FDA push­es back As­cendis' PDU­FA for long-act­ing hor­mone ther­a­py; De­bio­pharm takes home a win in mid-stage test for ADC

Dan­ish biotech As­cendis Bio­phar­ma has been work­ing on a treat­ment for pe­di­atric growth hor­mone de­fi­cien­cy, but the FDA says they’re go­ing to have to wait a lit­tle bit longer be­fore they can make their ap­proval de­ci­sion.

Reg­u­la­tors ex­tend­ed the re­view pe­ri­od by three months on Mon­day, As­cendis said, push­ing back the PDU­FA date to Sep­tem­ber 25. The ex­ten­sion was in re­sponse to ad­di­tion­al in­for­ma­tion As­cendis sub­mit­ted as part of its BLA.

“We have re­spond­ed to all out­stand­ing ques­tions from the FDA and be­lieve the com­plete pack­age we have sub­mit­ted sat­is­fies all of FDA’s re­quests,” CEO Jan Mikkelsen said in a state­ment.

The pro­gram in ques­tion is known as lon­apeg­so­ma­t­ropin, de­signed to de­liv­er the same un­mod­i­fied hu­man growth hor­mone used in dai­ly ther­a­pies, but at a pre­dictable rate once a week. Re­searchers have been work­ing on a ther­a­py for chil­dren, as well as for adults with growth hor­mone de­fi­cien­cy.

In their Phase III pe­di­atric tri­al back in 2019, the week­ly ther­a­py not on­ly demon­strat­ed non-in­fe­ri­or­i­ty to a dai­ly ver­sion, but al­so showed su­pe­ri­or­i­ty af­ter one year. As­cendis is hop­ing to cap­i­tal­ize on those re­sults, which sur­prised an­a­lysts at the time, by pro­duc­ing what would be the first long-act­ing hor­mone ther­a­py. — Max Gel­man

A non-Hodgkin’s lym­phoma ADC shows pos­i­tive re­sponse in 30% of pa­tients

Phase II da­ta from De­bio­pharm’s ADC naratux­imab em­tan­sine has shown promise for the treat­ment of one of the most ag­gres­sive forms of non-Hodgkin’s lym­phoma, the com­pa­ny said Mon­day.

The com­plete re­sponse rate was 31.6% in all pa­tients with dif­fuse large B cell lym­phoma, and 32% in pa­tients with more than two pri­or ther­a­pies. About 8% of pa­tients stopped treat­ment be­cause of side ef­fects.

De­bio­pharm CEO Bertrand Ducrey said in a state­ment:

We now see this al­ter­na­tive com­bi­na­tion tar­get­ing both CD37 and CD20 could ad­dress the un­met need of DL­B­CL pa­tients who have re­lapsed or not re­spond­ed to ear­li­er lines of treat­ment. These promis­ing ef­fi­ca­cy re­sults and good safe­ty pro­file are mo­ti­va­tion for fur­ther ex­plo­ration of the po­ten­tial ben­e­fit that this po­tent ADC tech­nol­o­gy could bring to pa­tients. We’ve clear­ly ar­rived at the stage of se­lect­ing a part­ner to un­fold the full val­ue of this nov­el ther­a­peu­tic op­tion.

Naratux­imab em­tan­sine treats pa­tients with dif­fuse large B-cell lym­phoma, which ac­counts for about 30-40% of all non-Hodgkin’s lym­phoma cas­es. About half of pa­tients aren’t cured by the first round of treat­ment. Naratux­imab em­tan­sine tar­gets the CD37 anti­gen to re­lease a tox­ic DM1 pay­load. The du­ra­tion of re­sponse last­ed longer than one year for 66% of pa­tients, the com­pa­ny said. — Josh Sul­li­van

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a rather narrow market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Den­mark's Gubra to col­lab­o­rate with Bay­er on pep­tides; Sam­sung and Bio­gen re­ceive FDA ap­proval for Lu­cen­tis biosim­i­lar

Danish biotech Gubra announced a research collaboration and license agreement with Bayer to develop peptide therapeutics to treat cardiorenal diseases. The collaboration will utilize Gubra’s peptide drug discovery platform to identify potential candidates.

This is not the first time Gubra has partnered with a company on peptide therapeutics — they partnered with Boehringer Ingelheim back in 2017 to create peptide therapeutics to treat obesity.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.