Roche and Genentech were unable to sufficiently demonstrate the benefit of using Columvi in an earlier treatment setting for DLBCL in a U.S. population, according to the FDA.
The rollercoaster week for Sarepta Therapeutics continued, with shares of the embattled gene therapy-turned-siRNA biotech down 37% Friday afternoon as media outlets reported FDA plans to request a stop to all shipments of the Duchenne muscular dystrophy therapy Elevidys following a third patient death linked to the underlying platform.
Despite the failure, BMS remains “encouraged” by Reblozyl’s clinical activity in myelofibrosis-associated anemia and will approach regulators to discuss potential submissions for this indication, for which few treatment options exist.
The panelists flagged safety concerns with Blenrep and GSK’s failure to optimize its dosing regimen for the antibody-drug conjugate in multiple myeloma.
The patient, who was being treated with an investigational gene therapy for limb-girdle muscular dystrophy, died of acute liver failure, the same complication responsible for the deaths of two boys taking Sarepta’s Duchenne muscular dystrophy treatment Elevidys.
In advance of CMS’ negotiated price for the blood thinner taking effect next year, partners Bristol Myers Squibb and Pfizer pitched the direct-to-consumer program as a way to allow uninsured, underinsured and self-pay patients to pay less out of pocket.
FEATURED STORIES
The weight-loss drug bonanza continued in the first quarter of 2024 for Novo Nordisk and Eli Lilly, as Amgen also posted strong results, while Biogen and BMS struggled early in the financial year.
Applications of the technology range from data collection to drug design to raising the alarm on product safety, but its adoption is also creating some anxiety.
Crackdowns on drug pricing have forced one major insulin out of the market. Are more to come?
While these technologies are now a therapeutic reality, the ASGCT 2024 annual meeting this week was a reminder of just how far we are from widespread use.
At a Thursday ASGCT 2024 session, CBER Director Peter Marks made the case for a better, “more convergent” global framework on cell and gene therapies, especially for rare diseases.
Launched in 2021, the public-private consortium on Wednesday updated ASGCT attendees on its efforts to bring adeno-associated virus gene therapies to more rare disease patients.
LATEST PODCASTS
In this episode presented by PII, BioSpace’s head of insights discusses how to relieve clinical trial patients of technological burden to improve compliance with guests Oliver Eden and Travis Webb.
Pfizer and Novo Nordisk continue to fight for ownership of obesity startup Metsera; CDER Director George Tidmarsh leaves his position amid an ongoing probe into his “personal conduct”; FDA reverses course on approval requirements for uniQure’s Huntington’s gene therapy; Sarepta’s exon-skipping Duchenne muscular dystrophy drugs fail confirmatory study.
In this episode presented by Element Materials Technology, BioSpace’s head of insights discusses how China, historically focused on manufacturing, is increasingly becoming an innovation leader, particularly in pharmaceuticals, with guests Dr. Jihye Jang-Lee and Dr. Khanh Courtney. Ultimately, balanced strategies involve domestic capacity investments coupled with global collaboration.
Job Trends
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from its Phase 3 program for the selective NaV1.8 inhibitor, VX-548, in the treatment of moderate-to-severe acute pain.
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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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There have already been several big biotech licensing deals in Q1. See inside for some of this quarter’s biggest licensing deals — from the surprising and pivotal to the lucrative and consequential. -
Jounce Therapeutics has received an unsolicited and non-binding acquisition proposal from Concentra Biosciences, looking to buy 100% of Jounce’s equity at a per-share price of $1.80 in cash. -
The SVB failure appears to have been caused by an underwritten public offering to raise $2.25 billion to cover security losses announced during SVB’s Q1 2023 mid-quarter update. -
Sanofi will acquire diabetes leader Provention Bio for $25 per share for a total of $2.9 billion, the companies announced Monday. -
Pfizer will acquire the Seattle-based antibody-drug conjugate leader in a $43 billion deal expected to close late in 2023 or early 2024.
WEIGHT LOSS
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Using electronic health records, healthcare analytics firm Truveta contends that Eli Lilly’s Mounjaro (tirzepatide) could achieve stronger and faster weight loss than Novo Nordisk’s Ozempic (semaglutide). -
To help cope with the high demand for weight-loss treatments, Eli Lilly is investing $2.5 billion in a German manufacturing facility after last week’s FDA approval of Zepbound for chronic weight management. -
Just a week after it secured FDA approval, Eli Lilly’s Zepbound now faces a challenge from Novo Nordisk’s investigational next-generation weight-loss candidate CagriSema in a Phase III trial. -
In this episode BioSpace’s Greg Slabodkin, Tyler Patchen and Lori Ellis discuss Zepbound, Wegovy, the weight loss race and the future of this drug class.
POLICY
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After back-to-back failures in 2021, Wave Life Sciences has finally aced a Phase Ib/IIa Huntington’s disease trial and is looking to a potential accelerated approval for its investigational antisense oligonucleotide. -
Scrutiny of WuXi Raises Potential Opportunities for Indian CDMOsAs congressional pressure increases on WuXi AppTec and other China-based companies over alleged ties to the Chinese government, India’s contract development and manufacturing organization sector could benefit. -
Citing issues with a third-party manufacturer, the FDA has issued another Complete Response Letter to AbbVie rejecting its New Drug Application for ABBV-951, a proposed treatment for motor fluctuations in adults with advanced Parkinson’s disease. -
If approved, ensifentrine would be the first non-steroidal, anti-inflammatory drug for patients with chronic obstructive pulmonary disease, offering an option with potentially fewer side effects. -
The FDA’s calendar is relatively light in July, with only five major deadlines, including one for a PD-1 blocker and another for an opioid overdose drug.
In this graceless age where people look at their phones when you are talking to them, only communicate via text or email, scroll head down on their phone while a speaker is presenting, post inappropriate pictures and commentary on Facebook, it’s no wonder many people do not send thank you notes.
Have you ever experienced a career epiphany but lacked the resolve to act on it? Don’t feel bad, especially if “acting on it” meant leaving a secure, albeit unfulfilling, job for a new career in a different industry.
When you are constantly hearing, “Thank you for your time, but we went with another candidate,” or you’re regularly submitting applications online, but not ever getting a response back, it can be frustrating.
The moment you step on the job market, you’re selling yourself. Yes, you, the job seeker, are a “brand,” and each touchpoint you have with a potential new employer is an opportunity to “sell” that brand, that vision of your candidacy, the promise of yourself as a future team member and employer.
Not only are interviewers tasked with evaluating whether or not you’re a good fit for their organization and the role, but you also should take every opportunity you can to evaluate if the position, company, and team is a good match for your own career goals and needs.
Career paths are not always perfectly linear and uninterrupted. Here are some ways you can display gaps in resumes that don’t appear like a weak point.
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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Bristol Myers Squibb’s $4.8 billion acquisition of Mirati pays off with strong data from the KRYSTAL-12 study of Krazati, showing that the KRAS inhibitor significantly improves progression-free survival. -
Keytruda can now be used in the European Union for patients with resectable non-small cell lung cancer at high risk of recurrence in combination with platinum chemotherapy, then continued as a monotherapy afterwards. -
Novocure’s stock spiked more than 15% Wednesday morning after the company announced its Tumor Treating Fields therapy met the primary endpoint in a late-stage trial. -
Regeneron’s bispecific antibody odronextamab was hit with Complete Response Letters from the FDA noting issues with the enrollment status of its confirmatory trials. -
AbbVie’s antibody-drug conjugate Elahere on Friday won the FDA’s full approval for the treatment of FRα-positive, platinum-resistant ovarian, fallopian tube and primary peritoneal cancers.
NEUROSCIENCE
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If Leqembi is granted priority review from the agency, full approval could come as soon as July, Ivan Cheung, chairman and CEO, Eisai Inc., told CNBC Thursday. -
Biogen’s 2022 fourth-quarter and full-year report comes the day after an additional warning on the company’s first Alzheimer’s drug came to light. -
The discovery of novel Alzheimer’s biomarkers is enabling better trial selection, earlier pipeline decision making and new targets for therapeutics. -
Phase I/II data showed AC Immune’s anti-amyloid-beta vaccine candidate could safely elicit an antibody response in patients with Alzheimer’s disease. Now, it will expand to Down Syndrome patients.
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Cassava Sciences reported Phase II data from its Alzheimer’s candidate simufilam Tuesday. The company’s stock dropped 17% following the reveal.
CELL AND GENE THERAPY
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The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred. Intellia is working with experts to create a risk management program for nex-z. -
Pfizer and Novo Nordisk continue to fight for ownership of obesity startup Metsera; CDER Director George Tidmarsh leaves his position amid an ongoing probe into his “personal conduct”; FDA reverses course on approval requirements for uniQure’s Huntington’s gene therapy; Sarepta’s exon-skipping Duchenne muscular dystrophy drugs fail confirmatory study. -
The potential approval of Vertex’s IgAN therapy povetacicept in 2026 comes amid launch headwinds for the company’s non-opioid pain medicine Journavx and gene therapy Casgevy. -
With a 100% response rate in a Phase II study, KYV-101 sets a new efficacy bar in generalized myasthenia gravis, according to analysts at William Blair. -
The clinical hold comes days after Intellia voluntarily paused enrollment and dosing in the same two studies.
