DUBLIN – Roche Holding AG has joined Jnana Therapeutics Inc. in its quest to drug the human repertoire of solute carrier (SLC) metabolite transporters as a means of tackling a swath of immune-mediated and neurological diseases. The Basel, Switzerland-based pharma is paying Boston-based Jnana $40 million up front and could pay over $1 billion more in research funding, preclinical, development and commercial milestones, as well as sales royalties, in a multitarget deal.
Acadia shares nicked on phase III top-line data with Nuplazid in MDD
Acadia Pharmaceuticals Inc. unveiled top-line results from its 298-patient phase III effort called Clarity with Nuplazid (pimavanserin) in major depressive disorder (MDD). The experiment, which combined two identical, double-blind, placebo-controlled MDD studies, yielded an efficacy and safety analysis that showed Clarity did not hit statistical significance on the primary endpoint: the 17-item Hamilton Depression Rating Scale (HAMD-17) total score change from baseline to the fifth week. Pimavanserin 34 mg, given once daily as an adjunctive treatment to standard antidepressant therapy, was associated with a mean reduction of 9 in HAMD-17 total score compared to 8.1 for placebo as an adjunctive treatment (p=0.296), the San Diego-based firm said. Nuplazid was approved in April 2016 for Parkinson’s disease psychosis. In other news, the FDA accepted for standard review – rather than priority treatment, as Acadia expected – the sNDA for Nuplazid to treat hallucinations and delusions associated with dementia-related psychosis with a PDUFA date of April 3, 2021. Shares (NASDAQ:ACAD) were trading midday at $45.10, down $10.32, or 18%.
Fulcrum, Myokardia collaborate on therapies for genetic cardiomyopathies
Fulcrum Therapeutics Inc. and Myokardia Inc. agreed to discover, develop and commercialize therapies for treating genetic cardiomyopathies. Myokardia will use Fulcrum’s target discovery engine for identifying therapeutics to control gene expression driving certain undisclosed genetic targets. When the transaction closes, Fulcrum will receive $12.5 million and may be eligible for research, development and commercial milestone payments. Fulcrum could also be reimbursed up to a $302.5 million for a first product passing through development and commercialization. Myokardia will develop and commercialize any therapeutics discovered through the collaboration. Cambridge, Mass.-based Fulcrum (NASDAQ:FULC) and Brisbane, Calif.’s Myokardia (NASDAQ:MYOK) shares were essentially flat at midday Tuesday.
COVID-19 vaccines on track, but uptake will remain a question
A safe, effective COVID-19 vaccine likely will be available by the end of the year or early next year, as will the supplies needed to deliver and administer the vaccines. That’s the message officials of five biopharma companies delivered to a House subcommittee today as they updated U.S. lawmakers on the progress being made on the vaccine front. While that’s good news, questions remain about how delivery of a vaccine will be prioritized and what impact a growing vaccine hesitancy will have on the public’s willingness to get vaccinated.
Neurological diseases index pushes higher this month
With an increasing number of neurological manifestations being reported in patients suffering from COVID-19 infection, the need for therapies to treat these conditions and other major CNS disorders has, once again, been highlighted. This may be one of the reasons why investors have turned their attention to the space once again with several members of the BioWorld Neurological Diseases index enjoying an uptick in their share valuations. These performances have helped push the index into positive territory for the first time this year.
Virtual meetings bring new challenges to adcoms
Interludes of classical music. Little kids talking in the background. Unmuted mics as panelists multitask. Periods of silence as speakers forget to unmute. And then the technical problems – lots of them. Such are the challenges of addressing a virtual FDA advisory committee in the time of COVID-19. “This is an important, critical day for the sponsor,” Steven Romano, executive vice president and chief scientific officer for Mallinckrodt plc, said of going before any adcom. Doing it virtually calls for a lot more sponsor preparation for what is already a nerve-wracking experience. Last week, Mallinckrodt and Glaxosmithkline plc were the first sponsors to have virtual adcoms in which the panelists voted on recommending FDA approval for a new drug. Both meetings were interesting experiences that other sponsors facing a virtual adcom could learn from.
Tubulis raises $12M in series A round to join ADC ‘renaissance’
DUBLIN – Tubulis GmbH raised €10.7 million (US$12.3 million) in a series A round to progress a next-generation antibody-drug conjugate (ADC) platform, which, it claims, will improve both the stability of ADC constructs and the efficiency with which they are generated. The Martinsried, Germany-based company was spun out of two German academic institutions, the Leibniz Research Institute in Berlin (FMP) and the Ludwig Maximilians University (LMU) in Munich, in 2019 and has, until now, been funded by a nondilutive grant of €3.4 million.
Strata Oncology, Elevation Oncology partner on CRESTONE enrollment
Strata Oncology Inc. has inked an agreement with newcomer Elevation Oncology Inc. to ramp up patient identification and enrollment for the latter’s phase II CRESTONE study evaluating the use of seribantumab in patients with recurrent, locally advanced or metastatic solid tumors of any origin that have a neuregulin-1 (NRG1) gene fusion. “NRG1 fusions are rare oncogenic drivers present across several tumor types, like lung and pancreatic,” explained Dan Rhodes, CEO of Strata Oncology. “Through system-wide delivery of StrataNGS, our tumor molecular profiling test optimized for small tissue samples, we believe our nationwide network of health systems will help drive identification and enrollment of patients into this ... study.” For its part, Elevation Oncology has made a splash with $32.5 million in funding in its debut.
Takeda puts Twist on innovative R&D efforts
HONG KONG – Takeda Pharmaceuticals Inc. is gaining access to the “library of libraries,” inking an agreement with Twist Bioscience Corp. in one of the Osaka, Japan-based firm’s recent efforts to build a sustained innovative R&D growth engine. The past few years has seen Takeda complete its $62 billion acquisition of rare disease specialist Shire plc and, more recently, divestitures of a primary care portfolio and noncore assets, with a pledge to focus on next-generation therapies involving a range of modalities. The recent agreement with Twist will give Takeda access to phage display libraries and help develop new antibody candidates. The two companies will use the library resource to discover, validate and optimize new drugs in the oncology, rare diseases, neuroscience and gastroenterology fields.
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