Clinical Trials
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Brainstorm’s ALS Data Fail to Persuade FDA Advisors, Who Vote Down the Stem Cell Therapy
NurOwn, Brainstorm Cell Therapeutics’ experimental stem cell therapy for ALS, did not win the backing of an independent panel of FDA advisors. Many advisory committee members said they want to see more data from another clinical trial, the same guidance the FDA has given the biotech for nearly three years.
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Ionis Posts Trial Data in Rare Disease That Position It to Finally Set Out on Its Own
Ionis Pharmaceuticals’ olezarsen has Phase 3 results showing the therapy handily beat a placebo at reducing fat levels in the blood due to a rare, inherited metabolic disorder with no FDA-approved drugs. Ionis plans early 2024 submissions for what could become the first medicine it commercializes without a partner.
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Payer’s Place: Dawn Maroney
Dawn Maroney, President, Markets of Alignment Health and CEO of Alignment Health Plan, to discuss how they are using technology to provide better service and care to consumers.
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Contamination Risk Sparks a Recall of GSK, Scynexis Antifungal Drug
Scynexis said no adverse events or contamination problems have been reported for Brexafemme, an antifungal approved for treating vaginal yeast infections. The biotech said the risk of cross-contamination stems from the processes of the small molecule’s third-party manufacturer.
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How Genome Sequencing Can Broaden the Scope of Newborn Screening
Standard newborn screening can detect dozens of inherited diseases, but genomics technology enables testing for hundreds of them. A North Carolina program evaluating the feasibility of bringing genomics to newborn screening is expanding with an initial goal of testing 5,000 babies in the next year.
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Once a Blockbuster Prospect, Is Travere’s Kidney Drug on Track to Become a Bust?
Seven months after Travere Therapeutics’ flagship drug received accelerated FDA approval in a rare kidney disease, the molecule has fallen short of the key goal of its confirmatory test. It’s the small molecule’s second Phase 3 failure this year.
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FDA Rejects ARS Pharma’s Nasal Spray Alternative to Injectable Epinephrine
ARS Pharmaceuticals frames its intranasal epinephrine spray as a needle-free alternative to products such as EpiPen. Though this spray won the backing of an FDA advisory committee, the agency is now requiring that ARS Pharma run another study to support a regulatory submission.
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BeiGene, Novartis Drug Alliance Ends; It’s Their Second Terminated Deal This Year
BeiGene welcomes the return of the immunotherapy’s rights as an opportunity to make it the backbone of its solid tumors strategy. But Novartis cedes an opportunity to gain a competitor to established cancer immunotherapies from Merck and Bristol Myers Squibb.
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BioNTech and CEPI Team Up to Develop Mpox Vaccine That Meets 100-Day Goal
The Coalition for Epidemic Preparedness Innovations, or CEPI, has pledged up to $90 million to BioNTech’s messenger RNA vaccine candidates for mpox. This research could help advance the scientific understanding of the application of mRNA technology to Orthopoxviruses, which includes mpox, formerly known as monkeypox.
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FDA Approves GSK Myelofibrosis Med That Has Edge Over Others in Drug Class
FDA approval of GSK’s Ojjaara in myelofibrosis introduces a new competitor to blockbuster Incyte drug Jakafi. Ojjaara was part of GSK’s $1.9 billion acquisition of Sierra Oncology last year.
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Applying Remote Patient Monitoring to Surgery Prep and Recovery, Oncology and Women’s Health
Join us to learn about the latest trends in remote monitoring and how to extend its benefits beyond chronic conditions to more patients – all while using fewer staff resources.
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RayzeBio, Neumora Perk Up the IPO Market, Raising $561M for Clinical Trials
RayzeBio’s IPO will support pivotal testing of a targeted radiopharmaceutical for cancer patients who progress after treatment with Novartis’s Lutathera. Neumora will apply its IPO cash toward Phase 3 testing of a depression drug with a novel mechanism of action.
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While Auto vs. Allo Cell Therapy Debate Continues, VCs Look for ‘Next Shiny Thing’
Allogeneic cell therapy hasn’t caught up to autologous cell therapy yet, and the disappointing data so far has soured the entire allogeneic field for at least one venture capitalist. The cell therapy debate was one of the topics covered during a panel discussion at the Biopharm America conference.
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FDA Approves Updated Covid-19 Vaccines to Address Now Circulating Variants
The FDA said the updated messenger RNA vaccines from Pfizer/BioNTech and Moderna will protect against the omicron subvariants now prevalent in circulation. Left out of the FDA decision is Novavax, whose protein-based vaccine is still under regulatory review.
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Devices & Diagnostics, BioPharma
Click Expands to Substance Use Disorder With Indivior Digital Therapeutics Pact
Digital therapeutics developer Click Therapeutics is partnering with Indivior to develop a mobile app for substance use disorder. Indivior is now Click’s third development partner, following deals with Boehringer Ingelheim and Otsuka Pharmaceutical.
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Star Therapeutics Lands $90M to Expand Its Drug Discovery Universe
Startup Star Therapeutics will use the Series B funding to support clinical development of a drug candidate for von Willebrand disease, a bleeding disorder. The capital will also support the formation of new biotech subsidiaries developing antibodies with therapeutic applications in hematology and immunology.
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Senate Bill Proposes Slashing ‘Red Tape’ Around Biosimilar Interchangeability
Legislation introduced by Utah Senator Mike Lee would eliminate testing to show a biosimilar can be substituted for a reference biologic product. Lee contends the current testing requirement adds costs and delays market access for these lower-cost biological medicines.
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Payer’s Place: Dr. Anil Singh
Dr. Anil Singh shares his insights into the strategies employed by the organization to identify the most effective digital solutions for their members.
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Discover the Next-Gen Platform for Integrated Collaborative Care
Beyond EHRs and digital front doors, reducing the gaps in patient care journeys.
