Re­gen­eron’s star bis­pe­cif­ic is linked to 2 deaths in a small study — which was no help for its Q1 call

One of Re­gen­eron’s star can­cer drugs in the pipeline has run in­to trou­ble, and that’s con­tribut­ing to some tur­bu­lence for the share price on Tues­day.

Is­rael Lowy

Re­gen­eron ex­ecs chose to dis­close in their Q1 call with an­a­lysts to­day that their close­ly watched bis­pe­cif­ic REGN1979 was linked with two deaths from cy­tokine re­lease syn­drome in a small PD-1 com­bo study in­volv­ing 30 pa­tients. 

This bis­pe­cif­ic hunts the B cell mark­er CD20 as well as the CD3 com­po­nent of the T cell re­cep­tor, and Re­gen­eron can­cer re­search chief Is­rael Lowy spot­light­ed a 100% over­all re­sponse rate as a monother­a­py at ASH late last year, with 8 com­plete re­spons­es and 2 par­tials among pa­tients with fol­lic­u­lar lym­phoma. That’s very ear­ly-stage da­ta from a tiny tri­al, but Phase I is the new Phase II in can­cer re­search, and those kinds of da­ta al­ways at­tract at­ten­tion in the field — par­tic­u­lar­ly when it comes from a high-pro­file out­fit like Re­gen­eron.

Ge­of­frey Porges

SVB Leerink’s Ge­of­frey Porges was on the call, and re­port­ed back:

The tri­al had on­ly en­rolled 30 pa­tients. Re­gen­eron sug­gest­ed the high­er CRS tox­i­c­i­ty was like­ly due to a height­ened im­mune re­sponse, and not­ed the CRS was al­so seen in con­junc­tion with in­creased tu­mor re­sponse. While the tox­i­c­i­ty is a pre­lim­i­nary set­back for the com­bi­na­tion, Re­gen­eron be­lieve they can in­di­vid­u­al­ly titrate dos­es and tune the se­quence of drugs to help con­trol the im­mune ac­ti­va­tion in a man­ner that cap­tures the in­creased an­ti­tu­mor ac­tiv­i­ty while lim­it­ing po­ten­tial CRS. Man­age­ment be­lieve this tun­abil­i­ty of dos­ing and se­quenc­ing is an ad­van­tage for their ther­a­peu­tic ap­proach ver­sus CAR-T ther­a­pies.

But don’t look for Re­gen­eron to back off. This com­pa­ny doesn’t back away from any­thing.

From Jef­feries’ Biren Amin:

An up­date on REGN1979 CD20xCD3 bis­pe­cif­ic is ex­pect­ed at 2 EU hema­tol­ogy mt­gs in Jun ’19, in­clud­ing da­ta on high­er dos­es, LT fol­low-up, and ef­fi­ca­cy in CAR-T fail­ures. Mgmt in­tends to ini­ti­ate 2 PII reg­is­tra­tional stud­ies for ‘1979, 1st in FL by mid-’19 and 2nd in DL­B­CL by YE ’19. A com­bo study of ‘1979 w/ Lib­tayo an­ti-PD-1 in 30 ad­vanced lym­phoma pts was al­so start­ed….

Re­gen­eron shares were down 6.5% late af­ter­noon Tues­day, but that wasn’t just about any pos­si­ble jit­ters about its bis­pe­cif­ic. In­creased R&D costs com­bined with flat Eylea sales — around the same time No­var­tis is ex­pect­ed to launch its ri­val brolu­cizum­ab — and a re­duc­tion in cash flow from its for­mer part­ner Sanofi helped dri­ve a miss on earn­ings per share and rev­enue. 

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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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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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Scoop: Roche scraps one of two schiz­o­phre­nia PhII tri­als due to missed pri­ma­ry end­point

Roche has terminated one of two Phase II trials testing its drug ralmitaront in patients with schizophrenia, the Big Pharma confirmed to Endpoints News.

The study was terminated last month, according to a June 22 update to the registry on clinicaltrials.gov. Begun in September 2020, the trial was looking at ralmitaront in patients with acute schizophrenia. The trial enrolled 286 patients out of an originally planned 308.

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Matt Gline, Roivant CEO (John Sciulli/Getty Images for GLG)

Roivant chops sick­le cell gene ther­a­py, der­ma­tol­ogy drugs to fo­cus on 'high­er val­ue pro­ject­s'

Roivant is sweeping a suite of drugs, including a gene therapy for sickle cell disease already in the clinic, out of its pipeline.

Six programs from four of its “vants” are being wound down as part of “a company-wide cost optimization and pipeline reprioritization initiative to reduce our expected operating expenses and prioritize our capital resources.”

When reached by Endpoints News, a spokesperson said, “We don’t anticipate a material reduction in headcount but we will likely reassign some folks to higher value projects as part of winding down specific programs.”

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Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.

Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

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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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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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