In 1-2 punch, Mol­e­c­u­lar Tem­plates cuts first-gen can­di­date and los­es a Big Phar­ma part­ner; Charles Riv­er teams up with Va­lence to pro­vide AI dis­cov­ery plat­form

It’s been sev­er­al months since a treat­ment-re­lat­ed death led the FDA to put a par­tial hold on Mol­e­c­u­lar Tem­plates’ on­ly first-gen­er­a­tion en­gi­neered tox­in body (ETB) can­di­date. Now the com­pa­ny is nix­ing the pro­gram, and Take­da is re­turn­ing the rights to one of its next-gen can­di­dates.

MT-3724, Mol­e­c­u­lar’s EBT in de­vel­op­ment for non-Hodgkin lym­phoma, will be no more, the com­pa­ny said on Mon­day morn­ing. The FDA first placed a par­tial hold on the can­di­date in No­vem­ber, fol­low­ing the death of a Phase II pa­tient who ex­pe­ri­enced Grade 5 cap­il­lary leak syn­drome.

“Marked­ly high phar­ma­co­ki­net­ic as­say read­ings were ob­served in this and oth­er sub­jects treat­ed with a spe­cif­ic lot of MT-3724 ma­te­r­i­al,” ac­cord­ing to Mol­e­c­u­lar.

The agency fol­lowed up with a full hold in late March, and re­quest­ed ad­di­tion­al in­for­ma­tion and the de­vel­op­ment of a new quan­ti­ta­tive as­say spe­cif­ic to MT-3724, which Mol­e­c­u­lar says “would take sig­nif­i­cant time and in­vest­ment away” from its pri­or­i­ties.

$MTEM shares dropped more than 25% on the news Mon­day.

In the same an­nounce­ment, Mol­e­c­u­lar said Take­da is back­ing out of a col­lab­o­ra­tion deal for TAK-169, a sec­ond-gen­er­a­tion ETB tar­get­ing CD38 for the treat­ment of mul­ti­ple myelo­ma. Take­da forked over $30 mil­lion up­front for the part­ner­ship back in 2018, and promised mile­stones up to $632.5 mil­lion.

Take­da is turn­ing the full rights over to Mol­e­c­u­lar, which will owe Take­da “low-sin­gle dig­it roy­al­ties” on fu­ture net sales. The drug, which was de­signed to over­come tu­mor re­sis­tance ob­served with Janssen and Gen­mab’s dara­tu­mum­ab (the first ap­proved mon­o­clon­al an­ti­body tar­get­ing CD38), is in a Phase I study.

Ac­cord­ing to Mol­e­c­u­lar, Take­da says its de­ci­sion to back out of the part­ner­ship was the re­sult of “on­go­ing port­fo­lio pri­or­i­ti­za­tion.”

So far, Take­da has en­rolled and treat­ed four sub­jects in the TAK-169 Phase I study, and Mol­e­c­u­lar says there have been no life-threat­en­ing tox­i­c­i­ties or signs of CLS.

Mol­e­c­u­lar’s EBTs use ge­net­i­cal­ly en­gi­neered ver­sions of the Shi­ga-like Tox­in A sub­unit. They go af­ter can­cer cells and al­so add anti­gens in­to the can­cer cells them­selves, help­ing prime the im­mune sys­tem to tar­get them for de­struc­tion. CEO and CSO Er­ic Po­ma says the com­pa­ny plans to “take im­me­di­ate steps to ac­cel­er­ate new site ac­ti­va­tion and pa­tient en­roll­ment in the TAK-169 study to gen­er­ate da­ta this year.” — Nicole De­Feud­is

Charles Riv­er part­ners with Va­lence to pro­vide AI dis­cov­ery plat­form

Big-name CD­MO Charles Riv­er Lab­o­ra­to­ries has swung an­oth­er deal, this time giv­ing its clients ac­cess to a new AI plat­form.

The Wilm­ing­ton, MA-based firm has teamed up with Va­lence Dis­cov­ery, the com­pa­nies an­nounced Tues­day, in an ef­fort to ex­pand Va­lence’s AI plat­form for mol­e­c­u­lar prop­er­ty pre­dic­tion, gen­er­a­tive chem­istry and mul­ti­pa­ra­me­ter op­ti­miza­tion. Fi­nan­cial terms of the deal were not dis­closed.

Va­lence says its plat­form en­ables the de­sign of small mol­e­cule drugs in new re­gions of chem­i­cal space, al­low­ing for rapid op­ti­miza­tion against po­ten­cy, se­lec­tiv­i­ty, safe­ty and phar­ma­col­o­gy cri­te­ria on a project-by-project ba­sis. Com­pa­nies tak­ing ad­van­tage of Charles Riv­er’s CD­MO of­fer­ings can opt in­to the Va­lence pro­gram.

Charles Riv­er has been busy to start off 2021, se­cur­ing a $104 mil­lion buy­out of Dis­tri­b­u­tion Bio in Jan­u­ary and Cog­nate in an $875 mil­lion ac­qui­si­tion in Feb­ru­ary. — Max Gel­man

With topline PhII da­ta in hand, Schol­ar Rock races to­ward piv­otal tri­al launch

Schol­ar Rock says it got the topline Phase II da­ta it was look­ing for with its spinal mus­cu­lar at­ro­phy (SMA) drug apite­gromab, and is rac­ing to launch a piv­otal tri­al by the end of this year.

The Phase II tri­al en­rolled pa­tients across three co­horts: pa­tients be­tween 5 and 21 years old with am­bu­la­to­ry type 3 SMA; pa­tients be­tween 5 and 21 years old with type 2 and non-am­bu­la­to­ry type 3 SMA who start­ed the treat­ment nusin­ersen when they were old­er than 5 years old; and pa­tients old­er than 2 years old with type 2 SMA who ini­ti­at­ed nusin­ersen younger than 5.

In the first co­hort, pa­tients re­ceived ei­ther apite­gromab ei­ther as a monother­a­py or in com­bo with nusin­ersen. A to­tal of 57% of pa­tients ob­served a main­te­nance or im­prove­ment on the Re­vised Ham­mer­smith Scale (RHS), and 22% of pa­tients achieved at least a 3-point in­crease in RHS score from base­line, ac­cord­ing to Schol­ar Rock.

In Co­hort 2, which ex­clud­ed one pa­tient who was al­so re­ceiv­ing an acetyl­cholinesterase in­hibitor and one who missed con­sec­u­tive dos­es due to Covid-19, the mean change from base­line on  Ham­mer­smith Func­tion­al Mo­tor Scale Ex­pand­ed was a 1.2-point im­prove­ment.

And in Co­hort 3, where pa­tients ei­ther re­ceived a low or high dose of apite­gromab in com­bi­na­tion with nusin­ersen, the mean change from base­line in HFMSE score was a 7.1-point and a 5.3-point im­prove­ment for the 20 mg/kg and 2 mg/kg dose arms, re­spec­tive­ly. The ma­jor­i­ty (59%) of pa­tients in Co­hort 3 achieved at least a 5-point in­crease in HFMSE and 35% of pa­tients achieved greater than a 10-point in­crease in HFMSE over base­line, ac­cord­ing to Schol­ar Rock. That analy­sis al­so ex­clud­ed three pa­tients who had missed dos­es due to Covid-19 chal­lenges.

Apite­gromab aims to boost mus­cle mass and strength in SMA pa­tients by in­hibit­ing the growth fac­tor myo­statin, which breaks down mus­cle mass in the body.

“These top-line 12-month da­ta pro­vide fur­ther sup­port to­wards es­tab­lish­ing apite­gromab as a po­ten­tial first mus­cle-di­rect­ed ther­a­py for pa­tients with SMA,” CMO Yung Chyung said in a state­ment. “The find­ings al­so of­fer im­por­tant in­sights in­to myo­statin bi­ol­o­gy and our sci­en­tif­ic ap­proach of tar­get­ing the la­tent forms of growth fac­tors. — Nicole De­Feud­is

Sor­ren­to Ther­a­peu­tics snaps up a late-stage TKI in ACEA Ther­a­peu­tics buy­out

Sor­ren­to Ther­a­peu­tics is buy­ing out ACEA Ther­a­peu­tics — and with it, a late-stage ty­ro­sine ki­nase in­hibitor, an ex­ten­sive li­brary of small mol­e­cules, and a 23-acre cGMP fa­cil­i­ty in Quzhou, Chi­na.

The com­pa­nies be­gan talk­ing about the merg­er back in Oc­to­ber, and of­fi­cial­ly signed the agree­ment on Mon­day.

With the deal, $SRNE is snap­ping up abiver­tinib, a small mol­e­cule TKI that was orig­i­nal­ly dis­cov­ered us­ing ACEA’s com­pound li­brary. The can­di­date has com­plet­ed a Phase III tri­al in NSCLC, and is cur­rent­ly in Phase II for Covid 19-in­duced res­pi­ra­to­ry com­pro­mise in the US and Brazil. It’s al­so get­ting AC0058, a next gen­er­a­tion BTK in­hibitor cur­rent­ly in a Phase Ib tri­al for lu­pus. — Nicole De­Feud­is

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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