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).

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,500+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

Endpoints Premium

Premium subscription required

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

Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,500+ biopharma pros reading Endpoints daily — and it's free.

Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,500+ biopharma pros reading Endpoints daily — and it's free.

Alon Seri-Levy, Sol-Gel Technologies CEO

Bridge­Bio com­pa­ny sells off rare dis­ease can­di­date to Gal­der­ma part­ner

Israeli biotech Sol-Gel Technologies announced Friday that it got its hands on a rare disease drug candidate from PellePharm for almost $75 million, amid claims that the drug has the potential to reach a $300 million market.

Execs said on a conference call Friday morning that patidegib, a hedgehog signaling pathway blocker, is being investigated to treat Gorlin syndrome, a rare genetic disorder that increases the risk of developing certain kinds of cancer such as basal cell skin cancer and medulloblastoma, a type of brain cancer. The disease affects around one in every 31,000 people, and an estimated 70,000 people worldwide.

Steve Harr, Sana Biotechnology CEO

Four years in, Sana gets first FDA go-ahead to bring can­cer treat­ment in­to the clin­ic

Sana Biotechnology is finally headed to the clinic.

Thursday afternoon, the biotech announced the FDA had cleared its application to start a clinical trial for its allogeneic, or “off-the-shelf,” CAR-T cell therapy targeting the antigen CD19 for patients with B-cell lymphomas and leukemias. Sana said its therapy, dubbed SC291, was designed to evade the immune system, which could help cell therapy produce a more durable response in patients, a concern that has followed such off-the-shelf therapies that use donor cells as opposed to a patient’s own cells.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 157,500+ biopharma pros reading Endpoints daily — and it's free.

Ying Huang, Legend CEO

J&J, Leg­end say Carvyk­ti beat stan­dard ther­a­py in ear­li­er-line blood can­cer

J&J and Legend Biotech’s next step in turning their CAR-T therapy Carvykti into a potential megablockbuster has succeeded, the companies said Friday.

Carvykti achieved the primary endpoint — progression-free survival — in an open-label Phase III study testing the treatment in second- to fourth-line multiple myeloma patients. The CARTITUDE-4 trial, for which there aren’t any hard data yet, represents the biggest development for Carvykti’s ability to compete with Bristol Myers Squibb’s Abecma since its approval last February.

CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.