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.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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Linda Marbán, Capricor CEO

Capri­cor hits pri­ma­ry end­point in Duchenne ex­ten­sion study, ready­ing its FDA pitch

It has been a very bumpy ride for Capricor Therapeutics over the past several years, including a halted J&J partnership and venture into penny stock status. However, the latest news from the San Diego-based biotech shows that its experimental Duchenne drug may be a durable, long-term treatment.

The company posted the one-year results from its open-label extension study for their candidate dubbed CAP-1002. The extension from its Phase II study, named HOPE-2, was conducted in boys and young men with later-stage Duchenne muscular dystrophy who paused and then resumed treatment after a year. Researchers measured patients in the one-year follow-up against the original study’s treatment arm effect and in two subsets of the original placebo group.

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Am­gen takes next step with its Chi­na am­bi­tions, out-li­cens­ing drugs to Fo­s­un Phar­ma

In a bid to increase its market share in China, Amgen has agreed to a partnership with a Shanghai biotech — a collaboration and out-licensing agreement for two of its drugs.

Amgen and Fosun Pharma announced a deal Monday in a bid to increase Amgen’s presence in the country. The stated goal so far is to commercialize Amgen’s blockbuster psoriasis drug Otezla alongside Parsabiv, a drug for secondary hyperparathyroidism in adults with chronic kidney disease and on a specific type of dialysis.