Michael Yang leads list of moves at Vi­a­Cyte, suc­ceed­ing Paul Laikind as CEO; Qasim Rizvi named CEO of Take­da part­ner KSQ

Michael Yang

→ The winds of change are fresh­en­ing off the coast in San Diego for stem cell biotech Vi­a­Cyte, which has ap­point­ed Michael Yang as pres­i­dent and CEO. Yang re­places Paul Laikind, who re­tired as Vi­a­Cyte’s CEO in Sep­tem­ber af­ter eight years at the helm, and leaves his pre­vi­ous gig as chief com­mer­cial of­fi­cer of Aca­dia Phar­ma­ceu­ti­cals ef­fec­tive to­day. Pri­or to his near­ly four years at Aca­dia, Yang was Janssen’s pres­i­dent in charge of their US im­munol­o­gy busi­ness, and be­fore that, he was the J&J sub­sidiary’s CNS pres­i­dent.

Else­where at Vi­a­Cyte, Brit­tany Bradrick has been pro­mot­ed to COO and CFO, six months af­ter she orig­i­nal­ly jumped on board as CFO of the biotech from In­sulet Cor­po­ra­tion. Fur­ther­more, Vi­a­Cyte has cho­sen Steve White as their chief tech­nol­o­gy of­fi­cer. White, a GSK and Mer­ck KGaA vet, was al­so an ex­ec at Bax­ter, where he just wrapped his 15-year tenure last year as VP, glob­al R&D.

The moves come as Vi­a­Cyte is ramp­ing up cell man­u­fac­tur­ing ca­pa­bil­i­ties for PEC-Di­rect, PEC-En­cap (in part­ner­ship with Gore) and PEC-QT (in part­ner­ship with CRISPR Ther­a­peu­tics).

Char­maine Lykins

→ As Yang ex­its, the op­por­tu­ni­ty opens for Char­maine Lykins to take over com­mer­cial re­spon­si­bil­i­ties at Aca­dia un­der the ti­tle of SVP, glob­al prod­uct plan­ning and chief mar­ket­ing of­fi­cer. Lykins de­vot­ed al­most 15 years to Eli Lil­ly in var­i­ous sales and mar­ket­ing roles, and she’s al­so been glob­al VP of mar­ket­ing for Lund­beck. Lykins joined Aca­dia three years ago as VP of mar­ket­ing and was pro­mot­ed to SVP in 2019.

Aca­dia has al­so pro­mot­ed Aman­da Mor­gan to SVP, chief rev­enue and cus­tomer of­fi­cer. Mor­gan, who owns ex­pe­ri­ence with such com­pa­nies as Bax­al­ta and Bax­ter, be­gan her Aca­dia ca­reer in 2017 as VP, sales and mar­ket ac­cess.

Qasim Rizvi

Join­ing forces at the start of this year with Take­da on a $100 mil­lion part­ner­ship to de­vel­op im­muno-on­col­o­gy can­di­dates, KSQ Ther­a­peu­tics has found a top ex­ec as well, bring­ing on Qasim Rizvi as CEO. A 12-year vet of Genen­tech who was fran­chise head of Tecen­triq, Rizvi makes the move af­ter a two-year run at Kiniksa — first as SVP, op­er­a­tions, then as chief com­mer­cial of­fi­cer. Pri­or to Genen­tech, he held sales, mar­ket­ing and busi­ness de­vel­op­ment ti­tles at No­vo Nordisk and Eli Lil­ly.

In an End­points News in­ter­view, KSQ CSO Frank Stegmeier out­lined that the Take­da deal is a “great val­i­da­tion” of the biotech’s CRISPR screen­ing plat­form, dubbed CRISPRomics. “I think what I’m par­tic­u­lar­ly ex­cit­ed about is it was very clear from the be­gin­ning of our dis­cus­sions, all the way through the end of the dis­cus­sions [with Take­da], that we have a very aligned ex­cite­ment about the po­ten­tial of next gen­er­a­tion on­col­o­gy drugs for can­cer pa­tients,” Stegmeier said.

Damien McDe­vitt

As­pen Neu­ro­science, a Parkin­son’s biotech out of San Diego that scored a $70 mil­lion Se­ries A as the in­evitabil­i­ty of the Covid-19 pan­dem­ic took hold in April, has tapped Damien McDe­vitt to lead the way as CEO. McDe­vitt gets an­oth­er op­por­tu­ni­ty to take con­trol of a com­pa­ny af­ter re­plac­ing Paula Soteropou­los at Akcea, which spun out from Io­n­is in 2017 on­ly to see it­self fold­ed back in­to Io­n­is to­ward the end of last sum­mer. McDe­vitt, who will al­so be a mem­ber of an As­pen board that’s chaired by Fa­heem Has­nain, was with Glax­o­SmithK­line for 21 years be­fore be­com­ing an ex­ec at Aca­dia and then Io­n­is/Akcea.

Shalin Shah

→ Fo­cused on testos­terone de­fi­cien­cy ther­a­pies, Raleigh, NC-based Mar­ius Phar­ma­ceu­ti­cals has de­cid­ed on the co-CEO route with Hi­man­shu Shah and Shalin Shah as the com­pa­ny seeks ap­proval for Kyza­trex, its drug for pri­ma­ry and sec­ondary hy­pog­o­nadism. Hi­man­shu Shah will re­main chair­man of the board in ad­di­tion to his new du­ties, while Shalin Shah takes the co-CEO reins af­ter serv­ing four years as Mar­ius’ CFO and EVP of strat­e­gy. Out­go­ing CEO Om Dhin­gra now shifts in­to the vice chair­man post.

→ Cam­bridge, MA-based bio­mol­e­c­u­lar con­den­sate biotech Faze Med­i­cines has tapped Philip Vick­ers as their CEO, a month af­ter tak­ing the tarp off an $81 mil­lion Se­ries A round. Vick­ers, an ex-re­search di­rec­tor at Mer­ck and Pfiz­er who was the glob­al head of R&D at Shire from 2013-17, had spent the last three-plus years at the helm of North­ern Bi­o­log­ics.

Ra­mi El­ghan­dour

→ Gaithers­burg, MD-based start­up Ar­cel­lx, which de­buted in Oc­to­ber 2019 and re­vealed BC­MA CAR-T da­ta at ASH, has tapped Ra­mi El­ghan­dour as chair­man and CEO. From 2012-19, El­ghan­dour was pres­i­dent and CEO of med­ical de­vice com­pa­ny Nevro, the mak­er of the Sen­za spinal cord stim­u­la­tion (SCS) sys­tem. He was al­so an in­vestor with John­son & John­son De­vel­op­ment Cor­po­ra­tion be­fore helm­ing Nevro.

→ Pre­ci­sion on­col­o­gy-fo­cused On­coD­NA has re­cruit­ed Bernard Courtieu as CEO, tak­ing over for Ed­uar­do Bra­vo who has been in­ter­im CEO since Ju­ly 2020. Courtieu joins with ex­pe­ri­ence as CEO of In­te­gra­Gen, a com­pa­ny he joined in 2007. Pri­or to that, Courtieu served at Gem­i­ni Con­sult­ing‘s life sci­ences di­vi­sion in France.

Mark Day

Mark Day is tak­ing on the role of CBO at Cam­bridge, MA-based Cog­ni­to Ther­a­peu­tics, just a cou­ple weeks re­moved from the break­through des­ig­na­tion they re­ceived from the FDA for their lead prod­uct, a neu­rostim­u­la­tion de­vice used to quell the symp­toms of Alzheimer’s. Day’s Big Phar­ma con­nec­tions date back to when he was prin­ci­pal lead for CNS dis­cov­ery at GSK. From there, he ven­tured off to Pfiz­er and Ab­bott, and then land­ed at Bris­tol My­ers Squibb, where he was their glob­al lead for CNS busi­ness de­vel­op­ment. He just spent a lit­tle less than a year as VP and head of the Mc­Quade Cen­ter for Strate­gic Re­search and De­vel­op­ment at Ot­su­ka.

→ Few biotechs are on a more rapid rise than Texas’ own Taysha Gene Ther­a­pies, launch­ing last April and then rais­ing rough­ly $181 mil­lion in its Sep­tem­ber IPO. This week, RA Ses­sions’ team has in­tro­duced two new ex­ecs with chief le­gal of­fi­cer and cor­po­rate sec­re­tary Tim­o­thy Douros and chief peo­ple of­fi­cer Tra­cy Porter. Douros leaves blue­bird bio, where he was their SVP, gen­er­al coun­sel, and he al­so held a se­ries of le­gal roles from 2002-15 at Cu­bist Phar­ma­ceu­ti­cals. Porter was pre­vi­ous­ly the head of hu­man re­sources at Au­dentes.

Sel­wyn Ho

Sel­wyn Ho has been named CBO of Con­nect Bio­phar­ma, which racked up $115 mil­lion in a Se­ries C last Au­gust for their pipeline of im­mune mod­u­la­tors. Ho, an ad­vi­sor to Con­nect Bio­phar­ma for the past year, has pre­vi­ous­ly been head of mar­ket ac­cess and head of strate­gic mar­ket­ing at Der­mi­ra and has held a hand­ful of ex­ec­u­tive roles at UCB. He al­so brings lead­er­ship ex­pe­ri­ence from Al­ler­gan, No­var­tis and As­traZeneca.

→ Fo­cused on der­ma­to­log­ic dis­eases, Wood­cliff Lake, NJ-based Tim­ber Phar­ma­ceu­ti­cals has wel­comed Big Phar­ma vet Alan Mendel­sohn as CMO. Mendel­sohn had been Sun Phar­ma’s as­so­ciate VP of der­ma­tol­ogy med­ical af­fairs the last four-plus years, and be­fore that, he was a se­nior di­rec­tor at Pfiz­er lead­ing the US rheuma­tol­ogy med­ical af­fairs team. Mendel­sohn al­so had a lengthy tenure at Cen­to­cor/J&J, help­ing de­vel­op the block­buster pso­ri­a­sis drug Ste­lara and even­tu­al­ly ris­ing to se­nior di­rec­tor, im­munol­o­gy R&D.

Tr­isha Mil­li­can

Ang­ie You-helmed Amu­nix, which banked $73 mil­lion in a Se­ries A last March to pro­pel their T cell en­gage­ment plat­form, has hand­ed the CFO reins to Zee­shan Mer­chant. He heads over to Amu­nix from his post at Mor­gan Stan­ley as ex­ec­u­tive di­rec­tor in the health­care in­vest­ment bank­ing group. Else­where at the South San Fran­cis­co biotech, Metacrine CFO and for­mer Ser­agon SVP of fi­nance Tr­isha Mil­li­can is now a mem­ber of Amu­nix’s board of di­rec­tors.

Gath­er­ing up €32 mil­lion ($36.2 mil­lion) in a Se­ries B last Ju­ly to chal­lenge Alex­ion’s An­dexxa, Dutch an­ti­co­ag­u­la­tion biotech Var­mX has en­list­ed Ger­ard Short as CMO. Short, a No­var­tis alum who was glob­al de­vel­op­ment team leader at Gen­zyme for Pompe dis­ease, pre­vi­ous­ly held the po­si­tion of SVP, med­ical sci­ence at Free­line Ther­a­peu­tics af­ter jump­ing on board late in 2015 as SVP, clin­i­cal and reg­u­la­to­ry.

Kirk Raab

→ For­mer Genen­tech CEO Kirk Raab passed away Jan. 21 at the age of 85 af­ter a bat­tle with Covid-19 ex­ac­er­bat­ed his al­ready ill health. In 1985, Raab be­came pres­i­dent and COO of Genen­tech, ris­ing to CEO in 1990 and stay­ing at the helm un­til his ouster in 1995. Raab be­gan his long ca­reer in the in­dus­try as a sales­man for Pfiz­er in 1959.

→ Mi­cro­bio­me-fo­cused Sec­ond Genome has named Su­san­nah Cantrell as CBO and Preeti Lal as CSO. Cantrell hops aboard from Tri­ci­da, where she most re­cent­ly served as EVP and CCO. Pri­or to that, Cantrell was serv­ing in roles at Gilead and Genen­tech/Roche. Mean­while, Lal joins the com­pa­ny with ex­pe­ri­ence from her times at Roche Di­ag­nos­tics, Gilead Sci­ences, and Genen­tech among oth­ers.

Made­lyn Caltabi­ano

→ An­oth­er Peer Re­view, an­oth­er ap­point­ment at No­vavax, which re­port­ed yes­ter­day that their Covid-19 vac­cine is 89.3% ef­fec­tive in a Phase III UK tri­al. This time at the Gaithers­burg, MD biotech, Made­lyn Caltabi­ano has jumped on board in the new­ly-formed role of SVP, glob­al pro­gram man­age­ment. Caltabi­ano is the for­mer SVP, glob­al project and al­liance man­age­ment at Mer­ck Re­search Lab­o­ra­to­ries and a long­time ex­ec at GSK from 1999-2013. She was most re­cent­ly the de­vel­op­ment lead for Nex­Eos Di­ag­nos­tics.

Last week, No­vavax lament­ed that Op­er­a­tion Warp Speed was at fault for man­u­fac­tur­ing-re­lat­ed de­lays in their Covid-19 vac­cine tri­al, al­low­ing J&J’s vac­cine can­di­date to cut in line ahead of No­vavax with con­tract man­u­fac­tur­er Emer­gent BioSo­lu­tions.

Er­ic Pas­tor

Spirovant, a gene ther­a­py play­er from the Vivek Ra­maswamy fam­i­ly of Vants sold to Sum­it­o­mo Dainip­pon and fo­cused on pul­monary dis­eases such as cys­tic fi­bro­sis, has brought in Er­ic Pas­tor as SVP of tech­nol­o­gy de­vel­op­ment and op­er­a­tions and Maria Lim­beris as VP of re­search. Pas­tor, the ex-head of gene ther­a­py de­vel­op­ment at Sanofi, comes from his VP, CMC op­er­a­tions po­si­tion at Vedere Bio. Lim­beris hails from the Uni­ver­si­ty of Penn­syl­va­nia, where she was the di­rec­tor, Pro­gram of Ex­cel­lence in Cys­tic Fi­bro­sis at the Jim Wil­son-led Or­phan Dis­ease Cen­ter.

→ Cana­di­an biotech Notch Ther­a­peu­tics, fo­cused on cre­at­ing al­lo­gene­ic T cell ther­a­pies from re­new­able stem cell sources, scored a new CEO (David Main) and CFO (Kam­ran Alam) last year. Now Chris Bond has been in­stalled as Notch’s SVP, pre­clin­i­cal and trans­la­tion­al sci­ences. The com­pa­ny is right up Bond’s al­ley; he was re­spon­si­ble for de­vel­op­ment of al­lo­gene­ic cell ther­a­py plat­forms as VP, cel­lu­lar en­gi­neer­ing at Kite. He’d al­so been se­nior di­rec­tor, lead dis­cov­ery at Juno pri­or to his new gig at Notch, which has of­fices in Toron­to and Van­cou­ver.

Bren­dan Luu

→ Af­ter start­ing 2021 with a new ex­ec in trans­la­tion­al vi­rol­o­gy, Ab­b­Vie’s hep C part­ner Enan­ta Phar­ma­ceu­ti­cals has made Bren­dan Luu SVP, busi­ness de­vel­op­ment. Luu makes the tran­si­tion from 17 years at Mer­ck KGaA, climb­ing up the lad­der to be­come the Ger­man phar­ma’s VP and head of on­col­o­gy busi­ness de­vel­op­ment the last three years.

→ Bris­tol My­ers Squibb alum Ronald Mar­cus has joined Steve Paul’s troops at Karuna Ther­a­peu­tics as SVP of med­ical. Be­fore mak­ing the leap to Karuna, which scrapped its plans to make KAR-020 a non-opi­oid treat­ment for pain last sum­mer af­ter Phase Ib da­ta showed no ben­e­fit, Mar­cus was CMO at Ter­ran Bio­sciences and the VP of R&D at Su­per­nus. Mar­cus was in Bris­tol My­ers’ neu­ro­science di­vi­sion from 1991-2013, even­tu­al­ly serv­ing as the phar­ma gi­ant’s ex­ec­u­tive di­rec­tor, neu­ro­science glob­al clin­i­cal re­search.

→ RNA-cen­tered Green­Light Bio­sciences out of Boston, which locked in a $102 mil­lion Se­ries D last June for mR­NA man­u­fac­tur­ing and Covid-19 vac­cine de­vel­op­ment, has called up­on Thomas Cramp­ton to be SVP for cor­po­rate af­fairs. Cramp­ton is a for­mer New York Times writer and most re­cent­ly the chief mar­ket­ing and com­mu­ni­ca­tions of­fi­cer of The Com­mons Pro­ject Foun­da­tion.

Mar­i­anne De Backer

Nor­bert Bischof­berg­er has as­sem­bled heavy hit­ters on his board of di­rec­tors at Kro­nos Bio, and this week is no dif­fer­ent with Bay­er BD chief Mar­i­anne De Backer. She joins such lu­mi­nar­ies on the board as Arie Bellde­grun and Otel­lo Stam­pac­chia at Kro­nos, which raised a whop­ping $287.5 mil­lion in its IPO last fall.

→ A new chap­ter for Jef­frey Lei­den be­gins at Oz Azam’s T cell im­munother­a­py out­fit Tmu­ni­ty as chair­man of the board. Lei­den was the pres­i­dent and CEO at Ver­tex from 2012-20 and is cur­rent­ly their ex­ec­u­tive chair­man.

→ Woburn, MA-based IM Ther­a­peu­tics (short for Im­munoMol­e­c­u­lar Ther­a­peu­tics) has elect­ed Jonathan Rig­by to the board of di­rec­tors. Rig­by has helmed Im­mune Reg­u­la­tion, which made Au­rinia CEO Pe­ter Green­leaf chair­man a month ago, since last April.

Bon­nie Bassler

Reza­fun­gin mak­er Cidara has added Bon­nie Bassler and Carin Canale-Theak­ston to the board of di­rec­tors. Bassler, the chair of the mol­e­c­u­lar bi­ol­o­gy de­part­ment at Prince­ton, joined the board at Roy­al­ty Phar­ma last sum­mer and is a board mem­ber at Re­gen­eron and Kalei­do Bio­sciences. Canale-Theak­ston found­ed and is CEO of life sci­ence com­mu­ni­ca­tions group Canale Com­mu­ni­ca­tions.

→ Wa­ter­town, MA al­lo­gene­ic cell ther­a­py biotech Platelet­Bio has elect­ed Steven Altschuler chair­man of the board. Altschuler, who co-found­ed Spark, is al­so chair­man at 89bio and oph­thalmic start­up As­cle­piX Ther­a­peu­tics.

→ Seat­tle CAR-T up­start Umo­ja Bio­phar­ma, which emerged in No­vem­ber with a $53 mil­lion Se­ries A, has named Robert Glass­man to the board of di­rec­tors. Glass­man, Or­biMed’s ven­ture part­ner in pub­lic eq­ui­ty, is al­so on the board at Phar­varis and Tarus Ther­a­peu­tics.

→ Bio­engi­neer­ing com­pa­ny Biostage has pulled in Her­man Sanchez to its board of di­rec­tors. Cur­rent­ly, Sanchez is a se­nior part­ner at Trin­i­ty Life Sci­ences‘ strat­e­gy con­sult­ing busi­ness.

Tal­is Bio­med­ical has wel­comed Melis­sa Gilliam to its board of di­rec­tors. Gilliam is a pro­fes­sor of health jus­tice and vice provost at the Uni­ver­si­ty of Chica­go.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; 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 still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

As Co­di­ak en­ters 'next phase of growth,' Jen­nifer Whel­er steps in­to CMO role; Mark Tim­ney lines up his sec­ond CEO op­por­tu­ni­ty af­ter Pur­due Phar­ma reign

Most CMOs don’t start out as art students. But after getting her bachelor’s in art history from Princeton University, Jennifer Wheler decided to go after an MD from Cornell, kicking off a decades-long career in biopharma. Her next move? Leading the team at Codiak BioSciences to deliver therapeutics using a form of cellular postal service called exosomes.

“I was captivated by the science and the potential for exosomes to have transformational impact in cancer and other disease areas,” the incoming CMO told Endpoints News via email. “Codiak has a potent combination of great science and wonderfully experienced and kind people — it doesn’t get much better.”

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.

The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).