Ready to de­clare a de­fin­i­tive come­back in two months, Im­munomedics stops PhI­II ear­ly, re­cruits new CEO

More than a year ago, hit by a sur­prise com­plete re­sponse let­ter from the FDA, Im­munomedics bid its then-CEO, Michael Pehl, adieu and be­gan a 15-month quest to re­solve the man­u­fac­tur­ing is­sues cit­ed in the CRL and seek a new leader — all the while mov­ing for­ward with a Phase III study on its lead drug for metasta­t­ic triple-neg­a­tive breast can­cer.

Harout Se­mer­jian

To­day the biotech said their stars are fi­nal­ly align­ing. Not on­ly is No­var­tis On­col­o­gy vet Harout Se­mer­jian com­ing on board as CEO to steer what they be­lieve will be a smooth sail to a new PDU­FA date in June, Im­munomedics has al­so been in­formed that their late-stage tri­al can be stopped ear­ly due to “com­pelling ev­i­dence of ef­fi­ca­cy.”

The turn­around is not ex­act­ly com­plete but it’s enough to win in­vestors over, near­ly dou­bling its shares $IM­MU to $18.77.

The rec­om­men­da­tion by the da­ta safe­ty mon­i­tor­ing com­mit­tee means that Im­munomedics doesn’t need to wait un­til a cer­tain num­ber of pa­tients to progress be­fore they be­gin an­a­lyz­ing the da­ta.

The Phase III AS­CENT study was de­signed to eval­u­ate how its an­ti­body-drug con­ju­gate dubbed sac­i­tuzum­ab govite­can im­pacts pro­gres­sion-free sur­vival, over­all sur­vival and re­sponse rates com­pared to chemother­a­py.

Be­hzad Ag­haz­adeh

The plan is to sub­mit the re­vised sta­tis­ti­cal analy­sis plan to the FDA in a mat­ter of hours, ex­ec­u­tive chair­man Be­hzad Ag­haz­adeh told End­points News Mon­day morn­ing.

“More im­por­tant­ly, it’s not so much that we don’t have to wait any longer for pa­tients to progress, the in­de­pen­dent da­ta safe­ty mon­i­tor com­mit­tee is in­di­cat­ing to us that the tri­al was suc­cess­ful,” he said.

He cau­tious­ly added that the ex­perts on the com­mit­tee ap­pear to in­di­cate that in ad­di­tion to the pri­ma­ry end­point in PFS, they are al­so see­ing pos­i­tive trends on oth­er end­points.

Julie Gralow

Julie Gralow, a Fred Hutch re­searcher who chaired the DSMC, called the re­sults “re­mark­able” — a com­ment that in­spired Cowen’s Phil Nadeau to note “there is lit­tle ques­tion that sac­i­tuzum­ab will soon be­come stan­dard of care in the treat­ment of re­lapsed and re­frac­to­ry mTNBC.”

To be clear, none of that should fac­tor in­to the FDA’s on­go­ing re­view of sac­i­tuzum­ab govite­can. Ag­haz­adeh still ex­pects an ac­cel­er­at­ed ap­proval by June 2. Af­ter all, ac­cord­ing to the com­pa­ny, the on­ly is­sues stop­ping reg­u­la­tors from do­ing so the first time around had to do with man­u­fac­tur­ing and oth­er pro­ce­dur­al mat­ters dis­cov­ered dur­ing in­spec­tions. Scott Canute, a board mem­ber who was tapped as ex­ec­u­tive di­rec­tor up­on Pehl’s de­par­ture, has helped en­sure re­me­di­a­tion is in place to ad­dress those con­cerns.

On the oth­er hand, de­spite the short­ened time­line, a full ap­proval wouldn’t come un­til 6, 9, or even 12 months from now.

Im­munomedics has pre­vi­ous­ly pre­sent­ed Phase II da­ta sug­gest­ing a 33% re­sponse rate and a me­di­an du­ra­tion of re­sponse of 7.7 months.

“It is 2 or 3 times high­er based on what we would ex­pect in chemother­a­py,” Ag­haz­adeh, who al­so man­ages Avoro Cap­i­tal (for­mer­ly ven­Bio Se­lect Ad­vi­sor), said. “For the re­sponse rate, gen­er­al­ly we would ex­pect high sin­gle-dig­it, low dou­ble-dig­it re­spons­es. And we would ex­pect about 3 to 4 months of du­ra­tion for chemother­a­py.”

For Nadeau, it all bodes well for fur­ther clin­i­cal de­vel­op­ment of sac­i­tuzum­ab govite­can, with 2025 sales pegged at $1.2 bil­lion:

With sac­i­tuzum­ab pro­duc­ing sim­i­lar Ph. II da­ta in r/r ER+/HER2- mBC and mTNBC, we think the AS­CENT re­sults par­tial­ly de-risk sac­i­tuzum­ab’s de­vel­op­ment in r/r ER+/HER2- mBC. IM­MU ex­pects to com­plete en­roll­ment in the Ph. III TROP­iCS-02 study in ER+/HER2- BC this year with in­ter­im da­ta (ORR) to be re­leased not long af­ter the com­ple­tion of en­roll­ment.

The re­spon­si­b­li­ty to re­al­ize all that — start­ing with a po­ten­tial near-term launch — now falls on the shoul­der of Se­mer­jian.

Se­mer­jian moved days ago from his last job as chief com­mer­cial of­fi­cer of Ipsen in Lon­don and, in a sign of the times, “is sit­ting at home in so-called self quar­an­tine to en­sure that he is able to safe­ly come to the com­pa­ny site on [his of­fi­cial start date of] April 16,” Ag­haz­adeh said.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

Credit: Shutterstock

How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

An­gion's or­gan dam­age drug strikes out again, this time in high-risk kid­ney trans­plant pa­tients

After flopping a test in Covid-19 earlier this year, Angion’s lead organ damage drug has now hit the skids again in kidney transplant patients.

Angion and partner Vifor Pharma’s ANG-3777 failed to beat out placebo in terms of improving eGFR, a measure of kidney function, in patients who had received a deceased donor kidney transplant and were at high risk of developing what is known as delayed graft function, according to Phase III results released Tuesday.

An image of Alzheimer's brain tissue. The red show gingipains, a protein from P. gingivalis, intermixing with neurons (yellow) and glial cells (green)

An Alzheimer's dark­horse fails its first big tri­al, but of­fers hope for a long-over­looked hy­poth­e­sis

Three years ago, Cortexyme emerged out of obscurity with some big-name backers and an unorthodox approach to treating Alzheimer’s.

They moved their drug into a pivotal study the next year, offering one of the first major tests for a hypothesis that has fluttered on the outskirts of Alzheimer’s research for decades: that, in many cases, the disease is driven by infectious agents — the havoc they wreak in the brain and the inflammation the body uses to try to fend them off. And that quashing the infection could slow patients’ cognitive decline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,800+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,800+ biopharma pros reading Endpoints daily — and it's free.

Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,800+ biopharma pros reading Endpoints daily — and it's free.

FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,800+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,800+ biopharma pros reading Endpoints daily — and it's free.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.