Praxis Precision Medicines has also announced a “successful” pre-NDA meeting with the FDA for its essential tremor drug candidate ulixacaltamide, for which an approval application is slated for early 2026.
The ACIP voted 8-3 to recommend delaying the hepatitis B vaccine, commonly given just after birth. The CDC itself has said the shot is safe and effective.
Investor optimism has waned as final minutes from uniQure’s pre-BLA meeting with the FDA convey that data from the company’s Phase I/II studies of AMT-130 are “unlikely” to provide the primary evidence to support a biologics license application.
Writing in separate editorials in two leading medical journals, former chiefs of federal scientific agencies issued warnings about the changes being proposed to vaccine frameworks by current officials.
The partnership will focus on Crescent’s PD-1/VEGF inhibitor CR-001 and Kelun-Biotech’s SKB105, both of which the companies plan to push into Phase I/II development for solid tumors early next year.
Companies who run their early-stage clinical development outside the U.S. would “experience higher fees” according to an FDA proposal made during the negotiation process for the eighth cycle of the user fee program.
FEATURED STORIES
The cholesterol-lowering drug is part of a suite of medicines that also includes MariTide and that Amgen Chief Medical Officer Paul Burton hopes will make the company the “undisputed leader in the management of cardiometabolic risk for patients” by 2030.
A new generation of companies is eyeing the existing, sizeable hair loss market, hoping that better formulations and new scientific targets will finally produce treatments that are safer and more effective, as well as commercially successful.
The nausea and other gastrointestinal side effects of weight loss drugs like Novo Nordisk’s Wegovy or Eli Lilly’s Zepbound will limit how much these drugs can help patients and stunt the overall obesity market unless we approach the problem head on.
While investment has slowed in radiopharmaceuticals, analysts predict increased interest to come as Novartis shows just how successful radiopharmaceuticals can be.
The upheaval of the Health and Human Services workforce and leadership leaves much to be desired in terms of delivery, recently retired FDA Chief Information Officer Vid Desai tells BioSpace, but the regulatory agency is evolving to be more open to much needed change.
Had Pfizer’s Freda Lewis-Hall not stepped in, SpringWorks’ rare disease treatment may never have reached patients. Pharmas can act now to help find the next Gomekli.
LATEST PODCASTS
This week’s release of the Make America Health Again report revealed continued emphasis on vaccine safety; Health Secretary Robert F. Kennedy Jr.’s faceoff with senators last week amounted to political theater; the FDA promises complete response letters in real time and shares details on a new rare disease framework; and Summit disappoints at the World Conference on Lung Cancer in Barcelona.
In this episode presented by Taconic Biosciences, BioSpace’s head of insights Lori Ellis discusses how preclinical research companies are helping drug developers navigate the current challenging funding environment with Mike Garrett, CEO.
Health Secretary Robert F. Kennedy Jr. will appear before the Senate Finance Committee Thursday, ahead of a vaccine advisory committee meeting later in September. Meanwhile, deal-making appetite appears healthy, and the weight loss space continues generating clinical data and other news.
Job Trends
The biopharma job market likely won’t turn around until 2026, according to two industry experts. Both cited a need for more investment and noted the impact of uncertainty on the industry.
Subscribe to GenePool
Subscribe to BioSpace’s flagship publication including top headlines, special editions and life sciences’ most important breaking news
SPECIAL EDITIONS
In this job market report we’re reviewing life sciences job market movement in Q3 and what to expect for Q4 and beyond.
In this special report, BioSpace examines how the biopharma industry is grappling with impending consequences of the Inflation Reduction Act.
DEALS
-
For $1.2 billion upfront and up to $10.2 billion in milestones, Takeda will gain access to a bispecific antibody fusion protein targeting both the PD-1 and IL-2 pathways, among other assets. -
Roche will gain worldwide rights outside of the Greater China region to Hansoh’s HS-20110, an antibody-drug conjugate in early-stage development for colorectal cancer. -
The partners have yet to disclose what their priority indications are, though EVOQ’s NanoDisc technology aims to enable the development of potentially curative treatments for autoimmune conditions such as celiac disease and type 1 diabetes. -
Novo Nordisk strikes another deal, this time with Omeros, amid a broader pipeline restructuring that recently claimed its cell therapy work. -
Bristol Myers Squibb and insitro first partnered in 2020 to develop induced pluripotent stem cell models of amyotrophic lateral sclerosis and frontotemporal dementia. Last December, BMS exercised its option for an ALS target.
WEIGHT LOSS
-
The reprioritization initiative extends Aldeyra’s cash runway into the second half of 2027. -
Viking Therapeutics CEO Brian Lian is watching the growth of interest in MASH and obesity but prepared to go it alone. -
Two patients experienced grade 3 liver enzyme elevations that were deemed related to Terns’ investigational obesity pill TERN-601. -
Rybelsus can now be used as a primary or secondary prevention pill to lower the risk of major adverse cardiovascular events in at-risk patients with type 2 diabetes. -
CMS Administrator Mehmet Oz clarified that a deal has not yet been sealed with the manufacturer of semaglutide, Novo Nordisk, or any other GLP-1 drugmaker.
POLICY
-
The FDA’s proposed Rare Disease Evidence Principles review process is a starting point for getting rare disease therapies across the finish line, but industry leaders say there are more concrete steps the regulator could take to help patients. -
After a tension-packed two days that saw recommended changes to the MMRV vaccine schedule and COVID-19 vaccine access, as well as a delayed hepatitis B vaccine vote, policy experts expressed concern with the reconstituted committee’s dearth of previous experience and understanding of their role. -
BMO Capital Markets analysts said the first day of the CDC vaccine advisory committee meeting Thursday had anti-vaccine overtones as the panel, which was revamped by Health Secretary Robert F. Kennedy, Jr. in June, voted to recommend that children under four receive the measles, mumps, rubella (MMR) vaccine separately from a chickenpox vaccine. Today the advisors will vote on changing the childhood schedule for the hepatitis B and COVID-19 vaccines. -
The House Committee on Energy and Commerce has cleared proposed legislation that could bring back the FDA’s rare pediatric priority review voucher program, which allows for expedited drug reviews. -
In this episode of Denatured, BioSpace’s head of insights Lori Ellis and Colin Zick, partner at Foley Hoag LLP, spend time discussing some of the points brought up in the Bioprocessing Summit last month. They explore the connections between hammers, AI, The Planet of the Apes and monoliths.
Carlos Doti has moved countries and changed jobs, but one thing hasn’t changed. Making an impact on patient care has always been a priority for the AstraZeneca executive.
Managers face a variety of challenges during their careers. Instead of falling into fight or flight, develop resilience to navigate the uncertain moments that pop up from time to time.
In our comprehensive guide to salary negotiation, we’ll teach you what a market salary is, how to research a market value salary and, ultimately, how to negotiate your salary according to market value.
During the job application and interview process, candidates who lie to prospective employers and those who don’t properly highlight their accomplishments can find it difficult to land—or keep—their next role. We asked experts how to sell yourself positively and honestly.
Dealing with a toxic co-worker can be exhausting, and it can make your workplace more stressful. There are a few ways to make it easier, including developing healthy coping mechanisms and even talking it out with your colleague.
Job security is a hot topic among biopharma professionals. A career coach offers advice for how to evaluate and build it up and what to do if that evaluation leaves you worried.
HOTBEDS
REPORTS
In the 2020 US Life Sciences Diversity & Inclusion report, BioSpace dives into how different segments of employees experience and perceive policies, attitudes and actions. Our data suggests that there are significant disparities between segments.
BioSpace surveyed our community to gain their insights and perspectives on work, their employers, and to understand who makes up the life science community.
How does being Black affect the workplace experience as a life sciences professional? BioSpace surveyed our community to gain a greater understanding of Black employees’ feelings of inclusion and their perspectives on employer DEI initiatives.
CANCER
-
The business separation, expected to be completed by the end of 2026, will result in two new companies, one focused on biopharma operations and the other on royalty management. -
The centerpiece of the acquisition is petosemtamab, Merus’ bispecific antibody targeting EGFR and LGR5, which in May demonstrated best-in-class potential for head-and-neck cancer. -
The FDA in September issued two rejections for spinal muscular atrophy therapies—both linked to manufacturing problems—and granted approvals in Barth syndrome and for a subcutaneous version of Merck’s Keytruda that could be key to the blockbuster’s future earnings. -
From more than 30 target action dates in the last three months of the year, BioSpace has narrowed the list to six regulatory decisions that could have far-reaching implications for biopharma and patients. -
The company was awaiting $70 million from HealthCare Royalty but missed an agreed-upon payment condition.
NEUROSCIENCE
-
Elecsys’ approval could help boost the uptake of currently approved Alzheimer’s disease therapies, including Biogen’s Eisai-partnered Leqembi, with CEO Chris Viehbacher recently noting that such biomarker-based tests could “remove some of the bottlenecks” in uptake. -
The centerpiece of the deal is orelabrutinib, a BTK inhibitor in late-stage development for multiple sclerosis that Biogen once paid $125 million for but abandoned after less than two years of testing. -
MapLight laid out the terms of its planned IPO in a regulatory filing on Monday, providing greater detail about what the funds will be used for. -
The hold was placed earlier this year when the FDA asked for more preclinical data, but the agency was slow to respond due to ‘strain’ on its capacity, according to Neurizon. -
While Bruton’s tyrosine kinase inhibitors are often hailed as the next big breakthrough in multiple sclerosis, Immunic Therapeutics and others are leveraging neuroprotective targets and remyelination to keep the disease at bay.
CELL AND GENE THERAPY
-
Rocket Pharmaceuticals’ strategic realignment initiative in July pulled funding from fanca-cel, which the biotech was developing for Fanconi anemia. -
The centerpiece of the collaboration is the gene editor ABO-101, being developed for primary hyperoxaluria type 1, a rare disease that leads to severe kidney stones. -
As industry leaders gather at the annual event in Phoenix, the cell and gene therapy space remains in a state of flux, with M&A activity and regulatory support signaling momentum while commercialization challenges continue to hinder broader investor interest. -
Smarter design through targeted delivery and human-relevant testing can save the industry from costly safety failures. -
Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways for cell and gene therapies ‘for small populations’ is receiving a warmer welcome—but experts warn it will take more to turn the tide for the fraught therapeutic space.
