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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; 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.

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Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Radek Spisek, Sotio CEO (Cellestia)

A qui­et Czech biotech bags $315M to dri­ve its blos­som­ing can­cer pipeline through the clin­ic

In the rather insular world of biotech, most innovation inevitably comes from a cluster of R&D hubs — Cambridge, San Francisco, etc. But sometimes success stories sprout from rocky soil, which is most certainly the case with Prague-based Sotio Biotech and its suddenly jam-packed pipeline of cancer drugs.

After years in quiet development, Sotio now has $315 million in new funds to play with from parent company PPF Group, an investment group founded in the Czech Republic, as the biotech looks to advance its growing pipeline through early- and mid-stage trials.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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