Mammoth chief business officer and head of therapeutics Peter Nell

Bay­er jumps on board Mam­moth­'s ul­tra-small CRISPR tech with sights set first on the liv­er

Ger­man drug gi­ant Bay­er has looked to rein­vent it­self in re­cent years, mov­ing on from its past as a pri­mar­i­ly con­sumer health brand in­to one built around next-gen ther­a­pies. Now, Bay­er is tak­ing a fly­er on one of the chil­dren of CRISPR maven Jen­nifer Doud­na’s hal­lowed lab work­ing on tiny ver­sions of the gene edit­ing tech.

Bay­er will pay $40 mil­lion up­front and more than $1 bil­lion in po­ten­tial down­stream mile­stones for up to five in vi­vo gene edit­ing can­di­dates from Mam­moth Bio­sciences, a Doud­na lab spin­out de­vel­op­ing ul­tra-small en­zymes for eas­i­er pack­ag­ing and de­liv­ery in­to cells, the part­ners said Mon­day.

Mam­moth, which signed a sim­i­lar dis­cov­ery deal with Ver­tex late last year, is de­vel­op­ing CRISPR tools us­ing en­zymes rough­ly one-third the size of Cas9, the DNA scis­sors that helped make CRISPR fa­mous, to add or delete tar­get­ed genes through dou­ble-strand­ed breaks.

The com­pa­ny has de­vel­oped gene edit­ing sys­tems us­ing both Cas14 and Casɸ, among oth­er vari­ants, which are two small­er ver­sions that al­low for far greater flex­i­bil­i­ty in terms of de­liv­ery to tar­get tis­sues. That means Mam­moth’s team can push its ed­i­tors in­to small­er ade­no-as­so­ci­at­ed vi­ral vec­tors (AAV) but al­so in­to cer­tain lipid nanopar­ti­cles (LNP), the tech­nol­o­gy used to shut­tle the mR­NA-based Covid-19 vac­cines in­to cells, ac­cord­ing to ear­ly da­ta.

Ac­cord­ing to Mam­moth CSO Lu­cas Har­ring­ton, the pos­si­bil­i­ty of fit­ting more “pay­load” in­to de­liv­ery ve­hi­cles could help in­crease edit­ing ef­fi­cien­cy in cells, po­ten­tial­ly help­ing over­come cur­rent hur­dles for CRISPR tech.

For Bay­er, this deal comes a bit more than a year af­ter the Ger­man drug gi­ant, best known for its con­sumer health brand, ac­quired AskBio, an AAV-de­liv­ered gene ther­a­py com­pa­ny whose pick­up trum­pet­ed Bay­er’s move in­to next-gen ther­a­peu­tics and away from its staid phar­ma­ceu­ti­cal past. That biotech came aboard with ex­per­tise in build­ing a bet­ter AAV vec­tor, a puz­zle box tech­nol­o­gy with some safe­ty con­cerns that com­pa­nies all across the gene ther­a­py sec­tor are look­ing to solve.

Soon af­ter that pick­up, Bay­er an­nounced it would prop up a gene ther­a­py um­brel­la with­in its phar­ma­ceu­ti­cals busi­ness with the goal of adding a di­ver­si­fied set of modal­i­ties to dri­ve its next-gen push.

“Bay­er was re­al­ly look­ing for in vi­vo ap­pli­ca­tions, and I think our propo­si­tion is that small­er CRISPR sys­tems make this a per­fect com­bi­na­tion,” Pe­ter Nell, Mam­moth’s chief busi­ness of­fi­cer and head of ther­a­peu­tic strat­e­gy, told End­points News. “For us, it’s al­so how a com­pa­ny runs a busi­ness and what they know. You iden­ti­fy peo­ple who un­der­stand this and aren’t go­ing naive­ly in­to this.”

Al­though five tar­get ar­eas are part of the deal, the part­ners are on­ly say­ing now that the first tar­get on the list is the liv­er, a tis­sue most com­pa­nies be­lieve is the eas­i­est to hit. From there, the col­lab­o­ra­tion could run in any num­ber of di­rec­tions, which will be re­vealed at a lat­er date. Mean­while, Bay­er and Mam­moth plan to work to­geth­er on a nonex­clu­sive ba­sis lever­ag­ing the biotech’s ca­pa­bil­i­ties in ex vi­vo gene edit­ing, the same sort of tech­nol­o­gy used to craft off-the-shelf cell ther­a­pies.

Back in Oc­to­ber, Mam­moth signed a sim­i­lar li­cens­ing pact with Ver­tex worth $41 mil­lion up­front and $650 mil­lion in down­stream mile­stones for two ther­a­peu­tic ar­eas that weren’t dis­closed at the time of the an­nounce­ment. A month be­fore, Mam­moth an­nounced $195 mil­lion in new fund­ing (in­clud­ing a $150 mil­lion Se­ries D round and pre­vi­ous­ly unan­nounced $45 mil­lion Se­ries C).

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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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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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Benjamin Oakes, Scribe Therapeutics CEO

CEO of Doud­na spin­out: With­in five years, genome ed­i­tors will have a 're­al­ly big im­pact' on pa­tients' lives

“CRISPR-by-design” is the idea behind Scribe Therapeutics, a company spun out from Jennifer Doudna’s Nobel-winning lab that’s competing in a closely-tracked field of genome editor companies just starting to make their way to the clinic.

After nabbing $100 million last March for its Series B funding round, Scribe is taking a different tack from some of its competitors, crafting a new enzyme isolated from bacteria called CasX, which has now been tweaked extensively and may be targeted to a range of genome-related diseases, offering a plethora of therapeutic options.

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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.