Today’s Daily Dose brings you news about Alnylam Pharma’s regulatory catalyst in December; argenx’ generalized myasthenia gravis trial data; Atara’s multiple sclerosis trial data and stock offering; Merck’s deals and collaboration to develop an effective response to the COVID-19 pandemic; Novavax’ progress in a COVID-19 vaccine trial and Osmotica Pharma’s near-term catalysts.
1. Alnylam Pharma To Face FDA In December
Alnylam Pharmaceuticals Inc.’s (ALNY) New Drug Application for Lumasiran in development for the treatment of primary hyperoxaluria type 1 is under Priority Review by the FDA, with a decision expected on December 3, 2020.
Primary hyperoxaluria type 1 is an ultra-rare disease in which excessive oxalate production results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones and nephrocalcinosis. Currently, there are no approved pharmaceutical therapies for PH1.
Lumasiran is also under review by the European Medicines Agency.
ALNY closed Tuesday’s trading at $136.43, down 2.91%.
2. Argenx Reports Positive Data From Generalized Myasthenia Gravis Trial
Shares of argenx SE (ARGX) jumped over 34 percent on Tuesday, following positive topline data from a phase III trial of Efgartigimod in generalized myasthenia gravis, dubbed ADAPT.
Myasthenia gravis is a rare and chronic autoimmune disease characterized by debilitating and potentially life-threatening muscle weakness. More than 85% of people with MG progress to generalized MG (gMG) within 18 months, where muscles throughout the body may be affected, resulting in extreme fatigue and difficulties with facial expression, speech, swallowing and mobility.
The ADAPT trial met its primary endpoint defined as percentage of responders on the Myasthenia Gravis Activities of Daily Living (MG-ADL) score among acetylcholine receptor-antibody positive (AChR-Ab+) generalized myasthenia gravis (gMG) patients. Responders are defined as having at least a two-point improvement on the MG-ADL score for at least four consecutive weeks.
In light of the encouraging data, argenx plans to submit a Biologics License Application for Efgartigimod in generalized myasthenia gravis by the end of this year.
argenx was trading around $25 when we alerted our premium subscribers to the stock on November 23, 2017 (Report titled “A Catalyst-rich Year Ahead!”).
The stock touched an all-time high of $218.18 in intraday trading on Tuesday before closing at $212.12, up 34.29%.
3. Atara Reports Multiple Sclerosis Trial Data
Atara Biotherapeutics Inc.’s (ATRA) additional safety and efficacy data from its ongoing phase I study of ATA188 for the treatment of progressive forms of multiple sclerosis has revealed that sustained disability improvements at 6 months were also maintained at 12 months with ATA188.
ATA188 is an off-the-shelf, allogeneic Epstein – Barr virus (EBV) – specific T-cell immunotherapy. Though the development of MS likely requires more than a single causative factor, EBV is the only risk factor identified to date that appears to be necessary for MS.
ATA188 offers a unique approach with the advantage of selectively targeting EBV-infected B cells and plasma cells in the circulation and in the central nervous system (CNS), noted the Company.
In other news, Atara announced that it has commenced an underwritten public offering of shares of its common stock and, to certain investors, pre-funded warrants to purchase shares of its common stock.
ATRA closed Tuesday’s trading at $11.32, down 23.57%.
4. A Busy Day For Merck
It’s been a busy day for Merck (MRK), signing research collaborations and inking an acquisition deal as part of its efforts to develop an effective response to the COVID-19 pandemic.
Merck and Ridgeback Biotherapeutics LP, a closely held biotechnology company, have entered into a collaboration agreement to develop EIDD-2801, an orally available antiviral candidate currently in early clinical development for the treatment of patients with COVID-19. In phase 1 testing, EIDD-2801 has been found to be well-tolerated.
As part of the agreement, Merck, through a subsidiary, will gain exclusive worldwide rights to develop and commercialize EIDD-2801 and related molecules. Ridgeback Bio will receive an undisclosed upfront payment, specified milestones, and a share of the net proceeds of EIDD-2801 and related molecules if approved. Merck will be responsible for clinical development, regulatory filings, and manufacturing.
Another research collaboration that Merck signed was with IAVI, a nonprofit scientific research organization, to develop an investigational vaccine against SARS-CoV-2 to be used for the prevention of COVID-19.
Under this agreement IAVI and Merck will work together to advance the development and global clinical evaluation of an IAVI-designed and engineered SARS-CoV-2 vaccine candidate. The vaccine candidate by the name rVSV is in preclinical development, and clinical studies are planned to start later in 2020. Merck will lead regulatory filings globally.
In yet another move to develop an effective COVID-19 vaccine in the near term, Merck has agreed to acquire privately-held Themis, which has a COVID-19 vaccine candidate in preclinical studies. The financial terms of the deal aren’t disclosed.
The clinical studies of Themis’ COVID-19 vaccine candidate are expected to start later this year.
MRK closed Tuesday’s trading at $77.26, up 1.17%.
5. Novavax To Report Initial Data From COVID-19 Vaccine Trial In July
Novavax Inc. (NVAX) has enrolled the first participants in a phase I/II clinical trial of its coronavirus vaccine candidate, NVX-CoV2373.
The trial is being conducted in two parts – phase I and phase II.
The phase I portion is designed to evaluate the immunogenicity and safety of NVX-CoV2373 and will enroll 130 healthy participants 18 to 59 years of age at two sites in Australia. The preliminary immunogenicity and safety results from the phase I portion are expected in July 2020.
The phase II portion, which will begin promptly following successful phase I results, is expected to be conducted in multiple countries, including the United States, and would assess immunity, safety, and COVID-19 disease reduction in a broader age range.
NVAX closed Tuesday’s trading at $48.17, up 4.47%.
6. Osmotica Pharma Gearing Up For Regulatory Catalyst
Osmotica Pharmaceuticals plc (OSMT) has a couple of catalysts to keep an eye on in the coming months.
The Company’s investigational product, RVL-1201 for the treatment of acquired blepharoptosis, or droopy eyelid, awaits the FDA decision on July 16, 2020. It also remains on track to submit an amended New Drug Application for Arbaclofen ER for spasticity associated with Multiple Sclerosis in the second quarter of this year.
Brian Markison, Chief Executive Officer, and Andrew Einhorn, Chief Financial Officer of Osmotica will be presenting at the 2020 Jefferies Virtual Healthcare Conference on June 3rd.
OSMT closed Tuesday’s trading at $5.32, up 19.02%.
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