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.

Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Adrian Gottschalk, Foghorn CEO (Foghorn)

Foghorn hits Nas­daq in $120M de­but as the biotech IPO boom shows no sign of slow­ing

It’s been a record year for biotech IPOs, and the execs at Nasdaq would like nothing better than to see that momentum continue into the first half of next year.

Since January, 72 biotech and biopharma companies have hit Wall Street, according to Nasdaq head of healthcare listings Jordan Saxe, together raising $13.2 billion.

The latest is Flagship’s Foghorn Therapeutics, which priced its shares last night at $16 apiece, the midpoint of a $15 to $17 range. The Cambridge, MA-based biotech — which initially filed for a $100 million raise on Oct. 2 — is netting $120 million from a 7.5 million-share offering. The proceeds will go right into its gene traffic control platform, including two lead preclinical oncology candidates.