No­var­tis may still be grap­pling with Kym­ri­ah sales, but his­toric CAR-T promise still shines through 5-year da­ta

Five years af­ter No­var­tis made his­to­ry with the ap­proval of the first CAR-T, Kym­ri­ah, we’re get­ting a clear­er pic­ture of just how im­por­tant the ad­vance was for some pa­tients.

The phar­ma gi­ant may still be hav­ing trou­ble man­u­fac­tur­ing the treat­ment or reach­ing long-cher­ished goals of block­buster rev­enue, but a new study shows that its pos­i­tive ef­fects have good odds of ex­tend­ing out for years. And for many, it’s look­ing cu­ra­tive.

The five-year re­lapse-free sur­vival rate was 44%, says No­var­tis, and the me­di­an RFS was 43 months. “The me­di­an event-free sur­vival (EFS) for pa­tients in re­mis­sion with­in three months of in­fu­sion (n=65) was 43.8 months.”

“These da­ta mark a mo­ment of pro­found hope for chil­dren, young adults and their fam­i­lies with re­lapsed or re­frac­to­ry B-cell ALL, as re­lapse af­ter five years is rare,” said Stephan Grupp, the in­au­gur­al di­rec­tor of the Su­san S and Stephen Kel­ly Cen­ter for Can­cer Im­munother­a­py at Chil­dren’s Hos­pi­tal of Philadel­phia. “Since the ap­proval of Kym­ri­ah near­ly five years ago, we have been able to of­fer a tru­ly game-chang­ing op­tion to pa­tients who pre­vi­ous­ly faced a five-year sur­vival rate of less than 10 per­cent.”

These con­tin­u­ing re­sults along­side the suc­cess of Yescar­ta at Kite un­der­score the orig­i­nal hope that arm­ing pa­tient cells for an at­tack on blood can­cers of­fered a break­through ap­proach that would save scores of lives. It’s al­so a re­minder that the im­me­di­ate goal of cre­at­ing a sec­ond-gen, off-the-shelf ap­proach hasn’t been easy, though progress is be­ing made.

Kym­ri­ah earned $587 mil­lion last year, a 24% gain. The rev­enue steadi­ly drags be­hind the ri­val Yescar­ta, from which Gilead earned $695 mil­lion last year.

The drug has had its ups and downs in the clin­ic as well, with a fail last year in a Phase III for ag­gres­sive B-cell non-Hodgkin lym­phoma, cost­ing a con­sid­er­able amount in the still hoped-for block­buster sales to come. But it al­so just won ap­proval as a third-line treat­ment for fol­lic­u­lar lym­phoma.

This al­so isn’t the first time that No­var­tis has re­port­ed stel­lar five-year da­ta. Last year the phar­ma gi­ant an­nounced that in a group of ad­vanced, treat­ment-re­sis­tant NHL pa­tients, 46% had achieved a com­plete re­mis­sion, with 31% mark­ing pro­gres­sion-free sur­vival at the five-year point. And re­searchers de­ter­mined that durable suc­cess here hinges on pa­tients’ re­sponse one year in­to the treat­ment.

Cor­rec­tion: No­var­tis with­drew and cor­rect­ed its ini­tial re­lease out­lin­ing sur­vival da­ta, chang­ing a va­ri­ety of the fig­ures.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Cedric Ververken, Confo Therapeutics CEO

Dai­ichi Sankyo inks $183M dis­cov­ery deal with GPCR biotech for CNS tar­get

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

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