CEO Gregory Flesher (Reneo)

No­vo and Abing­worth back a $95M Se­ries B for Re­neo Phar­ma­ceu­ti­cals, which aims to treat dis­eases of the mi­to­chon­dria

Less than two years af­ter its com­ing-out par­ty with a $50 mil­lion launch round, Re­neo Phar­ma­ceu­ti­cals re­turned to the ven­ture well and reeled in some high-pro­file back­ers.

The San Diego-based biotech an­nounced the clos­ing of its $95 mil­lion Se­ries B on Wednes­day morn­ing, co-led by No­vo Ven­tures and Abing­worth, to sup­port its re­search in­to ge­net­ic mi­to­chon­dr­i­al dis­eases. Wednes­day’s cash will give the com­pa­ny a three-year run­way, tak­ing them through the com­ple­tion of three ear­ly- to mid-stage tri­als for their lead pro­gram, REN001.

Gre­go­ry Flesh­er, the for­mer CEO of Novus Ther­a­peu­tics, is al­so join­ing Re­neo to steer the ship.

Re­turn­ing in­vestors in­clud­ed New En­ter­prise As­so­ci­ates, River­Vest Ven­ture Part­ners, Pap­pas Cap­i­tal and Lund­beck­fonden Ven­tures. In­vest­ing for the first time are Rock Springs Cap­i­tal, Ais­ling Cap­i­tal, Amzak Health and oth­ers.

REN001 is a PPAR-delta ag­o­nist be­ing stud­ied in pri­ma­ry mi­to­chon­dr­i­al my­opathies, fat­ty acid ox­i­da­tion dis­or­ders and McAr­dle dis­ease. All three con­di­tions are re­lat­ed to dif­fer­ent parts of mi­to­chon­dr­i­al func­tion, as pa­tients strug­gle with me­tab­o­liz­ing dif­fer­ent parts of food. Giv­en that mi­to­chon­dria pro­duce the en­er­gy need­ed for cells to thrive, the end re­sult is fa­tigue and hav­ing trou­ble do­ing any sort of phys­i­cal ac­tiv­i­ty for longer than a few min­utes.

Some­times, the en­er­gy lev­els are so low that the stress placed on the mus­cu­loskele­tal sys­tem and vi­tal or­gans can lead to oth­er dis­eases.

Flesh­er tells End­points News that REN001, a once-dai­ly pill, has the abil­i­ty to help cells ex­press cer­tain genes with­in the mi­to­chon­dria that in­crease a pa­tient’s me­tab­o­lism. He em­pha­sized that the drug is not a gene ther­a­py, and the ul­ti­mate goal is to both in­crease the amount of mi­to­chon­dria that are re­plen­ished once the old ones die off, in ad­di­tion to boost­ing en­zyme pro­duc­tion with­in the en­er­gy cen­ters.

Though the mi­to­chon­dria are smart and can adapt to some short­com­ings ini­tial­ly, like us­ing dif­fer­ent start­ing points if a cer­tain area is de­fi­cient, ATP pro­duc­tion ends up ta­per­ing off af­ter those first few min­utes be­cause there’s not enough build­ing blocks. In FAOD, the pa­tients can’t process fat in­to fat­ty acids and then ATP, for ex­am­ple, and in McAr­dle dis­ease, they can’t process glyco­gen in­to glu­cose, Flesh­er said.

“Any drug prod­uct or ther­a­peu­tic ap­proach that can ramp up one or more of the in­bound paths to cre­at­ing ATP can be re­al­ly help­ful for these pa­tients, and that’s why we’re ex­plor­ing mul­ti­ple sub­groups of pa­tients that can­not pro­duce ATP,” Flesh­er said.

Re­neo is plan­ning on start­ing a dou­ble-blind­ed, place­bo-con­trolled Phase II in PMM ear­ly next year with the goal of en­rolling about 200 pa­tients. The pri­ma­ry end­point will in­volve some sort of walk test, mea­sur­ing how far and how long pa­tients can trav­el by foot, which has a di­rect cor­re­la­tion with dai­ly qual­i­ty of life. Da­ta will like­ly read out to­ward the end of the Se­ries B run­way, Flesh­er said.

The com­pa­ny is al­so run­ning two Phase Ib tri­als in FAOD and McAr­dle dis­ease, which will come out with da­ta some­time in late 2021 or ear­ly 2022.

“To have a strong in­vestor syn­di­cate … it re­al­ly is, I’d prob­a­bly not say stamp of ap­proval, but more a recog­ni­tion that the ther­a­peu­tic ap­proach, the modal­i­ty and the pa­tient pop­u­la­tion is im­por­tant, and peo­ple would like to see a prod­uct ap­proved,” Flesh­er said.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, 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.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

The IPO 4-1-1: Four fil­ings, a pric­ing and a with­draw­al head­line this week's Nas­daq ac­tion as raise ap­proach­es $7.5B

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

Another week, another horde of biotechs is doing the Nasdaq dance.

This week saw four companies file their SEC paperwork ahead of expected debuts, another hit Nasdaq on Friday and a sixth formally withdrew its bid to go public. Aerovate Therapeutics, Ocean Biomedical and Acumen Pharmaceuticals all penciled in initial raises of $100 million, while Dermata Therapeutics is estimating a modest $18 million raise.

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Keiichi Fukuda, Heartseed CEO

Fresh off $598M deal with No­vo Nordisk, a Japan­ese stem cell com­pa­ny is on its way to the clin­ic with a dif­fer­ent ap­proach to treat­ing heart fail­ure

A common approach to treating heart failure with induced pluripotent stem cells involves grafting sheets of cells onto the surface of the heart to improve vascularization and blood flow. It’s the easiest method of transplantation — but you run the risk of not making an electrical connection with the heart and the cells not synchronizing with the patient’s heart muscle.

So what if you could inject spherical clusters of heart cells directly into the heart muscle wall? For Heartseed, that’s now the $37 million question.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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