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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect"

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Pearl Huang, former Cygnal Therapeutics president and CEO

UP­DAT­ED: Flag­ship builds a new start­up out of pieces from 2 of its biotechs. And a Roche vet leaves to do some­thing new

Flagship has crafted a new startup out of pieces from a pair of fledglings in the VC’s nest. And a prominent Roche veteran who ran one of the biotechs won’t be making the next leg of the journey.

The new company is called Sonata Therapeutics, which is picking up the work that Inzen was doing related to the cellular microenvironment and combining with Flagship’s Cygnal Therapeutics, which came out of stealth more than 3 years ago and put Pearl Huang — the BeiGene founder and former Roche SVP — at the helm.

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Amidst R&D reshuf­fle, Ver­tex ex­pands its pres­ence in Boston, aim­ing to be­come num­ber one

Vertex Pharmaceuticals has been one of the buzzier names in the bustling Boston biotech scene, but now the company is looking to vault to number one status — at least in terms of physical footprint.

At a ribbon cutting on Tuesday for its new Jeffrey Leiden Center for Cell and Genetic Therapies at the Boston Seaport, Vertex announced it would embark on a new project: The company will build a 344,000 square foot facility in the seaport to accommodate the company’s growing R&D needs, especially in its cell and gene therapies program.

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Martin Shkreli (Dennis Van Tine/MediaPunch/IPX)

In­fa­mous biotech ex­ec Mar­tin Shkre­li gets out of prison, hits the street

Martin Shkreli, the infamous biotech CEO who made headlines for his jeering assault on a legion of critics in and out of Congress, is back on the streets after 4 years inside a federal penitentiary.

Shkreli’s attorney put out a statement Wednesday afternoon saying that the “pharma bro” had been transferred to a halfway house in New York with a few more months to go under federal custody, slated to end September 14. Attorney Benjamin Brafman acknowledged the release and vowed that he and Shkreli are keeping quiet.

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De­spite fed­er­al ef­forts to di­ver­si­fy clin­i­cal tri­als, progress re­mains 'stag­nan­t' — re­port

While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report from the National Academies of Sciences, Engineering and Medicine.

Swaths of patients in racial and ethnic minority groups, as well as LGBTQIA+, pregnant and older adult populations continue to be left out of clinical trials. While some advances have been made in the last 30 years — women now account for roughly half of clinical trial participants — growth in other areas remains stagnant, according to the report, which was mandated by Congress and sponsored by the NIH.

Paul Chaplin, Bavarian Nordic president and CEO

Bavar­i­an Nordic se­cures BAR­DA con­tract for small­pox vac­cine

It seems that smallpox vaccination production is weighing on the mind of the US government. And manufacturer Bavarian Nordic is the latest company to benefit.

Just a few days after Emergent, a company that has made government contracts its lifeblood, acquired the exclusive rights to Tembexa from Chimerix, with a $225 million cash payment and an expected BARDA contract, the agency has offered a contract for smallpox vaccine production.

Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

House com­mit­tee unan­i­mous­ly ad­vances FDA user fee leg­is­la­tion with ac­cel­er­at­ed ap­proval tweaks

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.