Sarep­ta faces an­oth­er gene ther­a­py hic­cup as Re­genxbio sues over Jim Wilson's patent

It turns out a reg­u­la­to­ry de­lay isn’t the on­ly bump on Sarep­ta’s road to gene ther­a­py glo­ry.

The biotech is be­ing sued by Re­genxbio, which is al­leg­ing that its slate of gene ther­a­py pro­grams, in­clud­ing two for Duchenne mus­cu­lar dy­s­tro­phy and limb-gir­dle mus­cu­lar dy­s­tro­phy in­fringes on a patent orig­i­nal­ly owned by the Uni­ver­si­ty of Penn­syl­va­nia.

The law­suit, which was first re­port­ed by Bloomberg Law, adds an­oth­er wrin­kle as Sarep­ta strives to stay in the lead in a race to de­liv­er the first ge­net­ic fix for Duchenne mus­cu­lar dy­s­tro­phy. Af­ter mak­ing its name with two an­ti­sense drugs — nei­ther of which has been proven to have an ef­fect against the crip­pling dis­ease — the Cam­bridge, MA-based biotech has been viewed as the fron­trun­ner ver­sus Pfiz­er and trou­ble-prone Sol­id Bio. Roche was im­pressed enough to pay $1.15 bil­lion to ac­quire ex-US rights to the pro­gram.

But Re­genxbio wants Sarep­ta to stop step­ping on their patents — and pay up. The biotech, which is now based in Rockville, MD, is seek­ing dam­ages for past, present and/or fu­ture in­fringe­ment equal­ing “no less than a rea­son­able roy­al­ty.”

James Wil­son

At the cen­ter of the dis­pute is US Patent No. 10,526,617. Jim Wil­son, the gene ther­a­py lu­mi­nary who co-found­ed Re­genxbio, was cit­ed as an in­ven­tor on that patent, grant­ed this Jan­u­ary.

As it cov­ers a cul­tured host cell con­tain­ing a re­com­bi­nant nu­cle­ic acid mol­e­cule en­cod­ing the cap­sid pro­tein, the tech­nol­o­gy can be used to cre­ate ade­no-as­so­ci­at­ed vec­tors — both in an­i­mal stud­ies and for de­liv­er­ing a trans­gene in­to hu­mans.

“The vec­tors made us­ing the claimed sub­ject mat­ter of the ’617 Patent have unique prop­er­ties, e.g., an abil­i­ty to tar­get cer­tain types of cells in the body,” the law­suit states.

Specif­i­cal­ly, Re­genxbio al­leges, Sarep­ta’s Duchenne pro­gram — SRP-9001 — is man­u­fac­tured by a process that in­cludes mak­ing and us­ing a cul­tured host cell a re­com­bi­nant nu­cle­ic acid mol­e­cule en­cod­ing an AAVrh74 vp1 cap­sid pro­tein. The same cap­sid pro­tein is al­so in­te­gral to SRP-9003, the limb gir­dle can­di­date, and a host of oth­er fol­low-on gene ther­a­pies.

Doug In­gram

Re­genxbio is su­ing as Sarep­ta is sort­ing out a new re­quest from reg­u­la­tors that can push back its piv­otal study for Duchenne. While CEO Doug In­gram as­sured in­vestors that qual­i­ty con­trol is­sues such as this are not un­com­mon and pinned the de­lay on an over­bur­dened FDA, he stopped short of promis­ing con­crete time­lines.

Fo­cused on reti­nal, meta­bol­ic and neu­rode­gen­er­a­tive dis­eases for its in­ter­nal pipeline, Re­genxbio is per­haps more ac­com­plished as a gene ther­a­py tech provider. No­var­tis and Abeona have both li­censed its NAV tech plat­form.

It al­so doesn’t shy away from le­gal ac­tions. In its most re­cent quar­ter­ly re­port, Re­genxbio dis­closed that Abeona failed to make a $8 mil­lion pay­ment due in April, ef­fec­tive­ly ter­mi­nat­ing their li­cens­ing agree­ment. In re­sponse to an ar­bi­tra­tion claim Abeona filed in May “al­leg­ing we breached cer­tain re­spon­si­bil­i­ties to com­mu­ni­cate with Abeona re­gard­ing our pros­e­cu­tion of li­censed patents,” Re­genxbio filed a coun­ter­claim to ask for $28 mil­lion — in­clud­ing $20 mil­lion that would have been owed un­der the pact.

Last No­vem­ber Re­genxbio chal­lenged the FDA’s “ar­bi­trary and capri­cious” de­ci­sions to is­sue a full clin­i­cal hold on its di­a­bet­ic retinopa­thy tri­al and a par­tial hold on wet age-re­lat­ed mac­u­lar de­gen­er­a­tion. The com­pa­ny had with­drawn the IND for di­a­bet­ic retinopa­thy, and the FDA lift­ed the par­tial hold for wet AMD two months lat­er.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Vlad Coric, Biohaven CEO

Vlad Coric charts course for new Bio­haven with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Kite Phar­ma gets FDA to sign off on new Cal­i­for­nia-based vec­tor man­u­fac­tur­ing fa­cil­i­ty

Kite Pharma just got FDA approval to kick off operations at a new manufacturing campus.

The cancer-focused, CAR-T cell therapy player made the announcement Monday, saying that the federal regulatory agency gave the green light to Kite’s 100,000 square-foot, retroviral vector manufacturing facility in Oceanside, CA.

Kite’s global head of technical operations Chris McDonald tells Endpoints News that the facility has been in the works for about four years, after Kite teamed up with its parent company Gilead. Gilead acquired Kite Pharma for just shy of $12 billion in 2017.

Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

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Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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Benjamine Liu, TrialSpark CEO

Paul Hud­son and Tri­alSpark's mu­tu­al de­sire to speed up de­vel­op­ment con­verges in three-year, six-drug goal

A unicorn startup that originally set out to hasten clinical studies for biopharma partners dug further into its revised path of internal drug development by linking arms with Sanofi in a pact that the biotech’s CEO said originated from the top.

TrialSpark and the Big Pharma on Tuesday committed to in-licensing and/or acquiring six Phase II/Phase III drugs within the next three years.

“I’ve known Paul Hudson for a while and we were discussing the opportunity to really re-imagine a lot of different parts of pharma,” TrialSpark CEO Benjamine Liu told Endpoints News, “and one of the things that we discussed was this opportunity to accelerate the development of new medicines in mutual areas of interest.”

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