Astel­las, Pan­th­er­na add or­gan to mR­NA tie-up; Rock­et launch­es sale of six fig­ures worth of stock

Astel­las and Pan­th­er­na have ex­pand­ed their No­vem­ber 2021 pact sur­round­ing the lat­ter’s mR­NA plat­form to in­clude a new tar­get or­gan, the duo an­nounced Tues­day morn­ing, though they did not spec­i­fy what that tar­get is.

Ger­man biotech Pan­th­er­na is home to two plat­form tech­nolo­gies — one that de­signs mR­NAs for non-vac­cine ther­a­pies and an­oth­er that de­signs LNPs. Astel­las and Pan­th­er­na’s deal ap­pears to main­ly re­volve around the first plat­form, which Astel­las said it is us­ing to re­search di­rect re­pro­gram­ming, or turn­ing cells from one kind in­to an­oth­er with­out an in­ter­me­di­ate stem cell phase.

In a press state­ment, Astel­las’ EVP of ap­plied re­search and op­er­a­tions Tai­ji Sawamo­to said:

Through this agree­ment, which ex­pands the scope of our col­lab­o­ra­tion with Pan­th­er­na, we will cre­ate in­no­v­a­tive re­gen­er­a­tive med­i­cine pro­grams for new tar­get or­gan, and we ex­pect that we will be able to there­by ex­pand the treat­ment op­tions for dis­eases with high un­met med­ical needs.

No fi­nan­cial de­tails on the ex­pand­ed deal were dis­closed. — Lei Lei Wu

Rock­et Phar­ma­ceu­ti­cals of­fer­ing up $100 mil­lion worth of its shares

While Rock­et Phar­ma­ceu­ti­cals tout­ed some pos­i­tive da­ta in the spring, the com­pa­ny is now look­ing to Wall Street to of­fer up more of its shares.

The com­pa­ny an­nounced that it has kicked off an of­fer­ing of $100 mil­lion of Rock­et’s com­mon stock and it is let­ting un­der­writ­ers pur­chase up to $15 mil­lion of its shares. All the shares in the sale are to be sold from the com­pa­ny it­self, and the deal is “sub­ject to mar­ket and oth­er con­di­tions.”

The news has perked up the ears of Rock­et’s in­vestors, as the com­pa­ny has seen a 10% boost in its share price since $RCKT open­ing on Tues­day. The com­pa­ny is still fac­ing a 41% fall in its share price since Oc­to­ber 2021, how­ev­er.

The com­pa­ny is al­so rid­ing high as, in May, it showed topline re­sults from a Phase II tri­al in­volv­ing pa­tients with se­vere leuko­cyte ad­he­sion de­fi­cien­cy-I (LAD-1). Rock­et said among nine par­tic­i­pants, the over­all sur­vival at one year is 100% with its can­di­date RP-L201. The re­sults at the time were met with great pos­i­tiv­i­ty by an­a­lysts. — Tyler Patchen

Vac­cine doc­u­ments, young lead­ers and mar­ket tur­moil: End­points' 10 biggest sto­ries of 2022

It’s been a volatile year in the world of biopharma. Market declines reset M&A valuations, and may be beginning to tempt bigger buyers back into dealmaking. Russia’s war in Ukraine disrupted drug sales and clinical trials. A new generation of young biotech leaders emerged in the Endpoints 20(+1) Under 40. And as capital runs dry in a tough environment for raising new funds, companies big and small are taking a look at their headcounts and operations for ways to make it through lean times.

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Tom Riga, Spectrum Pharmaceuticals CEO

Spec­trum im­plodes af­ter a harsh pub­lic slap­down and now a CRL from Richard Paz­dur

The FDA has gone out of its way several times to flatten any expectations for Spectrum’s lung cancer drug poziotinib, including slamming the regulatory door in the biotech’s face four years ago when the their executive crew came calling for a breakthrough drug designation and encouragement from the oncology wing of the FDA.

That stinging early rebuke pointed straight down the path to a corrosive in-house agency review of Spectrum’s attempt to land an accelerated approval for the oral EGFR TKI and a public whipping that included a classic takedown by none other than Richard Pazdur, who slammed the company for “poor drug development” that led to confusion over the dose needed for a slice of NSCLC patients harboring HER2 exon 20 insertion mutations.

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

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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Robert Califf, FDA commissioner (Jose Luis Magana/AP Images)

Fourth ac­cel­er­at­ed ap­proval in Duchenne? Sarep­ta gets pri­or­i­ty re­view for gene ther­a­py amid FDA scruti­ny

Sarepta is once again on the accelerated approval path for a Duchenne drug, picking up a priority review Monday morning.

The FDA granted the accelerated review to SRP-9001, Sarepta announced Monday, which would become the biotech’s fourth Duchenne drug if approved. Much like SRP-9001 will do, each of the previous three therapies went through the accelerated approval pathway. But unlike the others, SRP-9001 is a gene therapy.

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Albert Bourla, Pfizer CEO (John Thys/POOL/AFP via Getty Images)

Pfiz­er CEO un­der fire from UK watch­dog over vac­cine com­ments — re­port

Pfizer CEO Albert Bourla told the BBC last December that he had “no doubt in my mind that the benefits, completely, are in favor” of vaccinating 5- to 11-year-olds for Covid-19. Almost a year later, those comments have reportedly landed him in trouble with a UK pharma watchdog.

Children’s advocacy group UsForThem filed a complaint with the UK’s Prescription Medicines Code of Practice Authority (PMCPA) last year accusing Bourla of making “disgracefully misleading” statements during the BBC interview, including one that “Covid in schools is thriving.” At the time, UK regulators had not yet cleared the vaccine for the 5 to 11 age group, though the vaccine did have a positive opinion from the EMA’s human medicines committee.

Sanofi's new headquarters, La Maison Sanofi, in Paris (Credit: Luc Boegly)

Sanofi wel­comes 500 staffers to new Paris HQ af­ter €30M ren­o­va­tion

When Paul Hudson took the helm at Sanofi back in 2019, he promised to reinvent the pharma giant — including its Paris headquarters. This week, the company set up shop in new “state-of-the-art” digs.

La Maison Sanofi, as the new HQ is called, is officially open for business, Hudson announced on Monday. The 9,000-square-meter (just under 97,000-square-foot) space accommodates 500 employees across the company’s government and global support functions teams, including finance, HR, legal and corporate affairs — and it was built with environmental sustainability and hybrid work in mind.

Sta­da to place $50M+ in­vest­ment in a new fa­cil­i­ty in Ro­ma­nia

While Romania may conjure up images of vast mountain ranges and tales of medieval kings, one generic manufacturer has broken ground on a new facility there.

German pharma company Stada said Monday that it has placed a €50 million ($51.9 million) investment into a 100,000 square-meter (1.08 million square-foot) site in Turda, Romania, a city in the Southeast of the country. According to a Stada spokesperson in an email to Endpoints News, the company has developed only 281,500 square feet of the site so far.

Rachael Rollins (Charles Krupa/AP Images)

US seeks jail time for co-CEO of New Eng­land com­pound­ing cen­ter af­ter dead­ly 2012 fun­gal out­break

The US attorney for the district of Massachusetts late last week called on the state’s district court to sentence the former co-owner of the now-defunct New England Compounding Center to 18 months of jail time for his role in the center’s quality deviations that led to more than 100 people dead from a fungal meningitis outbreak.

Gregory Conigliaro was convicted of conspiring with more than a dozen others at NECC to deceive the FDA and misrepresent the fact that the center was only dispensing drugs pursuant to patient-specific prescriptions.

FDA tells Catal­ent to fix is­sues at two man­u­fac­tur­ing sites on its own

The CDMO Catalent will have to fix issues at two manufacturing plants in the US and Europe that were subject to inspections by the FDA this summer, giving the company room to correct the issues without facing further regulatory action.

The FDA gave Catalent a “voluntary action indicated” response to two inspections at the contract manufacturer’s site in Bloomington, IN, and Brussels, Belgium. Fixing the issues on its own is a preferable outcome to facing an “official action indicated” response, meaning that an official warning would be sent out or a sit-down with the FDA would be required.

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