Over a year af­ter re­jec­tion, Im­munomedics emerges with po­ten­tial block­buster ap­proval

A tur­bu­lent 16 months for Im­munomedics has end­ed in a po­ten­tial block­buster ap­proval, as the FDA grant­ed an ac­cel­er­at­ed OK for their drug to treat an ag­gres­sive form of breast can­cer.

The drug, brand­ed as Trodelvy, is ap­proved for metasta­t­ic triple neg­a­tive breast can­cer — breast can­cers that lack the onco­genes that tar­get­ed ther­a­pies go af­ter and that have helped make the dis­ease treat­able for some pa­tients. The drug, which had break­through sta­tus and pri­or­i­ty re­view, showed a 33% re­sponse and me­di­an du­ra­tion of re­sponse of 7.7 months in 108 pa­tients en­rolled in their Phase II tri­al. Peak sales es­ti­mates can range as high as around $3 bil­lion.

The ap­proval comes over a month in ad­vance of the com­pa­ny’s June 2 PDU­FA date, con­tin­u­ing the agency’s trend of quick­ly ap­prov­ing can­cer drugs they deem ef­fec­tive.

Be­hzad Ag­haz­adeh

A lit­tle over a year ago, the FDA sur­prised both the com­pa­ny and in­vestors by re­ject­ing the drug. Al­though Im­munomedics $IM­MU said the con­cern had on­ly been with qual­i­ty con­trols over the fa­cil­i­ty where the drug is pro­duced, the re­jec­tion of the biotech’s main as­set shaved $1 bil­lion off the com­pa­ny’s mar­ket cap. Not long af­ter, an FDA in­spec­tion doc­u­ment raised con­cerns of a da­ta breach at one of the com­pa­ny’s fa­cil­i­ties. The CEO and CSO both lat­er ex­it­ed, and chair­man Be­hzad Ag­haz­adeh stepped in to run the com­pa­ny.

The drug, known chem­i­cal­ly as sac­i­tuzum­ab govite­can, is the com­pa­ny’s first ap­proval in their 37-year his­to­ry. An an­ti­body-drug con­ju­gate, it con­sists of a tu­mor-tar­get­ing an­ti­body at­tached to a cell-killing small mol­e­cule. The tu­mor en­gulfs the con­ju­gate and the mol­e­cule kills the can­cer cells. The idea first emerged in the ear­ly 2000s be­fore pe­ter­ing out, but has gained trac­tion again in re­cent years, most no­tably with the re­cent ap­provals of Seat­tle Ge­net­ics’ Ad­cetris and Pad­cev.

Seat­tle Ge­net­ics near­ly ac­quired the Im­munomedics drug in 2016. They agreed to an up-to $2 bil­lion deal worth $300 mil­lion be­fore ven­Bio, which owned a 9.9% stake in the com­pa­ny, led a cam­paign against the deal, with then-man­ag­ing parter Ag­haz­adeh ac­cus­ing the com­pa­ny’s lead­er­ship of try­ing to en­rich them­selves. The hus­band-and-wife team who then ran the com­pa­ny as CSO and CEO re­signed, and Ag­haz­adeh lat­er or­ga­nized a $250 mil­lion roy­al­ty deal to fund the com­pa­ny’s work.

Harout Se­mer­jian

An ac­cel­er­at­ed ap­proval is con­di­tion­al on the com­pa­ny con­duct­ing a fol­low-up study. That tri­al, though, has al­ready been stopped, with an in­de­pen­dent re­view board telling Im­munomedics ear­ly this month their drug had al­ready shown enough com­pelling ef­fi­ca­cy. On the same day of the tri­al news, the com­pa­ny al­so an­nounced that No­var­tis On­col­o­gy vet Harout Se­mer­jian had been named as the new CEO.

Com­mit­tee chair and Fred Hutch re­searcher Julie Gralow called the re­sults of the Phase III tri­al “re­mark­able.” The com­ment led Cowen’s Phil Nadeau to write, “There is lit­tle ques­tion that sac­i­tuzum­ab will soon be­come stan­dard of care in the treat­ment of re­lapsed and re­frac­to­ry mTNBC.”

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,400+ biopharma pros reading Endpoints daily — and it's free.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

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.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

Endpoints Premium

Premium subscription required

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

Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,400+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.

Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,400+ biopharma pros reading Endpoints daily — and it's free.

Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.