John Temperato, 9 Meters president and CEO

PhI­II dead end for celi­ac drug crush­es pen­ny stock biotech

A com­pound that’s been tout­ed as the first-ever drug to en­ter a Phase III tri­al for celi­ac dis­ease ap­pears to have run in­to a dead end.

At an in­ter­im sta­tis­ti­cal analy­sis de­signed to de­ter­mine how many more pa­tients it would take to de­tect a sta­tis­ti­cal­ly sig­nif­i­cant ef­fect of lara­zotide, 9 Me­ters Bio­phar­ma said it may have to shut­ter the study be­cause “the ad­di­tion­al num­ber of pa­tients need­ed to de­ter­mine a sig­nif­i­cant clin­i­cal out­come be­tween place­bo and lara­zotide is too large to sup­port tri­al con­tin­u­a­tion.”

The com­pa­ny will keep look­ing over the da­ta for any sil­ver lin­ings — sub­group ef­fects, par­tic­u­lar symp­tom al­le­vi­a­tion — and talk to the FDA be­fore fi­nal­iz­ing plans about the drug.

But ex­ecs are al­ready prepar­ing in­vestors for the worst case sce­nario.

“Pend­ing the fi­nal analy­sis, fi­nan­cial and hu­man re­sources pre­vi­ous­ly ded­i­cat­ed to this study will be re-de­ployed to ad­vance our de­vel­op­ment pro­gram for vurole­natide and our ear­ly-stage prod­uct can­di­dates,” pres­i­dent and CEO John Tem­per­a­to said in a state­ment, re­fer­ring to 9 Me­ters’ oth­er lead pro­gram, a GLP-1 re­cep­tor ag­o­nist.

Shares of the Raleigh, NC-based com­pa­ny $NMTR, which slid in­to pen­ny stock sta­tus ear­li­er this year, dropped an­oth­er 56% to $0.23.

Fo­cus­ing on pa­tients with celi­ac dis­ease who con­tin­ue to ex­pe­ri­ence gas­troin­testi­nal symp­toms, the Phase III tri­al had a 24-week de­sign, in­clud­ing 5 weeks for screen­ing and 12 weeks for dou­ble-blind treat­ment. It would en­roll 525 pa­tients, ran­dom­ized in­to three dos­ing arms: lara­zotide 0.25 mg, lara­zotide 0.5 mg and place­bo.

Once the first half of the tri­al pop­u­la­tion com­plet­ed their 12-week ef­fi­ca­cy por­tion, though, 9 Me­ters hired an in­de­pen­dent sta­tis­ti­cian to re-es­ti­mate, based on the da­ta from those treat­ed pa­tients, the ac­tu­al “treat­ment group size re­quired to de­tect a sta­tis­ti­cal­ly sig­nif­i­cant clin­i­cal ef­fect of lara­zotide.”

The pri­ma­ry end­point was mean change for celi­ac symp­tom sever­i­ty, as mea­sured by the CeD PRO score.

The tri­al be­gan back in 2019, be­fore In­no­vate Bio­phar­ma­ceu­ti­cals, the orig­i­nal de­vel­op­er of lara­zotide, merged with Or­biMed-backed pri­vate start­up RDD Phar­ma to be­come 9 Me­ters. More re­cent­ly, the com­pound’s in­ven­tors pro­posed us­ing it to treat se­vere post-Covid dis­ease in chil­dren.

Thanks in part to the RDD deal, 9 Me­ters said it has oth­er as­sets in de­vel­op­ment for rare gas­troin­testi­nal dis­or­ders, led by vurole­natide, which is in Phase II for short bow­el syn­drome.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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?”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.