Voy­ager's shares sink af­ter ex­ecs say FDA has re­versed course on an ac­cel­er­at­ed fil­ing for its gene ther­a­py

The FDA ap­pears to have lost its ap­petite for an ac­cel­er­at­ed mar­ket­ing ap­pli­ca­tion that could pave the way to an ear­ly OK for Voy­ager’s lead gene ther­a­py. The com­pa­ny an­nounced af­ter the mar­ket closed Wednes­day that the agency’s view has shift­ed dra­mat­i­cal­ly. 

Af­ter tan­ta­liz­ing in­vestors in Ju­ly with its claim that FDA of­fi­cials said in a Type C ex­change that they were open to a fil­ing based on the safe­ty and ef­fi­ca­cy seen in a Phase II study for VY-AADC in Parkin­son’s dis­ease, the new word is that reg­u­la­tors are now view­ing the mid-stage study as a nec­es­sary pre­lim­i­nary step to a piv­otal tri­al. In an ad­den­dum that ar­rived in late Oc­to­ber, the biotech said, the FDA now says that “al­though the da­ta from the Phase II tri­al may sup­port the safe­ty and ef­fi­ca­cy of VY-AADC and could be con­sid­ered in the BLA re­view, the FDA cur­rent­ly con­sid­ers the Phase II tri­al as an ear­ly phase ex­plorato­ry study.”

Voy­ager’s stock $VY­GR took a 12% dive in af­ter-mar­ket trad­ing.

But it gets worse when you plumb the com­pa­ny’s SEC fil­ing on its lat­est 10K. The biotech says it de­cid­ed to skip a for­mal sit-down with reg­u­la­tors in a Type C meet­ing af­ter they got the ini­tial writ­ten re­spons­es on the time­line from the FDA. But along with the note on their cur­rent view re­gard­ing Phase II’s ex­plorato­ry sta­tus, the com­pa­ny adds:

Con­sis­tent with this state­ment, the FDA fur­ther in­formed us that it has sig­nif­i­cant con­cerns re­gard­ing the abil­i­ty of the Phase 2 tri­al by it­self to pro­vide sub­stan­tial ev­i­dence of safe­ty and ef­fec­tive­ness and that the FDA gen­er­al­ly re­quires at least two ad­e­quate and well-con­trolled clin­i­cal tri­als to pro­vide sub­stan­tial ev­i­dence of ef­fec­tive­ness for sub­mis­sion of a mar­ket­ing ap­pli­ca­tion

Their gene ther­a­py is de­signed to dis­patch the AADC gene di­rect­ly in­to neu­rons of the puta­men where dopamine re­cep­tors are lo­cat­ed, en­abling the neu­rons to ex­press the AADC en­zyme and con­vert lev­odopa in­to dopamine to bet­ter con­trol symp­toms of the dis­ease.

In an up­date on their Phase Ib tri­al, the biotech sep­a­rate­ly not­ed that they picked their Phase II dose af­ter see­ing re­sults for the two high­est dose co­horts in their study, which in­clud­ed a small group of 10 pa­tients.

This com­bined analy­sis demon­strat­ed an in­crease from base­line in good ON time of 2.4 hours per day at 12 months, the time­point for the pri­ma­ry end­point in the Phase 2 tri­al, and 2.6 hours per day at 18 months, the lat­est time­point mea­sured for both co­horts. Of the com­bined ten pa­tients in Co­horts 2 and 3, sev­en pa­tients would be el­i­gi­ble for the Phase 2 tri­al based on lim­its in sever­i­ty of dysk­i­ne­sia and min­i­mum OFF time at base­line. For these sev­en pa­tients, the Phase 2 tri­al rel­e­vant group, the im­prove­ments in good ON time were 2.8 hours at 12 months and 2.5 hours at 18 months. These re­sults were achieved with clin­i­cal­ly mean­ing­ful and sus­tained re­duc­tions in dai­ly oral lev­odopa and re­lat­ed med­ica­tions.

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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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.”

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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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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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.

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.