FDA puts Vertex's new Type 1 diabetes cell therapy IND on hold … – Endpoints News

Briefly men­tioned in Ver­tex Phar­ma­ceu­ti­cals’ JPM pre­sen­ta­tion Mon­day, the FDA has put an IND for Ver­tex’s new Type 1 di­a­betes cell ther­a­py, VX-264, on hold. CEO Resh­ma Ke­wal­ra­mani not­ed the com­pa­ny has yet to re­ceive the FDA’s ques­tions on the ex­per­i­men­tal treat­ment.
VX-264 us­es the same islet cells as Ver­tex’s T1D cell ther­a­py VX-880, which is al­ready in the clin­ic. In June, Ver­tex pre­sent­ed da­ta on two pa­tients in the VX-880 tri­al — the first pa­tient be­came in­sulin-in­de­pen­dent at around 9 months af­ter treat­ment, while the sec­ond saw a 30% re­duc­tion in in­sulin use at around 5 months.
How­ev­er, for VX-264, the cells are put in a de­vice that cloaks them from the im­mune sys­tem, Ke­wal­ra­mani said, so that pa­tients do not need to take im­muno­sup­pres­sives along­side the treat­ment. She not­ed that VX-264 has been cleared for clin­i­cal tri­als in Cana­da.
She al­so said yes­ter­day that Ver­tex has filed exa-cel, its sick­le cell and be­ta tha­lassemia treat­ment, in the EU and UK, and plans to fin­ish the US sub­mis­sion this quar­ter. — Lei Lei Wu
In­sil­i­co Med­i­cine an­nounced Tues­day morn­ing that it is plan­ning to start a Phase II study of its AI-de­signed and dis­cov­ered drug for id­io­path­ic pul­monary fi­bro­sis (IPF) “in ear­ly 2023.”
IPF is a chron­ic dis­ease in which scar­ring on the lungs makes it hard­er to breathe. In­sil­i­co is wrap­ping up a Phase I study of the drug in healthy vol­un­teers in New Zealand, and will test the drug in its in­tend­ed pa­tient pop­u­la­tion in the next phase.
In the niche of AI drugs for IPF, As­traZeneca is al­so in the run­ning. It’s part­nered with Benev­o­len­tAI for three tar­gets in the dis­ease. The duo has been work­ing to­geth­er since 2019 and signed a sec­ond deal in 2022. — Lei Lei Wu
Pri­vate­ly-held Re­Code Ther­a­peu­tics is re­ceiv­ing a $10 mil­lion eq­ui­ty in­vest­ment from the Cys­tic Fi­bro­sis Foun­da­tion for its work on an mR­NA drug for CF.
The Cal­i­for­nia ge­net­ic med­i­cines biotech will use the cash, which could grow by $5 mil­lion, to take the in­haled mR­NA-based can­di­date through pre­clin­i­cal stud­ies and ear­ly in-hu­man tri­als, Re­Code said Tues­day. Re­Code is us­ing a so-called se­lec­tive or­gan-tar­get­ing lipid nanopar­ti­cle to de­liv­er the CFTR mR­NA, which aims to tell the body to pro­duce a healthy ver­sion of the CFTR pro­tein.
“In ad­di­tion to the fund­ing, our team will have fur­ther ac­cess to the CF Foun­da­tion’s world-renowned re­searchers and lab fa­cil­i­ties to ac­cel­er­ate our de­vel­op­ment of a nov­el mR­NA treat­ment for CF pa­tients not el­i­gi­ble for cur­rent treat­ments,” Re­Code CEO Shehnaaz Suli­man said in a state­ment.
The news fol­lows Re­Code’s Mon­day an­nounce­ment that it is work­ing with Bay­er’s AskBio on liv­er and lung dis­ease tar­gets. — Kyle LaHu­cik
Transforming the US healthcare system will be complex, but an emerging class of tech-enabled providers are poised to support a breakthrough. Learn why the move to value-based care offers a potentially huge market opportunity, despite being in its early stages.
The unsustainable nature of US healthcare spending has long been a widely accepted fact, and expenditures touched 19.7% of GDP in 2020. The country now spends an estimated $12,318 per person on healthcare annually – far above Germany, the next biggest spender at $7,382 per head.
SAN FRANCISCO — After two years of Covid-stalled virtual dealmaking, the JP Morgan Healthcare Conference returned as an in-person event on Monday, with plenty of excitement in the air.
Attendees crammed into the Westin St. Francis, clogging the hallways with few masks on faces, to listen to pharma and biotech execs offer their latest positive news and expectations for 2023. Bristol Myers Squibb and Biogen drew standing room only crowds but offered few new updates. Protesters chanted briefly outside the hotel accusing Gilead of price-gouging their HIV drugs and left by lunchtime. The line for free lunch stretched hundreds of people long, wrapping around the conference rooms.
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SAN FRANCISCO — A new study for the Huntington’s disease drug tominersen, developed in tandem by Roche and Ionis, is expected to start imminently, Endpoints News has learned. The Phase II trial will examine whether tominersen can slow the progression of Huntington’s in patients aged 25 to 50 with very early or subtle signs of the disease.
Roche previously stopped a tominersen study in Huntington’s early in March 2021, in which the drug appeared to make later-stage patients worse off than those who took placebo. But Roche and Ionis began planning a new study last January after a post-hoc analysis identified a possible efficacy signal in a younger population.
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While bluebird bio is expected to file an application with the FDA for its sickle cell disease (SCD) drug candidate in the first quarter, it’s already raising awareness online. Two websites — one for healthcare providers called “Change for SCD” and another for patients and caregivers called “Spark Sickle Cell Change” — feature real physicians and patients talking about sickle cell disease stigma and other issues.
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Arrowhead Pharmaceuticals reported Monday that its Takeda-partnered program reduced liver scarring and mutant protein levels in patients with alpha-1 antitrypsin deficiency, a rare liver disease. However, a better-than-expected placebo performance sent shares of Arrowhead $ARWR down more than 20%.
The Phase II SEQUOIA study showed the pair’s RNAi drug candidate fazirsiran reduced markers of AATD, a condition in which the body does not produce enough AAT, a protein made in the liver to protect the lungs.
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One of the early gene editing hopefuls, Editas Medicine, has struggled to keep its ship on the right path. The biotech’s latest woes include pipeline cuts, layoffs and the CSO’s exit.
The Boston-area biotech is letting go 20% of its workforce, getting rid of science chief Mark Shearman at the end of March and retuning the clinical focus. The search for a new CSO is already underway, the biotech said Monday morning.
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Kronos Bio and Roche’s Genentech announced a drug discovery deal to hunt for and develop small molecule drugs aimed at transcription factors that can drive the proliferation of cancer.
Under the two-program agreement, Kronos will get $20 million upfront, plus as much as $554 million in developmental and commercial milestone payments, in addition to potential single-digit sales royalties.
Kronos launched in 2018 with CEO Norbert Bischofberger, the longtime leader of Gilead’s R&D efforts, at the helm. He took the company public in 2020, though the shares have struggled since the IPO. Late last year, the company pulled the plug on a Phase III trial of its acute myeloid leukemia drug entospletinib, pivoting instead to an earlier-stage compound.
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SAN FRANCISCO — A Bay Area startup out to create a next-gen player in TCRs has just hit a key milestone following its recent debut, inking its first big alliance with a big-name pharma while also bringing in some added insights from clinical operations that can help shape their work.
Initially ramped up in stealth back in 2017 with early backing from Sean Parker and the Parker Institute for Cancer Immunotherapy, 3T Biosciences announced a $40 million A round a few months ago. Now, CEO Stefan Scherer has forged a partnership with Boehringer Ingelheim, a large German pharma that remains private.
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Doctors who treat Alzheimer’s patients say they’re likely to prescribe Eisai and Biogen’s new therapy approved Friday, but sounded notes of caution over the lack of insurance coverage for the drug and a number of worrying side effects, including several reports of patient deaths.
The drug, which was known as lecanemab and will be sold under the brand name Leqembi, reduced the rate of cognitive decline by 27% in a study of nearly 1,800 people with early-stage Alzheimer’s, offering new hope after decades of failed clinical trials and controversy surrounding the approval of Aduhelm in 2021. The results, doctors stated, make the drug an option for patients like those in the trial, with mild cognitive impairment and elevated levels of amyloid, a protein in the brain associated with Alzheimer’s.
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