Vanda claims that two generics to its sleep disorder drug Hetlioz were approved despite inadequate data and is requesting that FDA Commissioner Marty Makary revisit the decision.
Krystal Biotech’s decision follows the FDA’s rejection last month of Replimune’s RP1, which works similarly to Krystal Biotech’s KB707. The biotech said this has introduced “heightened uncertainty” regarding a potential accelerated pathway for the candidate.
Among the problems cited were cat hair, bacterial contamination and instrument defects.
Washington has had notable life sciences job growth over the past 10 years, according to a Life Science Washington report. The association’s CEO and a biopharma CEO discuss what makes the state stand out, including its talent pool, AI leadership and entrepreneurship support.
In this episode presented by Cresset, BioSpace’s head of insights Lori Ellis discusses clinical trial fail rates and AI’s potential to reduce preclinical costs with Mutlu Dogruel, VP of AI and Mark Mackey, CSO of Cresset.
Stealth BioTherapeutics originally submitted elamipretide for approval in January 2024, only to receive a rejection in May of this year. In addition to accepting the biotech’s resubmission, the FDA has promised to take action on the candidate by Sept. 26.
FEATURED STORIES
After Merck noted the issue in its Q2 earnings call without providing specifics, analysts are left in the dark about the HPV vaccine’s future in China.
Experts say the time is now to develop and provide widespread access to genetic medicines for the rarest diseases. What’s more, they say it is a moral imperative.
Longeveron and Lexeo Therapeutics are working on CGT therapies to treat Alzheimer’s disease, but it’s not clear whether they have a better chance of success than traditional approaches.
Active immune therapies hold promise for preventing or slowing disease onset, but some experts warn of potential safety risks.
Ultracompact CRISPR systems, which are in some cases one-third the size of Cas9, are being designed to be more specific and enable in vivo gene editing in difficult to reach tissues.
The BIOSECURE Act’s prohibition on doing business with China-based companies may have implications for biotech and biopharma markets on both sides of the Pacific.
LATEST PODCASTS
In this episode of Denatured presented by AnaptysBio, Jennifer Smith-Parker speaks to Dr. Joe Murray, Mayo Clinic; Marilyn Geller, the Celiac Disease Foundation; and Dr. Paul Lizzul, AnaptysBio, about the challenges and opportunities facing celiac disease treatment.
Pfizer deals again in obesity space as Wave and Structure drop splashy weight loss results; what CDER Director Richard Pazdur’s sudden retirement means for biopharma; neuro diseases take center stage at CTAD; and more.
In this episode of Denatured, Jennifer C. Smith-Parker speaks to Stacey Adam, PhD, Vice President of Science Partnerships at the Foundation for the National Institutes of Health and Patrick Smith, Senior Vice President, Translational Science at Certara, to discuss the latest regulatory news and the future for new approach methologies (NAMs) development.
Job Trends
Arkuda Therapeutics today announced that it entered into an option and asset purchase agreement with Janssen Pharmaceuticals, Inc. (Janssen), a Johnson & Johnson company, which grants Janssen an exclusive option to purchase Arkuda’s portfolio of lysosomal function enhancers.
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SPECIAL EDITIONS
In this deep dive, BioSpace investigates China’s rise as a biotech powerhouse.
In this deep dive, BioSpace explores the next big thing in obesity.
BioSpace did a deep dive into biopharma female executives who navigated difficult markets to lead their companies to high-value exits.
DEALS
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The proposed regulatory framework provides a tougher stance on mergers, impacting industries such as biopharma where the FTC’s recent lawsuit seeks to block Amgen’s Horizon Therapeutics buy. -
The Swiss pharma is expanding its neuroscience pipeline with an upfront $500 million payment to DTx Pharma and additional payments of up to $500 million upon completion of certain milestones. -
The Federal Trade Commission is asking for more information regarding Pfizer’s planned $43 billion acquisition of Seagen, according to the latter’s Securities and Exchange Commission filing on Friday.
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This week: Cancer license deals from J&J and BeiGene, a potential $7B acquisition by Roche and confirmed $1.9B Lilly buy, EU fine for Illumina, and more legal challenges to the Inflation Reduction Act -
Eli Lilly said Friday it plans to pay up to $1.925 billion to acquire Versanis and its lead asset, bimagrumab, a monoclonal antibody that aims to reduce fat mass without affecting muscle mass.
WEIGHT LOSS
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Eli Lilly has partnered with Amazon Pharmacy to help fill online orders of its obesity drug Zepbound—as well as migraine and diabetes medicines—placed through the pharma’s online portal LillyDirect. -
Novo Nordisk’s blockbuster weight-loss drug Wegovy was approved on Friday by the FDA to reduce the risk of cardiovascular death, heart attack and stroke in adults who have cardiovascular disease and are obese or overweight. -
Novo Nordisk’s early-stage amylin and GLP-1 co-agonist elicited a 13.1% reduction in body weight, with an overall favorable safety profile, the Danish drugmaker reported Thursday at an investor event. -
After stopping the study early due to strong efficacy, Novo Nordisk released data from the FLOW study showing significant benefits of semaglutide in patients with type 2 diabetes and chronic kidney disease. -
Eli Lilly is planning to launch its blockbuster diabetes drug Mounjaro next year in India, the world’s most populous nation, ahead of its main competitor Novo Nordisk which is eyeing an Indian launch of Wegovy in 2026.
POLICY
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Vertex has filed a complaint against the Department of Health and Human Services, seeking to make its fertility preservation program available to federally insured patients needing Casgevy treatment. -
Why I advocated on Capitol Hill this month for the renewal of the FDA’s Priority Review Voucher program -
The Federal Trade Commission criticized the business practices of pharmacy benefit managers this week, but drugmakers are also at fault for the high costs of medicines.
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The Senate on Thursday unanimously passed a bill aimed at limiting the number of patents drugmakers can introduce and making it easier for generic and biosimilar competitors to enter the market. -
The Federal Trade Commission plans to file lawsuits against the three largest pharmacy benefit managers over allegedly steering patients away from less expensive drugs, according to The Wall Street Journal.
To navigate each step along the way from an interview to a final offer, you should have a clear understanding of a timeline of events. These job search tips can help you with that.
Have you thought about what benefits are important to you? Would you request certain benefits from your current employer, or during an interview for a new job?
Most people avoid networking connections due to uncomfortable situations. In contrast, strategic networking can be extremely beneficial for your career.
According to a recent BioSpace survey, 69% of employers indicated “finding quality talent” will be a significant challenge in 2019. Are you the quality talent life science employers are looking for? Don’t delay, apply now and show these employers you possess the quality skills they need!
There are many common misbeliefs around chronic procrastinators. they don’t care about work; they are lazy, etc. Here are some steps for overcoming procrastination.
Are you familiar with the medical writing profession? If not, you could be missing out on one of the most flexible and rewarding careers in life sciences.
HOTBEDS
REPORTS
In this Employment Outlook report, BioSpace explores current workforce sentiment, job activity trends and the prospective job and hiring outlook for 2025, particularly as it compares to the previous year.
BioSpace’s third report on diversity, equity, inclusion and belonging in life sciences examines dramatic shifts in attitude around diversity initiatives.
CANCER
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The hold on BioNTech and MediLink’s antibody-drug conjugate candidate BNT326/YL202 has halted enrollment in a Phase I U.S. trial in patients with non-small cell lung cancer or breast cancer, following multiple deaths. -
Coherus BioSciences and Junshi Biosciences’ PD-1 inhibitor Loqtorzi significantly boosted progression-free and overall survival in a late-stage study of patients with advanced hepatocellular carcinoma. -
The use of artificial intelligence in the development of cancer vaccines allows for individualized therapy, but the prospect of an ever-changing product poses new challenges for drug developers and regulators. -
AbbVie’s antibody-drug conjugate Elahere, developed by ImmunoGen, elicited a nearly 52% objective response rate in heavily pretreated patients with folate receptor-alpha-positive, platinum-sensitive ovarian cancer. -
BMS presented late-stage results on Tuesday at the ASCO annual meeting which showed the combination of Opdivo and Yervoy lowered the risk of death by 21% in patients with unresectable hepatocellular carcinoma, compared to two kinase inhibitors.
NEUROSCIENCE
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After showing some unfavorable results for its data, AC Immune on Monday announced it will be getting back its Alzheimer’s disease candidates from Roche’s Genentech. -
With recent scientific advances, milestone approvals and increased dealmaking, the future of treatment for neurological diseases looks brighter—but continued investment, collaboration and patient-focused efforts are key. -
The deal, announced late Wednesday, will provide AbbVie with access to Cerevel Therapeutics’ pipeline of clinical-stage and preclinical candidates for psychiatric and neurological diseases. -
For forms of Alzheimer’s, frontotemporal dementia and Parkinson’s caused by genetic defects, gene therapy could change the treatment landscape. -
To protect the central nervous system, the blood-brain barrier bars entry to around 98% of molecules—but approaches like Roche’s trontinemab could spell new hope in Alzheimer’s and beyond.
CELL AND GENE THERAPY
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Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a non-profit sponsor to win FDA approval. -
For traditional approval, CAR T therapies will need to establish superiority over current standard treatments, including already-approved CAR T products. -
As big pharmas including Takeda and Novo Nordisk flee the cell therapy space and smaller biotechs shutter their operations, these players are sticking around to take the modality as far as it can go. -
The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access to patients two years and older in what one Stanford Medicine professor called a “game changing advance” for the field. -
Experts suggest the FDA’s Advanced Manufacturing Technologies designation could be a lifeline for improving production processes for approved cell and gene therapies.
