Tony Coles, Cerevel

A Tony Coles Parkin­son's biotech, Yu­man­i­ty, con­tin­ues its back­slide as lead pro­gram earns a par­tial hold

A Tony Coles biotech look­ing to de­vel­op new drugs for Parkin­son’s dis­ease and oth­er neu­ro­log­i­cal con­di­tions ran in­to trou­ble Wednes­day as the FDA slapped its lead pro­gram with a par­tial clin­i­cal hold.

Yu­man­i­ty will need to pause stud­ies eval­u­at­ing mul­ti-dose ad­min­is­tra­tion of the drug, dubbed YTX-7739, in Parkin­son’s, the biotech said. Though Yu­man­i­ty did not elab­o­rate on what ex­act­ly reg­u­la­tors took is­sue with, the FDA is al­low­ing a planned study of a sin­gle dose to go ahead.

More de­tails will be shared with Yu­man­i­ty in the next 30 days, but un­til then the mul­ti-dose study can­not be­gin en­rolling pa­tients. Shares of Yu­man­i­ty $YMTX fell about 25% af­ter Wednes­day’s open­ing bell.

YTX-7739 is Yu­man­i­ty’s on­ly can­di­date to reach hu­man stud­ies since the com­pa­ny’s found­ing in 2014, and it’s al­so be­ing stud­ied in glioblas­toma and oth­er dis­eases. Parkin­son’s is the lead in­di­ca­tion, how­ev­er, and the biotech has banked on a po­ten­tial suc­cess here as a proof-of-con­cept for the whole plat­form.

Wednes­day’s news comes about two months af­ter Yu­man­i­ty re­vealed some of the first da­ta look­ing at whether the pro­gram could mod­i­fy Parkin­son’s dis­ease pro­gres­sion. In a Phase Ib study, YTX-7739 failed to show any sig­nif­i­cant dif­fer­ence from place­bo in most clin­i­cal mea­sure­ments and ex­plorato­ry bio­mark­ers af­ter 28 days, which the biotech said was ex­pect­ed.

The biotech al­so high­light­ed a small­er sub­set of pa­tients on whom Yu­man­i­ty con­duct­ed a quan­ti­ta­tive elec­troen­cephalo­gram to mea­sure brain ac­tiv­i­ty, which the biotech said was sta­tis­ti­cal­ly sig­nif­i­cant and might sug­gest pos­si­ble ef­fects on synap­tic func­tion. Yu­man­i­ty fur­ther said the com­pound ef­fec­tive­ly in­hib­it­ed its tar­get, stearoyl-CoA de­sat­urase, some­thing the biotech has de­scribed as a po­ten­tial­ly nov­el way to treat Parkin­son’s and oth­er dis­eases.

In­vestors, though, didn’t take that par­tic­u­lar­ly well, shun­ning the stock and send­ing it down about 33% and to­ward pen­ny stock ter­ri­to­ry at the time. Shares have on­ly con­tin­ued to spi­ral in the months since and Wednes­day’s hold put Yu­man­i­ty be­low the $2 mark for the first time.

The Phase Ib study in ques­tion on­ly looked at a 20 mg sin­gle dose, and Yu­man­i­ty pre­vi­ous­ly said it ex­pects to start a Phase II tri­al some­time this year.

The FDA hold marks an even deep­er con­trast be­tween Yu­man­i­ty and one of Coles’ oth­er neu­rol­o­gy biotechs in Cerev­el Ther­a­peu­tics, tak­ing the rem­nants of Pfiz­er’s old neu­ro­science unit. Where­as in­vestors have con­tin­ued to show their dis­ap­point­ment in Yu­man­i­ty, Cerev­el has been on a sharp rise since a da­ta re­veal last June showed its schiz­o­phre­nia pro­gram sig­nif­i­cant­ly low­ered symp­tom sever­i­ty com­pared to place­bo.

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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David Loew (Ipsen)

Ipsen snags an ap­proved can­cer drug in $247M M&A deal as an­oth­er bat­tered biotech sells cheap

You can add Paris-based Ipsen to the list of discount buyers patrolling the penny stock pack for a cheap M&A deal.

The French biotech, which has had plenty of its own problems to grapple with, has swooped in to buy Epizyme $EPZM for $247 million in cash and a CVR with milestones attached to it. Epizyme shareholders, who had to suffer through a painfully soft launch of their EZH2a inhibitor cancer drug Tazverik, will get $1.45 per share along with a $1 CVR tied to achieving $250 million in sales from the drug over four consecutive quarters as well as an OK for second-line follicular lymphoma by 1 Jan. 2028.

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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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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.

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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Alarmed by side ef­fect, FDA slaps clin­i­cal hold on Sarep­ta's next-gen Duchenne drug

Sarepta Therapeutics’ next-gen Duchenne muscular dystrophy drug has been hit with a clinical hold after investigators flagged a serious case of low magnesium levels in one patient’s blood.

Screening and dosing will be halted in what is known as Part B of the Phase II MOMENTUM study, which has enrolled about half of the planned patients. Sarepta said it will be submitting information on all cases of the condition, known as hypomagnesemia, per the FDA’s request and proposing some changes to the risk mitigation and safety monitoring plan.

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When will the FDA re­scind a break­through des­ig­na­tion? New draft guid­ance spells out the com­pli­cat­ed de­tails

Although it’s exceedingly rare for the FDA to rescind a breakthrough designation once it’s granted, there has been a recent uptick — as in 2020 and 2021 combined, the agency rescinded 17 BTDs, compared to just 18 rescinded from 2015 to 2019 combined.

Protagonist Therapeutics saw this reality up close and personal in April after a clinical hold lifted on its experimental blood cancer drug, as the company revealed that the FDA sought to revoke the BTD. The decision, Protagonist says, stems from “observed malignancies” related to the hold, initially imposed in September 2021.

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