Pearl Freier
@PearlF
Founder of Cambridge BioPartners, Inc.
New Journal of Clinical Oncology @JCO_ASCO paper estimated potential savings of $661.8M for US taxpayer-funded Medicare if replace current Medicare Part D median formulary prices w/ @costplusdrugs pricing for 7 generic oncology drugs. ascopubs.org/doi/full/10.12… h/t @RuchikaTalwarMD
you could put together a pretty great newspaper with all the talented people leaving the Washington Post
Some professional news: After 11 years of columnizing at WaPo, I'm taking the buyout. This is my last column. It is my advice to any other lucky pundits who land a perch like this—11 principles I've aspired to, even if I haven't always achieved them: wapo.st/3TU2fGw
A big first for SMM! EU clears J&J's Darzalex as first therapy for smoldering multiple myeloma. Congrats to the entire #myeloma community who fought hard for this to happen #mmsm. firstwordpharma.com/story/5983164
Ness Birmingham said that clear regulatory rules at FDA for personalized therapeutics would "unlock investment, while reimbursement frameworks will ensure payers can cover these treatments responsibly. Absent this, safe and effective therapies may never reach patients."
In order to turn personalized therapies for rare diseases (like baby KJ) into a new standard of care, Birmingham said FDA should establish a dedicated oversight group within FDA to review personalized therapeutics, set transparent safety and ethics standards & ensure rigorous…
In order to turn personalized therapies for rare diseases (like baby KJ) into a new standard of care, Birmingham said FDA should establish a dedicated oversight group within FDA to review personalized therapeutics, set transparent safety and ethics standards & ensure rigorous…
Former Intellia Tx CEO now operating partner at @khoslaventures Ness Birmingham published an op-ed in @thehill calling for the FDA to have a dedicated framework that clarifies the rules, requirements & incentives including a new pathway for bespoke therapeutics & n-of-1 drugs
JD Vance came on stage this afternoon at Winning the AI Race and framed Trump's immigration crackdown as good for American workers, who could work in new American AI factories.
A landmark study published in @Nature by @aaronmring et al. has revealed that autoantibodies — immune proteins traditionally associated with autoimmune disease — may profoundly influence how cancer patients respond to immunotherapy. bit.ly/4o4HANS
Information below on *no-cost* genetic testing for patients with suspected genetic cardiomyopathy:
In collaboration with @BroadGenomics & @MassGenBrigham Lab for Molecular Medicine, @everygenepbc (everygene.com) is now offering nationwide *no-cost* genetic testing for patients with suspected genetic cardiomyopathy prnewswire.com/news-releases/… @broadinstitute…
CNN's @owermohle has a new report out today detailing how FDA's new Elsa AI tool is quite unrealiable, undercutting claims about how it will be used to make FDA more efficient: cnn.com/2025/07/23/pol…
It's also ironic though that the new FDA appeared to move quickly to approve Juul's e-cigarettes this month as part of the Make America Healthy Again movement. $XBI
Shifting FDA standards are making drug approvals less predictable wrote @steveusdin1 of @BioCentury. Less predictability for patients too. The list of examples is getting longer by the day, more below: $XBI
Shifting FDA standards are making drug approvals less predictable wrote @steveusdin1 of @BioCentury. Less predictability for patients too. The list of examples is getting longer by the day, more below: $XBI
Shifting FDA standards are making approvals less predictable. Replimune, surprised by a CRL Tuesday, joins 3 cos disrupted since May - Ultragenynx, Caprior, Immunitybio . Can firms still rely on pre-January FDA guidance? What are the new standards? biocentury.com/article/656558
It's unusual for the FDA to be behind the European Commission on a drug approval like this & prev a patient population w/ltd options. Were too many people laid off at the FDA or are there other problems? $XBI ODAC gave a positive vote in favor of approval h/t @VincentRK & NCCN…
The European Commission was able to make the below drug regulatory decision before the FDA does & cancer drug Darzalex (Johnson & Johnson) is now the 1st ever approved treatment for high risk smoldering multiple myeloma (SMM)—based on Phase 3 AQUILA trial. More below $JNJ #mmsm
“Until now, the absence of approved therapies for high-risk smouldering multiple myeloma has left clinicians with limited options beyond observation, despite evidence that 50 percent of this patient population progress to active multiple myeloma within two years,” said…
A historic day for myeloma!! After 5 decades of research. European Commission / EMA approves Daratumumab as the first ever approved treatment for high risk smoldering myeloma based on the results of the AQUILA trial. @thanosdimop @PlasmaCellPete globenewswire.com/news-release/2…
"and the obvious major caveat is if upon review, the fda review team found things in the data they didn't like. this would be totally understandable... although still uncommon given breakthrough status presumes some reasonable level of review of the data." $XBI
on the $repl crl. the issue with the rejection is that investors believe that the company had alignment with fda on its filing plan, supported by receiving breakthru status last november. the issues called out by fda (single arm, population, contribution of components) are all…
"as fda wants to set new expectations, honoring guidance given in the past is really important to make high unmet need areas investible given long execution timelines." $XBI
on the $repl crl. the issue with the rejection is that investors believe that the company had alignment with fda on its filing plan, supported by receiving breakthru status last november. the issues called out by fda (single arm, population, contribution of components) are all…
"investors will default to areas with low uncertainty as fda staff feedback on edge cases is considered unreliable - or everyone will want to hear it 'directly from the top' which obviously isn’t feasible."
on the $repl crl. the issue with the rejection is that investors believe that the company had alignment with fda on its filing plan, supported by receiving breakthru status last november. the issues called out by fda (single arm, population, contribution of components) are all…
Headed back to Boston this morning from a short visit to NY and I have great stuff coming up over on @BiotechTV. Today I’ll be visiting Replimune, and tomorrow Abivax and Monte Rosa. Please let me know any good questions to ask. $REPL $ABVX $GLUE