Caroline Godfrey, PepGen CEO

RA Cap­i­tal backs Ox­ford spin­out Pep­Gen with a $45M Se­ries A, seek­ing to treat Duchenne and oth­er sim­i­lar dis­eases

Pe­ter Kolchin­sky

Less than two months af­ter Pe­ter Kolchin­sky and Raj Shah an­nounced a new $461 mil­lion fund, the part­ners at RA Cap­i­tal Man­age­ment ap­pear to have made an­oth­er in­vest­ment.

RA is head­lin­ing a $45 mil­lion Se­ries A round for the Ox­ford, UK-based biotech Pep­Gen, which fo­cus­es on se­vere neu­ro­mus­cu­lar dis­eases like Duchenne mus­cu­lar dy­s­tro­phy. The com­pa­ny will use the fund­ing to ad­vance a slate of what they’re call­ing “cell-pen­e­trat­ing” pep­tides — com­bined with some of their pro­pri­etary con­ju­gates — in­to the clin­ic.

“We be­lieve Pep­Gen’s PP­MOs have enor­mous po­ten­tial for the treat­ment of se­vere neu­ro­mus­cu­lar and car­diac dis­or­ders,” RA ven­ture part­ner Ramin Farzaneh-Far told End­points News in an email. “The fi­nanc­ing re­flects our con­fi­dence, and that of our syn­di­cate part­ners, in the tech­nol­o­gy.”

Ox­ford Sci­ences In­no­va­tion, Pep­Gen’s seed in­vestor, al­so par­tic­i­pat­ed in the round, as well as the Uni­ver­si­ty of Ox­ford and Cure­Duchenne Ven­tures. Wednes­day’s cash will al­so al­low Pep­Gen to build out a cor­po­rate team in the new Boston head­quar­ters and ex­pand the R&D hub in the UK, Farzaneh-Far said.

Raj Shah

The move from RA comes short­ly af­ter Shah told End­points News in Oc­to­ber that the cash for its Nexus I life sci­ences fund, rough­ly $300 mil­lion, was churned through at a rel­a­tive­ly “rapid” pace. In just 15 months of in­vest­ment, RA had spent about 80% of their fund, which prompt­ed the Nexus II raise.

Though the new fund built off large­ly the first, the cash pools re­main sep­a­rate. Farzaneh-Far de­clined to com­ment to which Nexus fund Wednes­day’s in­vest­ment be­longed.

Pep­Gen it­self was spun out of Ox­ford in 2018 in or­der to fur­ther de­vel­op the pep­tides at the heart of its re­search. The biotech says that the cell-pen­e­trat­ing na­ture of the pep­tides, when con­ju­gat­ed with phos­pho­ro­di­ami­date mor­pholi­no oligomers or PP­MOs, could al­low for en­hanced de­liv­ery of oligonu­cleotides to key tis­sues, while al­so im­prov­ing safe­ty com­pared to oth­er med­i­cines.

Specif­i­cal­ly, Pep­Gen is hop­ing to leapfrog the ex­on-skip­ping ap­proach­es al­ready avail­able in or­der to re­store dy­s­trophin ex­pres­sion in DMD pa­tients, CEO and co-founder Car­o­line God­frey said in a state­ment.

One of the ar­eas where Pep­Gen says its pro­grams are ben­e­fi­cial is in the car­dio­vas­cu­lar co­mor­bidi­ties that of­ten ac­com­pa­ny DMD. Be­cause the pep­tides can pen­e­trate cells, the com­pa­ny says its drug can­di­dates strong­ly dis­trib­ute to car­diac tis­sue.

“With the re­cent ap­provals of treat­ments that gen­er­ate small in­creas­es in dy­s­trophin in skele­tal mus­cle, pa­tients may be am­bu­lat­ing and liv­ing longer, but this in turn is ex­pect­ed to shift the bur­den of mor­bid­i­ty and mor­tal­i­ty to­wards an epi­dem­ic of heart dis­ease, which is not ad­e­quate­ly ad­dressed by cur­rent DMD ther­a­pies,” Farzaneh-Far said in an ear­li­er state­ment.

This past sum­mer, the FDA green-lit the third DMD drug when Japan­ese de­vel­op­er NS Phar­ma gained an ac­cel­er­at­ed ap­proval for vil­to­larsen. That fol­lowed a wild back-and-forth be­tween reg­u­la­tors and Sarep­ta, who orig­i­nal­ly re­ject­ed their DMD can­di­date in Au­gust 2019 but re­versed course lat­er that year.

The agency, how­ev­er, still doesn’t have full ef­fi­ca­cy da­ta on any of the three ap­proved DMD drugs, as the OKs were all based on the same dis­ease bio­mark­er.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Andrew Phillips, Nexo Therapeutics CEO

Scoop: Ver­sant, NEA launch new biotech helmed by ex-CEO of pro­tein de­grad­er C4 Ther­a­peu­tics

Long-time biotech venture firms Versant and New Enterprise Associates are backing a new startup run by former C4 Therapeutics chief executive Andrew Phillips.

The fledgling biotech has raised at least $30 million so far, according to paperwork filed with the SEC this week. The round could balloon to $60 million.

Phillips, who left protein degradation startup C4 in 2020 to be a managing director at Cormorant Asset Management, is running the show of the new venture as president, the SEC filing outlines. He also served as interim CEO of Cormorant-backed and Hansoh Pharmaceutical-partnered Blossom Bioscience last year.

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Alzheimer’s drug bites the dust; Re­struc­ture, re­struc­ture, re­struc­ture; Land­mark di­a­betes OK; 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.

Being in the news business can give one a warped sense of time — it feels like quite a while since we published some of these stories below. But next Saturday’s Endpoints Weekly will definitely be shorter, as we take off Thursday and Friday for Thanksgiving. We will still have the abbreviated edition in your inbox at the usual time.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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