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.”

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

In­cyte wins ac­cel­er­at­ed ap­proval for PD-1 in rare skin can­cer

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.

FDA re­jects Ab­b­Vie's in­fu­sion ther­a­py for Parkin­son's, re­quests more in­fo on pump de­vice

The FDA rejected AbbVie’s 24-hour infusion therapy for Parkinson’s, saying it needs more information on a device used to administer the treatment before it can clear it.

The Chicago-area drugmaker said in a press release that the complete response letter from the agency didn’t include any requests for more efficacy or safety trials related to the drug, known as ABBV-951. The company said it aims to “resubmit the NDA as soon as possible.”

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A new study finds that many patient influencers are sharing prescription drug experiences along with health information.

So­cial me­dia pa­tient in­flu­encers ‘danc­ing in the gray’ of phar­ma mar­ket­ing, more clar­i­ty need­ed, re­searcher says

It’s no surprise that patient influencers are talking about their health conditions on social media. However, what’s less clear is what role pharma companies are playing, how big the patient influencer industry is, and just how is information about prescription drugs from influencers relayed — and received — on social media.

While University of Colorado associate professor Erin Willis can’t answer all those questions, she’s been researching the issue for several years and recently published new research digging into the communication styles, strategies and thinking of patient influencers, many of whom partner with pharma companies.

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Drug short­age so­lu­tions brought be­fore Sen­ate Home­land Se­cu­ri­ty com­mit­tee

With more than 300 active drug shortages, the Senate Committee on Homeland Security and Governmental Affairs had its hands full on Wednesday with multiple experts testifying on drug shortages and possible solutions.

A picture of the shortage situation. presented by Erin Fox, an adjunct professor at the College of Pharmacy at the University of Utah, explained how some patients have died due to drug shortages, including with medication errors when substitutes were dosed incorrectly or when an emergency product was not available.

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