intellectual property rights on research-stage therapeutic candidates for the treatment of multiple sclerosis, spinal muscular atrophy and additional neurological disorders. As part of the agreement, Skyhawk received an upfront payment of $74M from Biogen and may receive potential future milestone payments and royalties. A portion of the upfront payment will be allocated to future research services, with the remainder expensed in Q1 as research and development.
Flexion price target lowered to $36 from $42 at Needham
Needham analyst Serge Belanger lowered his price target on Flexion to $36 after its yesterday’s guidance for FY19 Zilretta sales of $65M-$80M came in below the Street consensus of $97.8M. The analyst states that although the outlook may appear disappointing at first, investors may welcome the “expectation reset” that should yield a “more accurate guidepost” for Zilretta growth. The analyst also lowered his FY19 EPS view to ($3.89) from ($2.63) to reflect the slower launch ramp but kept his Buy rating on the shares based on the company’s potential for its product to serve as the front-line treatment for Osteoarthritis knee pain.
Catalyst Pharmaceuticals announces publication of Firdapse trial data
Catalyst Pharmaceuticals announced the online publication in Sage Open Medicine of the results of an investigator-sponsored Phase IIb clinical trial evaluating Firdapse for the treatment of MuSK antibody positive Myasthenia Gravis, or MuSK-MG. The positive topline results from this trial were previously announced, but the full results of safety, efficacy and other clinical data are now available. The MSK-001 trial was a randomized, double-blind, placebo-controlled, double crossover design. The co-primary endpoints were statistically met as well as the secondary endpoints. The study provided evidence that amifampridine phosphate was safe and effective in treating MuSK-MG patients. The study was conducted in Milan, Italy.
MannKind to host conference call
Conference call to discuss company developments will be held on January 4 at 9 am.
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