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Home » Newsletters » BioWorld

BioWorld

March 12, 2019

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Axovant showcases early gene therapy data in Parkinson's, Tay-Sachs trials

Turnaround bets placed by Axovant Sciences Ltd. on early stage gene therapies in the wake of its failed bid to tackle Alzheimer's disease (AD) showed hints of paying off, boosting company shares (NASDAQ:AXGT) by 14.2 percent to $1.69 on Monday. Positive interim results from two patients in a phase II study of AXO-Lenti-PD in Parkinson's disease (PD) and initial data on its Tay-Sachs disease candidate, AXO-AAV-GM2, put both programs on track for further development. Read More

FDA once again a winner in Trump's 2020 budget, drug prices a target

Opening the yearly U.S. spending face-off, President Donald Trump Monday presented Congress with his proposed fiscal 2020 budget, which calls for a 12 percent cut in spending at the Department of Health and Human Services (HHS). Read More

Row continues over pricing of CF drug Orkambi in U.K.

LONDON – Vertex Pharmaceutical Inc. CEO Jeff Leiden and the U.K.'s health minister, Matt Hancock, met in London on Monday in a bid to end the impasse over the price of the cystic fibrosis drug Orkambi (lumacaftor/ivacaftor). Read More

Bacteria in crosshairs, Snipr nets $50M series A for dual effort in CRISPR

Snipr Biome ApS was "flying a little bit under the radar" until the company had "something worth listening to," CEO Christian Grondahl told BioWorld, and the day for talking arrived with the Copenhagen-based company raising $50 million in series A cash. Read More

U.S., Canadian firms tackle opioid crisis with subdermal implant

TORONTO – The Probuphine implant developed by San Francisco's Titan Pharmaceuticals Inc. comprises match-stick sized rods inserted into the patient's upper arm. It aims to provide an ongoing low dose of buprenorphine and help recovering addicts stick to their daily medication regimens. Montreal-based Knight Therapeutics Inc., which enjoys exclusive Canadian distribution rights for the implant, is gearing up for cross-country sales, notably in British Columbia, the province hardest hit by opioid abuse and fatalities. Read More

Maze Therapeutics raises $191M to unlock the genetic secrets of disease

DUBLIN – Maze Therapeutics Inc. recently secured $191 million in initial funding to join the ranks of biotechnology firms that aim to uncover new biology and new drug targets by sifting through large volumes of genomic and phenotypic data and testing the hypotheses those analyses generate by carrying out genome-wide perturbations within cellular disease models.  Read More

What's the next global disruption? Speakers offer views from China

SHANGHAI – The first Wuxi Healthcare Forum held in China was a notably upbeat and optimistic affair. Several panel moderators encouraged blue sky thinking by asking a variant on the same question: What technologies and advances are the most exciting or have the greatest potential to disrupt the industry in the next five to 10 years? Speakers from China, the U.K., Europe, the U.S. and Canada gamely shared their prognostications for the future. Read More

Financings

Correvio Pharma Corp., of Vancouver, British Columbia, said it made an amendment to its existing agreement with CRG Servicing LLC, which provides for an additional $10 million. The company will pay a small access fee to CRG. The additional funding may be drawn at Correvio's discretion in increments of $2.5 million through Sept. 30. Read More

Other news to note

Stemline Therapeutics Inc., of New York, licensed the worldwide rights to SL-1001, a RET kinase inhibitor, from CRT Pioneer Fund LP. Stemline plans to start clinical development of SL-1001 in 2020. Terms of the deal weren't disclosed. Read More

Clinical data for March 11, 2019

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Regulatory actions for March 11, 2019

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