Presage teams with Mer­ck on its Phase 0 test­ing; Kem­Pharm AD­HD drug wins ap­proval in chil­dren aged 6 and up

Seat­tle-based Presage Bio­sciences, which ap­proach­es drug de­vel­op­ment through its mi­cro­dos­ing plat­form, has some new part­ner­ships and cash to come with them.

Presage closed a $13 mil­lion fi­nanc­ing round Tues­day, aim­ing to ex­pand its net­work of clin­i­cal tri­al sites and ad­vance de­vel­op­ment of its mi­cro­dos­ing in­jec­tion de­vices. They al­so closed part­ner­ship deals with Mer­ck and Mav­er­ick Ther­a­peu­tics.

The fi­nanc­ing in­clud­ed $7 mil­lion from new in­vestors, in­clud­ing the Lab­Corp Ven­ture Fund, Bris­tol My­ers Squibb, and In­Harv Part­ners. An ad­di­tion­al $6 mil­lion con­vert­ible note from Take­da Ven­tures will con­vert to eq­ui­ty.

Presage’s big idea cen­ters around what they call Phase 0 test­ing, which us­es mi­cro­dos­es of mul­ti­ple drugs and com­bos on pa­tients to gauge their phar­ma­co­dy­nam­ic ef­fects. Their tech al­lows re­searchers to pep­per a tu­mor with mul­ti­ple ther­a­pies while in use by pa­tients, pro­vid­ing a bet­ter snap­shot of rel­a­tive ac­tiv­i­ty.

Tues­day’s new re­search col­lab­o­ra­tions with Mer­ck and Mav­er­ick will uti­lize Presage’s plat­form to eval­u­ate new on­col­o­gy pro­grams in these Phase 0 tri­als, bring­ing their to­tal num­ber of part­ners to five.

Kem­Pharm AD­HD drug wins ap­proval in chil­dren 6 and up

Kem­Pharm scored a new ap­proval on Wednes­day, and it’s send­ing their stock soar­ing.

The Cel­e­bra­tion, FL-based biotech notched the FDA OK for Azs­tarys, a once-dai­ly treat­ment for AD­HD in chil­dren aged six and old­er. Azs­tarys, for­mer­ly dubbed KP415, con­sists of serdexmethylphenidate, Kem­Pharm’s pro­drug of the drug Fo­calin, co-for­mu­lat­ed with an im­me­di­ate-re­lease ver­sion of the prod­uct.

Shares of Kem­Pharm $KMPH sky­rock­et­ed as much as 90% in ear­ly Wednes­day trad­ing, set­tling in at around a 70% in­crease lat­er in the morn­ing. Azs­tarys will be com­mer­cial­ly avail­able as soon as the sec­ond half of this year, and that process will be led by a sub­sidiary of Gur­net Point Cap­i­tal.

The ap­proval will al­so trig­ger a mile­stone pay­ment from Gur­net Point Cap­i­tal to Kem­Pharm, which may to­tal up to $468 mil­lion.

AD­HD has been a lu­cra­tive mar­ket for drug­mak­ers, ac­count­ing for near­ly $18 bil­lion of rev­enue in 2019, Kem­Pharm said.

The pe­di­atric field has proven tough for com­peti­tors, though, with the FDA spurn­ing Su­per­nus’ pitch last No­vem­ber. Reg­u­la­tors cit­ed con­cerns about an in-house lab used to an­a­lyze their ex­per­i­men­tal drug.

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.

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.

Endpoints Premium

Premium subscription required

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

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,700+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,700+ biopharma pros reading Endpoints daily — and it's free.

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.

Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.