Shankar Musunuri, Ocugen CEO

Ocu­gen hits an­oth­er snag in bring­ing its Bharat Biotech-part­nered Covid-19 vac­cine to the US

Back in Ju­ly, Ocu­gen and Bharat Biotech un­veiled some Phase III da­ta sug­gest­ing their Covid-19 vac­cine, Co­v­ax­in, was 77.8% ef­fec­tive at pre­vent­ing over­all dis­ease in In­dia. They’re now look­ing to launch an im­muno-bridg­ing study to see if those num­bers hold up in the US — but on Fri­day, the FDA said not so fast.

Reg­u­la­tors have placed a clin­i­cal hold on the part­ners’ IND, Ocu­gen shared on Fri­day. Ocu­gen’s stock $OCGN sank 9% on the news Fri­day, but was up al­most 7% in pre-mar­ket trad­ing on Mon­day.

“The FDA plans to iden­ti­fy the spe­cif­ic de­fi­cien­cies that are the ba­sis for clin­i­cal hold and in­for­ma­tion on how to ad­dress those de­fi­cien­cies,” the Malvern, PA-based com­pa­ny said in a state­ment. “The Com­pa­ny ex­pects to re­ceive for­mal writ­ten com­mu­ni­ca­tion with the ad­di­tion­al in­for­ma­tion from the FDA and plans to work with the FDA in an ef­fort to re­solve its ques­tions as prompt­ly as pos­si­ble.”

The news comes just a few weeks af­ter Ocu­gen filed for emer­gency use au­tho­riza­tion of the Bharat Biotech-part­nered shot for just pe­di­atric use based on an im­muno-bridg­ing study con­duct­ed in In­dia.

Co­v­ax­in, a whole-viri­on, in­ac­ti­vat­ed vac­cine, was de­vel­oped by Bharat Biotech along with the In­di­an Coun­cil of Med­ical Re­search. The World Health Or­ga­ni­za­tion grant­ed it an emer­gency use list­ing ear­li­er this month, stat­ing that the jab is “ex­treme­ly suit­able for low- and mid­dle-in­come coun­tries due to easy stor­age re­quire­ments.”

In Ju­ly, Bharat an­nounced that the vac­cine was 77.8% ef­fec­tive against Covid-19 of any sever­i­ty, and 93.4% ef­fec­tive against se­vere dis­ease in a Phase III tri­al with more than 25,000 par­tic­i­pants in In­dia. Few­er than 0.5% of pa­tients ex­pe­ri­enced se­vere side ef­fects, the In­di­an biotech said at the time.

Ear­li­er this year, Ocu­gen an­nounced plans to file for an EUA in the US in June. But when June rolled around, the com­pa­ny said it de­cid­ed to pur­sue a bi­o­log­ics li­cense ap­pli­ca­tion in­stead, up­on rec­om­men­da­tion by the FDA. At the end of Oc­to­ber, Ocu­gen filed an IND to con­duct a Phase III im­muno-bridg­ing study in “sev­er­al hun­dred” US adults who’ve nev­er been vac­ci­nat­ed, or re­ceived two dos­es of an mR­NA vac­cine at least six months pri­or.

Just a week lat­er, the com­pa­ny sub­mit­ted an EUA for use in kids ages 2 to 18 based on an­oth­er im­muno-bridg­ing study con­duct­ed in In­dia. Ac­cord­ing to Ocu­gen, an im­muno-bridg­ing study showed that neu­tral­iz­ing an­ti­body re­spons­es in the pe­di­atric group were equiv­a­lent to those seen in adults.

“Our re­search sug­gests that peo­ple are seek­ing more choic­es when se­lect­ing a vac­cine, es­pe­cial­ly for their chil­dren,” CEO Shankar Musunuri said. “Hav­ing a new type of vac­cine avail­able will en­able peo­ple to dis­cuss with their child’s physi­cian the best ap­proach for them to low­er their child’s risk of con­tract­ing COVID-19.”

The clin­i­cal hold may not be a good sign for Ocu­gen’s pe­di­atric EUA, though we’ll have to wait and see.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

FDA un­veils new draft guid­ance to help with oligonu­cleotide ther­a­peu­tics de­vel­op­ment

While oligonucleotides, a wide variety of synthetically modified RNA or RNA/DNA hybrids that bind to a target RNA sequence to alter RNA and/or protein expression, have been winning approvals in recent years (e.g. Novartis’ cholesterol drug Leqvio), the regulatory agency is offering new draft guidance for those looking to follow a similar path.

The non-binding guidance, titled “Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics Guidance for Industry” deals with pharmacokinetic, pharmacodynamic, and safety assessments required as part of oligonucleotide therapeutics R&D.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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