Richard DiMarchi, MBX Biosciences

Suc­cess­ful sci­en­tist-in­vestor al­lies team up again — this time for a stab at rare meta­bol­ic dis­ease

A sci­en­tist-ex­ec­u­tive team that, over the last decade, sold one di­a­betes biotech to Roche for up-to $537 mil­lion and two oth­ers to No­vo Nordisk for undis­closed sums are back with a new com­pa­ny.

Kent Hawry­luk

Uni­ver­si­ty of In­di­ana bi­ol­o­gist Richard Di­Marchi and se­r­i­al en­tre­pre­neur Kent Hawry­luk have launched MBX Bio­sciences, rais­ing $36.4 mil­lion in a Fra­zier Health­care Part­ners-led round to get it start­ed. For now, the pair are keep­ing their dis­eases and drug tar­gets close to the vest, say­ing on­ly that MBX will de­vel­op the same kind of mol­e­cules they de­vel­oped in pre­vi­ous com­pa­nies — large pep­tide ther­a­peu­tics — but with a fo­cus on rare en­docrine dis­or­ders, as op­posed to di­a­betes.

“There’s just a very rich his­to­ry in the field of pep­tides as mirac­u­lous med­i­cines, start­ing with in­sulin and glucagon and ex­tend­ing through a host of more re­cent reg­is­tra­tions,” Di­Marchi told End­points News. “So it is rare en­docrine dis­eases, it is large mol­e­cules, it is re­lat­ed to things I’ve done his­tor­i­cal­ly.”

The com­pa­ny is based both in the 100,000-per­son city of Carmel, In­di­ana, out­side In­di­anapo­lis, where there are 6 full-time em­ploy­ees, and at Di­Marchi’s lab in Bloom­ing­ton, where sev­er­al full-time sci­en­tists work un­der a spon­sored re­search arrange­ment. “Al­though these days, it feels like we’re based on Zoom,” Hawry­luk told End­points.

Be­fore found­ing his first biotech with Hawry­luk, Di­Marchi led the en­docrine di­vi­sion for two decades at Eli Lil­ly, where he helped de­vel­oped Hu­ma­log, Eli Lil­ly’s best-sell­ing in­sulin. In 2005, he and Hawry­luk found­ed Mar­ca­dia to de­vel­op a sol­u­ble and sta­ble ana­log for the meta­bol­ic hor­mone glucagon, a po­ten­tial treat­ment for di­a­betes and obe­si­ty. Six years lat­er, Roche snapped up the tiny biotech for $287 mil­lion in cash and up-to $250 mil­lion in mile­stones.

Roche end­ed up re­tir­ing the pro­gram short­ly there­after when they left meta­bol­ic dis­ease al­to­geth­er, Di­Marchi said, but he cred­its their work with es­tab­lish­ing the frame­work for oth­ers to en­ter the field. He specif­i­cal­ly named Zealand Phar­ma, which this year filed an NDA for dasiglucagon. He al­so named their work on a hor­mone that can hit both GIP and GLP-1 re­cep­tors, po­ten­tial­ly al­low­ing for a stronger drug for obe­si­ty, di­a­betes and NASH.

“It was our di­rec­tion that I be­lieve led oth­ers,” Di­Marchi said. “There’s prob­a­bly 2 dozen of those clin­i­cal can­di­dates cur­rent­ly be­ing ad­vanced across the globe by more than half a dozen phar­ma­ceu­ti­cal com­pa­nies.”

Lat­er, Di­Marchi and Hawry­luk launched MB2 and Cal­ib­ri­um in 2012 and 2014 to de­vel­op oth­er pro­tein-based di­a­betes drug and sold both to No­vo Nordisk in 2015 for an undis­closed sum. The progress that had been made in di­a­betes, Di­Marchi said, dic­tat­ed that they fo­cus else­where.

That work be­gan last Ju­ly, when Di­Marchi re­turned to a full time role at In­di­ana af­ter a few years work­ing part-time with No­vo Nordisk. He start­ed work­ing on syn­the­siz­ing new mol­e­cules and de­vel­op­ing in vit­ro as­says. He re­cruit­ed back Hawry­luk, who was then CBO of the RNA biotech Avid­i­ty Bio­sciences and who came over in Jan­u­ary af­ter se­cur­ing a $100 mil­lion crossover round.

There’s no time­line yet for the clin­ic or drug tar­gets. For now, Hawry­luk said, they’re look­ing for a CMO and en­joy­ing hav­ing an old band back to­geth­er.

“The jam ses­sions are a lot of fun,” he said.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Warren Huff, Reata CEO

Rea­ta sug­gests Friedre­ich's atax­ia pro­gram could be de­layed, send­ing stock plung­ing

Reata Pharmaceuticals $RETA made waves last October when its drug omaveloxolone produced positive trial results in treating a rare neurological disorder, but the candidate’s path forward became much murkier Monday.

In a report of quarterly earnings, the biotech divulged that the FDA is considering delaying omaveloxolone’s NDA pending completion of a second trial. That could push back approval by at least a year given that the target population, individuals with Friedreich’s ataxia, is limited and progression of the hard-to-treat illness is notoriously slow. The Covid-19 pandemic would also hinder Reata’s ability to complete an additional trial.

Vi­da Ven­tures co-leads Dyne's $115M megaround for next-gen oli­go ther­a­pies aimed square­ly at mus­cles

Dyne Therapeutics started out last April with a modest $50 million to mine targeted muscle disease therapies from its in-house conjugate technology. The biotech has now convinced more investors that it’s got gems on its hands, closing $115 million in fresh financing to push its next-gen oligonucleotide drugs into the clinic.

Vida Ventures and Surveyor Capital led the round, joined by a group of other new backers including Wellington Management Company, Logos Capital and Franklin Templeton.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Eli Lil­ly teams with Pieris on HER2+ tu­mors; Op­di­vo + Yer­voy best chemo in mesothe­lioma

Despite the FDA putting a partial clinical hold on its lead program only a few weeks ago, Boston-based Pieris Pharmaceuticals is plowing forward with a new collaboration.

Pieris will work with Eli Lilly to further advance studies on PRS-343, a 4-1BB/HER2 bispecific for HER2-positive tumors, in combination with the latter’s ramucirumab and paclitaxel for the second-line treatment of patients with HER2-positive gastric cancer in a single-arm, Phase II study.

In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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