The drug, for hereditary angioedema, is Ionis’ second wholly owned asset.
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.
FEATURED STORIES
Not exactly known for its dealmaking, Sarepta Therapeutics has thrown down a massive wad of cash to work with Arrowhead Pharmaceuticals on RNAi-based medicines.
Intra-Cellular submitted its application to the FDA for Caplyta’s approval in major depressive disorder, potentially opening up an additional $1 billion in sales. Still, the stock remains “cheap,” according to Jefferies analysts.
Despite hotly debated biomarkers and failed or delayed confirmatory trials, the accelerated approval program has a track record of propelling R&D for some of medicine’s most challenging illnesses.
Emboldened by technological advances and a deeper knowledge of glioblastoma, Merck, Kazia Therapeutics, CorriXR Therapeutics and others are targeting the often-fatal brain tumor.
Projected to be worth over $38 billion in the global healthcare market by 2032, AI simulations have the potential to streamline clinical trials and help address inequities in underserved patient populations.
Eli Lilly topped the list of the 20 biggest pharmas by market cap with a more than 39% improvement year-to-date in its share price. Other companies have not been so lucky.
LATEST PODCASTS
How can startup leaders support long-term sustainable growth and investment? BioSpace’s Lori Ellis speaks with venture capital guests Ansbert Gadicke, Martin Gershon and Mike Goguen for their advice and recommendations on how biopharma startups should approach funding.
What needs to happen for funding in biopharma to bounce back? BioSpace’s Lori Ellis discusses the macroeconomic environment and biopharma funding outlook with venture capital guests Ansbert Gadicke, Martin Gershon and Mike Goguen.
This week, we discuss the two major FDA approvals for sickle cell from Vertex/CRISPR and bluebird bio; Axcella and the future of long-covid treatments, Vanda’s $100m purchase and AI regulatory developments in Europe.
Job Trends
Sage Therapeutics, Inc., a biopharmaceutical company leading the way to create a world with better brain health, announced that it will host a live webcast on Thursday, April 25, 2024 at 8:00 a.m. ET to review first quarter 2024 financial results and discuss recent business updates.
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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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Just weeks after Wegovy won FDA approval for cardiovascular disease, Novo Nordisk has bought mid-stage biotech Cardior Pharmaceuticals and its miRNA-targeting candidate for heart failure. -
The contract manufacturer plans to expand its U.S. footprint with a former Roche facility in Vacaville, California, which Lonza contends is one of the largest biologics manufacturing facilities in the world by volume. -
Following in the footsteps of Bristol Myers Squibb and Eli Lilly, AstraZeneca on Tuesday jumped into the radiopharmaceuticals space by acquiring Fusion Pharmaceuticals in a deal worth $2.4 billion. -
Contineum Therapeutics joined the 2024 initial public offering class on Friday with an SEC filing. The biotech will use the IPO proceeds to complete a Phase II trial for its most mature candidate targeting multiple sclerosis. -
At the center of the deal is Amolyt Pharma’s late-stage candidate eneboparatide for the rare disease hypoparathyroidism. AstraZeneca also gains ownership of AZP-3813, which is being assessed for acromegaly in a Phase I trial.
WEIGHT LOSS
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This week, Q2 earnings from Novo Nordisk and Eli Lilly revealed that the competition between the pharma giants’ weight-loss drugs Wegovy and Zepbound is getting closer. -
Eli Lilly comfortably cleared analyst estimates in the second quarter after improving supply of its blockbuster tirzepatide brands Mounjaro and Zepbound, which together generated more than $4 billion in sales. -
Novo Nordisk’s semaglutide brands Ozempic and Wegovy fell short of analyst expectations in the second quarter, mainly held back by supply headwinds. The company’s shares dropped more than 7% in Wednesday morning trading. -
A day after Eli Lilly’s obesity and weight-loss therapies were removed from the regulator’s database, Novo Nordisk also made strides in boosting the supply of all but one of semaglutide’s shortages. -
The company is projecting that future growth will be driven by geographic and label expansions for its rare disease assets, as well as potential approvals in obesity.
POLICY
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Kennedy’s confirmation hearing on Wednesday became heated as Democratic senators grilled the nominee for HHS Secretary on his previous statements about vaccine safety. -
The U.S. Centers for Medicare and Medicaid Services has named Novo Nordisk’s Wegovy, Ozempic and Rybelsus as part of the second round of the IRA drug price negotiation program, even as the pharma challenges the program. -
Donald Trump continues to make waves in biopharma; Sage rejects Biogen’s unsolicited takeover offer; the obesity space sees more action with new company launches, IPOs and fresh data; and experts get ready for an important era in the Duchenne muscular dystrophy space. -
Ahead of Robert F. Kennedy Jr.’s confirmation hearings, experts—and RFK’s own family—expressed concerns about his vaccine-related views, though the same experts are largely unfazed by the level of power he and Marty Makary could ultimately wield over the FDA. -
From March 2020 through September 2022, Biohaven wielded meals at high-end restaurants and paid speaking opportunities to induce healthcare providers to prescribe its migraine therapy Nurtec ODT, according to the Department of Justice.
Keep these tips for virtual meetings in mind the next time you’re getting ready to log on to a video conference and want to keep your attention span in check.
You can use a time management tool In order to exceed your office productivity while working from home. Here’s one such tool that you can use and boost performance.
BioSpace interviewed Dr. Heike Blockus, an associate research scientist at Columbia University. Blockus shared her thoughts on the neuroscience career path.
Now that a majority of us are working remotely, and it may stay like that for the foreseeable future, one thing we’ve left behind is the commute to the office.
Writing a interview follow up email that expresses your enthusiasm and competence for the available role can help you stand out. Let’s check how to write one.
Whether you’re an experienced professional in the field or just embarking on your new career, it’s time to look at your qualifications and career plans from an entirely different perspective.
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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Bank of America analysts said prior to Thursday’s readout that Tyra Biosciences’ TYRA-300 could rival Johnson & Johnson’s kinase inhibitor Balversa, which has suffered from safety concerns and poor tolerability. -
The regulator’s approval on Friday of Vyloy for gastric or gastroesophageal junction cancer makes it the first and only claudin 18.2–targeted therapy approved in the U.S. for these indications, according to Astellas. -
Pharmaceutical companies are combining antibodies with radioisotopes in a bid to more precisely deliver radiation to cancers and tumors. -
The move is a blow to Gilead’s cancer portfolio. Trodelvy, an antibody-drug conjugate granted accelerated approval for bladder cancer in 2021, failed its confirmatory trial earlier this year. -
The regulator has delayed its respective decision dates on whether to grant full approval to Amgen’s Lumakras in metastatic colorectal cancer and Intercept Pharmaceuticals’ Ocaliva for primary biliary cholangitis.
NEUROSCIENCE
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Sangamo Therapeutics announced Tuesday it secured an exclusive licensing agreement with Roche’s Genentech, which is paying $50 million in near-term upfront fees and milestone payments to develop novel genomic medicines for neurodegenerative diseases. -
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. -
Eisai presented a plethora of data on the drug at the Alzheimer’s Association International Conference, including a study showing the consequences of pausing treatment. -
Second-quarter earnings season continues with Big Pharma beating Wall Street expectations, the author of an encrypted email sent to BioSpace has a proposal for Moderna and Merck, Roche and Viking seek quicker entry to the obesity market, and AAIC is in full swing.
CELL AND GENE THERAPY
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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. -
With 15 patients started across its three gene therapies, bluebird bio claims a 138% year-over-year revenue growth and aims to initiate up to around 100 new patients in the current year. -
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. -
An autologous and personalized regulatory T cell therapy is safe in patients with type 1 diabetes, but does not help preserve β-cell function. -
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.
