Im­munomedics must en­dure an­oth­er de­lay as FDA re­jects can­cer drug cit­ing man­u­fac­tur­ing is­sues

We all like to root for the un­der­dog. Fol­low­ing an arid spell stretch­ing over three decades that in 2017 led to the ouster of Im­munomedics founder/CSO and his CEO wife, the com­pa­ny (and an­a­lysts) were hope­ful the drug de­vel­op­er would fi­nal­ly win its first FDA nod for its treat­ment for a dif­fi­cult-to-treat form of breast can­cer. The man­age­ment shake­up, at the be­hest of an ac­tivist share­hold­er cam­paign, had re­ju­ve­nat­ed the com­pa­ny’s for­tunes by at­tract­ing in­vest­ment and ramp­ing up the drug’s de­vel­op­ment. But the FDA on Thurs­day hand­ed the biotech a re­jec­tion cit­ing man­u­fac­tur­ing is­sues, adding yet an­oth­er de­lay to the reg­u­la­to­ry time­line.

The New Jer­sey-based com­pa­ny’s shares plum­met­ed about 33.5% in ear­ly Fri­day trad­ing.

The drug — called IM­MU-132 or sac­i­tuzum­ab govite­can — is an an­ti­body drug con­ju­gate (ADC) or an armed an­ti­body which is de­signed to de­liv­er a spe­cif­ic pay­load of a chemother­a­py di­rect­ly to a tu­mor, to re­duce the tox­i­c­i­ty as­so­ci­at­ed with the con­ven­tion­al ad­min­is­tra­tion of chemother­a­py. The FDA grant­ed the drug pri­or­i­ty re­view on the ba­sis of da­ta from an on­go­ing sin­gle-arm Phase II study in pa­tients with metasta­t­ic triple-neg­a­tive breast can­cer (mTNBC) who have un­der­gone pri­or ther­a­py. Sac­i­tuzum­ab govite­can, which is al­so be­ing test­ed in a con­fir­ma­to­ry Phase III study in mTNBC pa­tients, was ex­pect­ed to win ac­cel­er­at­ed ap­proval on Jan­u­ary 18. An­a­lysts had sug­gest­ed avail­able mid-stage drug da­ta in­di­cat­ed it was a sig­nif­i­cant im­prove­ment over stan­dard-of-care and had as­signed it peak sales ex­pec­ta­tions of $1 bil­lion and more.

Michael Pehl

In its last up­date in De­cem­ber, Im­munomedics re­port­ed da­ta from the 108-pa­tient mid-stage study — as as­sessed by in­de­pen­dent re­view — demon­strat­ed the drug in­duced par­tial or com­plete tu­mor shrink­age in 34% of pa­tients, with a me­di­an du­ra­tion of re­sponse of about 9 months.

To be sure, the com­pa­ny was not ex­pect­ing the re­jec­tion. It hired a sales force back in No­vem­ber in an­tic­i­pa­tion of the Jan­u­ary ap­proval and told an­a­lysts in De­cem­ber that man­u­fac­tur­ing is­sues the US health reg­u­la­tor had flagged in Au­gust had been since ad­dressed.

In a De­cem­ber 20 note, Jef­feries an­a­lysts said that while man­u­fac­tur­ing is­sues could be a source of con­cern, they re­mained “con­fi­dent in ‘132 as the com­pa­ny be­lieves all is­sues have been re­solved and it has in­vest­ed heav­i­ly in the com­mer­cial in­fra­struc­ture pri­or to ap­proval. Ad­di­tion­al­ly, we would high­light that IM­MU has se­cured an agree­ment with Sam­sung Bi­o­log­ics as a sec­ondary man­u­fac­tur­er…pro­vid­ing risk mit­i­ga­tion if is­sues can­not be re­solved at cur­rent site.”

On Thurs­day, Im­munomedics un­der­scored the sac­i­tuzum­ab govite­can re­jec­tion had noth­ing to do with the drug’s clin­i­cal da­ta.

“The is­sues re­lat­ed to ap­prov­abil­i­ty in the CRL were ex­clu­sive­ly fo­cused on Chem­istry, Man­u­fac­tur­ing and Con­trol mat­ters and no new clin­i­cal or pre­clin­i­cal da­ta need to be gen­er­at­ed,” Im­munomedics chief Michael Pehl said in a state­ment, adding that the com­pa­ny in­tend­ed to re­quest a meet­ing with the FDA to bet­ter un­der­stand the agency’s re­quire­ments.

But with no light at the end of the gov­ern­ment shut­down tun­nel, who knows how long that will take.

Lessons for biotech and phar­ma from a doc­tor who chased his own cure

After being struck by a rare disease as a healthy third year medical student, David Fajgenbaum began an arduous journey chasing his own cure. Amidst the hustle of this year’s JP Morgan conference, the digital trials platform Medable partnered with Endpoints Studio to share Dr. Fajgenbaum’s story with the drug development industry.

What follows is an edited transcript of the conversation between Medable CEO Dr. Michelle Longmire and Dr. Fajgenbaum, and it is full of lessons for biotech executives charged with bringing the next generation of medicines to patients.

No­var­tis gets a boost in block­buster mul­ti­ple scle­ro­sis race with Roche

In the first step of what’s likely to be a long and uphill battle for the drugmaker, the FDA has accepted Novartis’s BLA submission for a new multiple sclerosis drug and given it priority review. The PDUFA date for the potential blockbuster drug is in June.

The drug, known as ofatumumab or Arzerra, has performed consistently well across late-stage trials in patients with the most common form of MS, including in head-to-head studies against Sanofi’s old blockbuster Aubagio. But, if the drug is approved, Novartis will find itself in a crosstown game of catch-up; since a 2017 approval, Roche’s Ocrevus has become the second best-selling MS drug on the market, nearly eclipsing Biogen’s Tecfidera last quarter with over a $1 billion in sales.

Coro­n­avirus out­break threat­ens short­age of 150 drugs — re­port

American patients who suffer from conditions other than Covid-19 could feel the impact of the coronavirus due to shortage of drugs — as 150 prescription drugs are now reportedly on a list of at-risk therapies. The list spans “antibiotics, generics and some branded drugs without alternatives,” Axios reported citing sources familiar with the list. The FDA declined to comment.

Although factories in China are gradually reopening, restrictions in travel and disruptions at transit hubs are still slowing down production. An Indian company that relies on active pharmaceutical ingredients (API) from China told Bloomberg last week that it’s seeing prices of commonly used drugs jump by 40% to 70%.

Juergen Horn

An­i­mal health vet Juer­gen Horn makes new an­ti­body play for pets, rak­ing $15M in Se­ries A haul

Zoetis forked over $85 million in 2017 to acquire Nexvet Biopharma and its pipeline of monoclonal antibodies. Juergen Horn, Nexvet’s former chief product development officer, has now secured $15 million for his own biologic company for animals: Invetx.

Buoyed by emerging advances in gene therapies for humans, scientists have started looking at harnessing the technology for animals setting up companies such as Penn-partnered Scout Bio and George Church-founded Rejuvenate Bio. But akin to Nexvet, Invetx is working on leveraging the time-tested science of monoclonal antibodies to treat chronic diseases that afflict man’s best friend.

Tim Mayleben (file photo)

Es­pe­ri­on's goldilocks cho­les­terol fight­er wins FDA ap­proval — will its 'tra­di­tion­al' pric­ing ap­proach spur adop­tion?

It’s more effective than decades-old statins but not as good as the injectable PCSK9 — the goldilocks treatment for cholesterol-lowering, bempedoic acid, has secured FDA approval.

Its maker, Esperion Therapeutics, is betting that their pricing strategy — a planned list price of between $10 to $11 a day — will help it skirt the pushback the PCSK9 cholesterol fighters, Repatha and Praluent, got from payers for their high sticker prices.

The sky-high expectations for the pair of PCSK9 drugs that were first approved in 2015 quickly simmered — and despite a 60% price cut, coupled with data showing the therapies also significantly cut cardiovascular risk, sales have not really perked up.

Esperion is convinced that by virtue of being a cheaper oral therapy, bempedoic acid will hit that sweet spot in terms of adoption.

“We’re kind of like the old comfortable shoe,” Esperion’s chief commercial officer Mark Glickman remarked in an interview with Endpoints News ahead of the decision date. “It’s an oral product, once-daily and nontitratable — these are things that just resonate so true with patients and physicians and I think we’ve kind of forgotten about that.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,900+ biopharma pros reading Endpoints daily — and it's free.

As coro­n­avirus out­break reach­es 'tip­ping point,' GSK lends ad­ju­vant tech to Chi­nese part­ner armed with pre­clin­i­cal vac­cine

As the coronavirus originating out of Wuhan spreads to South Korea, Italy and Iran, stoking already intense fears of a pandemic, GlaxoSmithKline has found another pair of trusted hands to place its adjuvant system. China’s Clover Biopharmaceuticals will add the adjuvant to its preclinical, protein-based vaccine candidate against SARS-CoV-2.

Clover, which is based in the inland city of Chengdu, boasts of a platform dubbed Trimer-Tag that produces covalently-trimerized fusion proteins. Its candidate, COVID-19 S-Trimer, resembles the viral spike (S)-protein found in the virus.

Drug ap­provals: FDA pub­lish­es dataset of CDER ap­provals since 1985

To provide researchers with more accurate and accessible data about historic drug approvals, the FDA on Friday released a dataset containing information about all new drugs and biologics approved by the Center for Drug Evaluation and Research (CDER) dating back to 1985.

“FDA created the compilation to facilitate data accessibility, transparency, and accuracy when researchers seek information about an approved drug,” the FDA writes, adding that the compilation should accurately reflect “the state of each application at the time of initial regulatory approval.”

Deborah Dunsire

The fourth CGRP mi­graine drug is here. Time for Lund­beck to prove it's worth $2B

They may be late, but Lundbeck is now officially in the game for preventing migraine with CGRP drugs.

The FDA has OK’d eptinezumab, the prize in Lundbeck’s $2 billion acquisition of Alder. Like rival offerings from Amgen/Novartis, Eli Lilly and Teva, the antibody blocks the calcitonin gene-related peptide, which is believed to dilate blood vessels in the brain and cause pain.

It will now be sold as Vyepti. The company has yet to announce a price. Amgen and Novartis had set the wholesale acquisition cost of their pioneering Aimovig at $6,900 for a year’s supply before raising it slightly this year; Lilly and Teva had followed suit with Emgality and Ajovy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,900+ biopharma pros reading Endpoints daily — and it's free.

Tal Zaks (Moderna via YouTube)

For two decades, a new vac­cine tech­nol­o­gy has been slow­ly ap­proach­ing prime time. Now, can it stop a pan­dem­ic?

Two months before the outbreak, Moderna CMO Tal Zaks traveled from Cambridge, MA to Washington DC to meet with Anthony Fauci and the leaders of the National Institutes of Health.

For two years, Moderna had worked closely with NIH researchers to build a new kind of vaccine for MERS, one of the deadliest new viruses to emerge in the 21st century. The program was one test for a new technology designed to be faster, cheaper and more precise than the ways vaccines had been made for over a century. They had gathered evidence the technology could work in principle, and Fauci, the longtime head of the National Institute of Allergy and Infectious Diseases and a longtime advocate for better epidemic preparedness, wanted to see if it, along with a couple of other approaches, could work in a worst-case scenario: A pandemic.

“[We were] trying to find a test case for how to demonstrate if our technology could rapidly prepare,” Zaks told Endpoints News.

Zaks and Fauci, of course, wouldn’t have to wait to develop a new test. By year’s end, an outbreak in China would short circuit the need for one and throw them into 24/7 work on a real-world emergency. They also weren’t the only ones with new technology who saw a chance to help in a crisis.

An ocean away, Lidia Oostvogels was still on vacation and relaxing at her mother’s house in Belgium when her Facebook started changing. It was days after Christmas and on most people’s feeds, the news that China had reported a novel virus to the World Health Organization blurred into the stream of holiday sweaters and fir trees. But on Oostvogels’s feed, full of vaccine researchers and virus experts, speculation boiled: There was a virus in China, something contained to the country, but “exotic,” “weird,” and maybe having to do with animals. Maybe a coronavirus.

Lidia Oostvogels

“I was immediately thinking like, ‘Hey, this is something that if needed, we can play a role,'” Oostvogels told Endpoints.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 72,900+ biopharma pros reading Endpoints daily — and it's free.