While Cytokinetics’ Phase III data for reldesemtiv missed the primary endpoint of slow vital capacity at 12 weeks, the drug did show benefits over placebo in amyotrophic lateral sclerosis, Piper Jaffray analyst Edward Tenthoff tells investors in a research note. He points out the company intends to advance reldesemtiv into a Phase III ALS trial. The analyst reiterates an Overweight rating on Cytokinetics and boosted his price target for the shares to $14 from $13.
Monday, May 6, 2019
Ovid up on OV101 data in rare inherited disorders
Ovid Therapeutics (NASDAQ:OVID) is up 16% premarket on robust volume on the heels of two presentations at the American Academy of Neurology Annual Meeting in Philadelphia on lead candidate OV101.
The first highlighted data from the Phase 2 STARS study in adolescents and adults with Angelman syndrome, an inherited condition affecting the central nervous system. A Phase 3 study in children with Angelman, NEPTUNE, should launch in H2.
The second pertained to the approaches to assess outcomes in Fragile X syndrome as a precursor to data the company expects to announce in H2 from the ROCKET trial.
OV101 (gaboxadol) is a delta (δ)-selective GABAA receptor agonist designed to specifically target the disruption of tonic inhibition, a central physiological process of the brain that is believed to be the cause of certain neurodevelopmental disorders.
Abbott renews smartphone-based cardiac monitor with more accurate detection
Abbott has launched a new, smarter version of its small implantable monitor for detecting irregular heartbeats, that the company says now reduces the rate of false positives by 97%.
About the size of a paperclip, the Confirm Rx insertable cardiac monitor connects directly to a smartphone app via Bluetooth, to offer continuous remote detection of arrhythmias without the need for additional transmitter hardware.
The loop recording device, which has been cleared by the FDA and received a CE mark, is placed just under the skin above the heart during an outpatient procedure. Additionally, Abbott’s mobile app is available in nearly 40 languages.
Abbott first picked up the Confirm Rx monitor and its smartphone app through its $25 billion acquisition of St. Jude Medical in January 2017. The device made its international commercial debuts later that year, including in Europe and the U.S.
The tracker is aimed at the estimated 33 million people living with the most common irregular heartbeat, atrial fibrillation, with many going undiagnosed.
It’s also designed for people with active lifestyles, and offers 24/7 monitoring compared to devices that need to be removed and placed in a charging station, which can lead to gaps in the detection of sporadic and infrequent arrhythmias.
BioMarin: EC OKs Palynziq (pegvaliase injection) for Phenylketonuria
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the European Commission (EC) has granted marketing authorization for Palynziq® (pegvaliase injection) at doses of up to 60 mg once daily, to reduce blood phenylalanine (Phe) concentrations in patients with phenylketonuria (PKU) aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first enzyme substitution therapy approved in Europe to target the underlying cause of PKU by helping the body to break down Phe. In addition, the EC acknowledged that the Phase 3 trial and extension study is suggestive of an improvement in inattention and mood symptoms.
On March 1, 2019 BioMarin announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), adopted a positive opinion for the company’s Marketing Authorization Application (MAA) for Palynziq. This is BioMarin’s second approved treatment for this rare genetic disease.
Bruker price target raised to $46 from $39 at Needham
Needham analyst Stephen Unger raised his price target on Bruker to $46 and kept his Strong Buy rating after its Q1 earnings beat, also raising his FY19 EPS view to $1.60 from $1.56 on the expected contribution of its RAVE LLC acquisition. The analyst adds that he had some reservations around Bruker’s growth in Asia-Pacific heading into the print, but the segment generated 19.9% revenue growth to $152M, above his forecasted $132M.
Lundbeck Acquires Abide Therapeutics for $250M, Gains San Diego Discovery Site
With a $250 million cash down payment, Denmark’s H. Lundbeck A/S snapped up San Diego-based Abide Therapeutics to gain a novel discovery platform and a U.S.-based research hub.
Deborah Dunsire, president and chief executive officer of Lundbeck said the acquisition of Abide provides the company with a “differentiated chemo-proteomic platform to discover new classes of drugs for a broad spectrum of brain diseases starting with those that harness the therapeutic potential of the endocannabinoid system.” Dunsire said Abide’s research and development program provides both an immediate and long-term boost to its pipeline. Bringing Abide under the Lundbeck portfolio puts the Danish company in a position to deliver multiple new and transformative treatments for brain diseases, Dunsire said in a statement.
In its announcement, Lundbeck did not provide details regarding the 40 employees at Abide, including its leadership team. Kevin Finney, who joined Abide in January as president and chief operating officer, told Xconomy that the company had actually been in the midst of a financing round and eying an initial public offering when the Lundbeck offer came along. The deal was too good to pass up, Finney said, according to the report.
Abide Chief Executive Officer Alan Ezekowitz noted Lundbeck’s commitment to developing therapies for the brain and said it was that expertise that convinced the company’s board of directors to support the deal. Lundbeck, he said, “was the best way to attain Abide’s goal to develop novel therapeutics that make a fundamental difference in the lives of patients with a range of neurological and mood disorders.” Ezekowitz added that Lundbeck’s support for the La Jolla discovery site means that we can continue to leverage the insights of co-founder Ben Cravatt’s laboratory at Scripps Research and maintain its “outstanding discovery team.”
What attracted Lundbeck to acquiring Abide is that company’s lead molecule, ABX-1431. Abide’s ABX-1431 is a potent selective inhibitor of the serine hydrolase monoacylglycerol lipase (MGLL). ABX-1431 is designed to increase the power of endocannabinoid signaling to restore homeostatic balance in the central nervous system. The company believes it has the potential to address multiple indications in psychiatry and neurology. ABX-1431 is currently in an exploratory Phase IIa trial for the treatment of Tourette syndrome and a Phase I trial for neuropathic pain. In addition to the clinical and pre-clinical programs targeting MGLL, Abide has a rich pipeline of inhibitors targeting other serine hydrolases that may be pursued as future novel treatments.
In addition to the $250 million paid upfront to Abide, Lundbeck will also pay an additional $150 million in future development and sales milestones. After the deal closes, Abide’s laboratory in La Jolla will become a U.S. drug discovery hub for Lundbeck. Lundbeck is covering the costs of the acquisition of Abide from its existing cash reserves. The transaction is expected to close during the second quarter of 2019. Lundbeck said the deal will not have any impact on Lundbeck’s 2019 financial guidance range provided in February 2019.
GlycoMimetics completes enrollment for Phase 3 trial evaluating rivipansel
GlycoMimetics (GLYC) announced that patient enrollment has been completed in the Phase 3 RESET clinical trial, which is evaluating the efficacy and safety of rivipansel for the acute treatment of vaso-occlusive crisis in sickle cell disease. In 2011, GlycoMimetics and Pfizer Inc. (PFE) entered into a worldwide license agreement for the development and, if approved by applicable regulatory authorities, commercialization of rivipansel. Since completion of the Phase 2 clinical trial, Pfizer, has been responsible for clinical development of rivipansel, including the RESET clinical trial. Under the Company’s license agreement, GlycoMimetics is eligible to receive payments of up to $80M upon the achievement of specified development milestones, up to $70 million upon the achievement of specified regulatory milestones, and up to $135M upon the achievement of specified levels of annual net sales of licensed products. GlycoMimetics is also eligible to receive tiered royalties, with percentages ranging from the low double digits to the low teens, based on net sales of rivipansel worldwide
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