BMO Capital Markets analysts said the results potentially position lepodisiran as “one of the most durable assets in development to date” in the competitive Lp(a) space, where drugs are designed to lower the risk of cardiovascular events such as heart attack and stroke.
Ayrmid’s offer is 50% higher than bluebird’s previously announced deal with Carlyle and SK Capital Partners.
According to BMO Capital Markets, Rybelsus’ outcomes in SOUL were “inconsistent,” failing to significantly lower cardiovascular death and nonfatal stroke.
Werner held roles at Bristol Myers Squibb, AstraZeneca and Novartis before landing at Alltrna, where she works to develop tRNA-based treatments for a range of diseases.
In the coming two weeks, the FDA is expected to announce three big decisions, including one for a dry eye disease therapy.
The European Union’s CHMP said that the benefits of the drug, already approved in the U.S., do not outweigh the risk of potentially fatal brain swelling and bleeding.
FEATURED STORIES
Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic for cardiovascular diseases including congestive heart failure, chronic refractory angina and cardiomyopathy.
Among the 55 novel drugs that crossed the regulatory finish line last year were notable new mechanisms of action, coming particularly in the oncology and neurosciences spaces.
An FDA committee’s September 2024 vote to limit the use of Merck’s Keytruda and BMS’ Opdivo in stomach and esophageal cancers based on PD-L1 expression levels reflects an emerging trend that leverages ever-maturing datasets.
FROM BIOSPACE INSIGHTS
In a year when eradicated diseases are on the uptick in America, how will American children survive RFK Jr.’s vaccine scrutiny and inconsistency? Two experts call on pharma and regulatory bodies to rebuild trust.
LATEST PODCASTS
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.
This week, in (our inaugural episode!) BioSpace’s Greg Slabodkin, Tyler Patchen and Lori Ellis discuss the good, the bad and the ugly of biopharma’s reported Q3 earnings. They also tackle what’s going on at Pfizer, ADCs, deals and more.
Job Trends
Pfizer Highlights Progress in Accelerating Breakthrough Cancer Medicines at ASCO 2024 Annual Meeting
Pfizer Inc. highlights its progress in advancing new potential standards of care in Oncology at the 2024 American Society of Clinical Oncology Annual Meeting, taking place May 31 to June 4 in Chicago.
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SPECIAL EDITIONS
A new generation of checkpoint inhibitors is emerging, with some showing more promise than others. From recent TIGIT failures to high-potential targets like VEGF, BioSpace explores what’s on the horizon in immuno-oncology.
Peter Marks, the venerable head of the FDA’s Center for Biologics Evaluation and Research, has been forced out. In this special edition of BioPharm Executive, BioSpace takes a deep dive into the instability of the HHS.
Year-over-year BioSpace data show biopharma professionals faced increased competition for fewer employment opportunities during the first quarter of 2025.
DEALS
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On the heels of ArriVent and CG Oncology’s upsized IPOs last week, Alto Neuroscience and Fractyl Health on Monday announced their respective plans to go public. -
Late Thursday, ArriVent Biopharma announced its upsized initial public offering to support the development of its EGFR inhibitor furmonertinib, following CG Oncology going public earlier in the day.
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The bladder cancer-focused company’s larger-than-expected Nasdaq debut on Thursday opens this year’s batch of planned initial public offerings, which include at least half a dozen biotechs. -
Most respondents expressed concerns about the economic environment, as competition for jobs jumped more than 130%. -
This week, Lori, Greg and Tyler discuss the first surge of IPO activity this year plus gene therapy pricing,
WEIGHT LOSS
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The plethora of genes involved in obesity presents an intriguing opportunity for both gene silencing and ex vivo gene therapy approaches. -
Building on last year’s potential $7 billion partnership with Flagship Pioneering, Pfizer and Flagship-founded ProFound Therapeutics will work on discovering new obesity candidates. -
Seeking to target the lucrative obesity market, Merck is focusing its R&D efforts on next-generation GLP-1 therapies that offer additional benefits beyond weight loss, according to company executives. -
Sen. Bernie Sanders (I-Vt.), chair of the Senate health committee, has proposed issuing a subpoena to Novo Nordisk President Doug Langa forcing him to testify regarding the company’s pricing for Wegovy and Ozempic. -
Seeking to carve out its own niche in the obesity space, Syntis Bio launched on Tuesday to develop an oral weight-loss treatment that mimics the effects of gastric bypass surgery.
POLICY
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The combination therapy is one step closer to becoming a potential new first-line standard of care for patients with unresectable or metastatic urothelial carcinoma in Europe. -
Despite their initial kicking and screaming, drugmakers seem confident the Inflation Reduction Act’s Medicare Drug Price Negotiation Program will not greatly impact their bottom lines. -
Executives from the three largest pharmacy benefit manager companies testified Tuesday before Congress that rising drug prices in the U.S. are due to pharma companies taking advantage of market exclusivities and excessive charges. -
Allarity Therapeutics announced Monday that the Securities and Exchange Commission has made a preliminary determination recommending an enforcement action against the company for alleged violations of federal securities laws. -
The Department of Health and Human Services’ Office of the Inspector General found that bluebird bio’s fertility support program for its gene therapies could potentially violate federal anti-kickback statutes.
What are the benefits of becoming a remote worker? Let’s explore together.
If you’re considering preparing for your next interview, know that you need to learn the art of drafting a perfect cover letter to show that you’re a great match for the position.
BioSpace recently interviewed Elizabeth Elting, founder and CEO of the Elizabeth Elting Foundation, to discuss her thoughts on how the pandemic has affected women in the workforce.
A well-designed career development program provides the right guidance and training for workers to improve their professional careers.
Do you know that growing emotional intelligence at workplace can be beneficial for career growth? If not, have a look at the article and learn some tips.
Controlling interruptions when working from home can be challenging, but a little planning and some established rules can help.
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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In June, the regulator placed a partial clinical hold on a Phase I trial of the companies’ antibody-drug conjugate after three patient deaths were reported. -
The unit’s closure comes as Genentech’s parent Roche rethinks its cancer business, an effort that has included the discontinuation of three early-stage candidates and a T-cell partnership with Adaptimmune. -
Imfinzi’s perioperative approval comes after both the FDA’s staffers and a panel of external experts expressed concerns about overtreatment when using the PD-L1 blocker both before and after surgery. -
The Biden administration on Thursday touted discounts of up to 79%, but many of these first 10 drugs are already sold well below list price.
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The European Union has approved the first-ever combination therapy consisting of an immunotherapy and a PARP inhibitor for the treatment of endometrial cancer, AstraZeneca announced Wednesday.
NEUROSCIENCE
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Takeda on Monday said it is paying AC Immune $100 million upfront for an option on a Phase Ib/II Alzheimer’s disease candidate that could activate the immune system to clear amyloid beta plaques. -
With its $525 million investment, Royalty Pharma will acquire the royalties and milestones for ImmuNext’s anti-CD40 therapy frexalimab, which is currently in Phase III trials for multiple sclerosis. -
While Teva missed Wall Street expectations in the first quarter of 2024, it reported Wednesday a 5% increase in Q1 revenues while reporting that the company’s schizophrenia candidate scored a late-stage trial victory -
Amylyx’s recent decision to withdraw its ALS drug Relyvrio from the market highlights an important business decision for companies: when to continue marketing or investigating a drug that has failed a pivotal or confirmatory study. -
Neurocrine Biosciences on Tuesday won the FDA’s approval for a sprinkle capsule formulation of Ingrezza, providing a more convenient route of treatment for Huntington’s disease patients with trouble swallowing.
CELL AND GENE THERAPY
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Asgard Therapeutics, a Swedish gene therapy biotech, has closed a $32 million Series A round with help from prominent pharma players as it prepares for a 2026 IND. -
The FDA approved Bristol Myers Squibb’s Breyanzi for chronic lymphocytic leukemia and small lymphocytic leukemia prior to Friday’s adcomm for the company’s other CAR-T therapy, Abecma. -
With an advisory committee meeting slated for Friday, the regulator has posted briefing documents in which it has raised concerns about early deaths in patients treated with Bristol Myers Squibb’s Abecma and Johnson & Johnson’s Carvykti. -
Allogene Therapeutics and Arbor Biotechnologies will use their allogeneic CAR T and next-generation gene-editing platforms to develop novel off-the-shelf CAR-T therapies for autoimmune diseases. -
Vertex and CRISPR Therapeutics are setting up treatment centers for patients with beta thalassemia and sickle cell disease to compete with bluebird’s established infrastructure.
