FDA re­jects Ac­er's rare dis­ease drug, asks for new tri­al — shares crater

Ac­er Ther­a­peu­tics’ bid to re­pur­pose celipro­lol — a be­ta-block­er on the mar­ket for hy­per­ten­sion — as a treat­ment for a rare, in­her­it­ed con­nec­tive tis­sue dis­or­der has hit a se­vere set­back. The New­ton, Mass­a­chu­setts-based com­pa­ny on Tues­day said the FDA re­ject­ed the drug and has asked for an­oth­er clin­i­cal tri­al.

The com­pa­ny’s shares $AC­ER cratered near­ly 77% to $4.47 in Tues­day morn­ing trad­ing.

Chris Schelling LinkedIn

The drug, brand­ed as Ed­si­vo, is un­der de­vel­op­ment for vas­cu­lar Ehlers-Dan­los syn­drome (vEDS) — a rare dis­or­der caused by mu­ta­tions large­ly in the COL3A1 gene, which caus­es ar­te­r­i­al dis­sec­tions and rup­tures that can lead to an ear­ly death. It is es­ti­mat­ed to af­fect be­tween 1 in 50,000 and 1 in 200,000 peo­ple, and there are no treat­ments ap­proved to treat it.

The com­pa­ny sub­mit­ted an ap­pli­ca­tion to mar­ket the drug based on a small, 54-pa­tient study — the Be­ta-Block­ers in Ehlers-Dan­los Syn­drome Treat­ment (BBEST) —  which was de­signed to show the drug could pre­vent ar­te­r­i­al dis­sec­tions and rup­tures in vEDS pa­tients. The tri­al test­ed the drug against a place­bo and was stopped ear­ly as celipro­lol was work­ing well; pa­tients giv­en the drug, com­pared with no treat­ment, saw a re­duc­tion in ar­te­r­i­al events, such as rup­ture or dis­sec­tion, by three­fold. How­ev­er, the study was un­der­pow­ered. Re­searchers ac­knowl­edged that they did not “reach our tar­get num­ber of pa­tients”.

In April, Paris vEDS Pa­tient Reg­istry Da­ta was pub­lished in the Jour­nal of the Amer­i­can Col­lege of Car­di­ol­o­gy. It “de­scribed the long-term out­come of 144 COL3A1+ vEDS pa­tients, most of whom (~90%) were treat­ed with celipro­lol and mon­i­tored for up to 20 yrs (me­di­an 5.3 yrs). Key da­ta high­lights in­clude low­er rates of mor­tal­i­ty and ar­te­r­i­al com­pli­ca­tions in this pa­tient group than that ex­pect­ed from the nat­ur­al his­to­ry of the dis­ease. Celipro­lol-treat­ed pa­tients had an 80.7% sur­vival rate at ~11 yrs vs 48.5% for those not treat­ed,” Need­ham’s Serge Be­langer wrote in a note.

Be­langer was op­ti­mistic the drug would win ap­proval, and es­ti­mat­ed a tar­get mar­ket op­por­tu­ni­ty be­tween $200 mil­lion and $400 mil­lion, based on ex­pect­ed Ed­si­vo an­nu­al pric­ing of $100,000-$200,00 per pa­tient.

On Tues­day, Ac­er chief Chris Schelling said the com­pa­ny is ex­pect­ed to re­spond to the FDA in the third quar­ter.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect"

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

House com­mit­tee unan­i­mous­ly ad­vances FDA user fee leg­is­la­tion with ac­cel­er­at­ed ap­proval tweaks

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.

Pearl Huang, former Cygnal Therapeutics president and CEO

UP­DAT­ED: Flag­ship builds a new start­up out of pieces from 2 of its biotechs. And a Roche vet leaves to do some­thing new

Flagship has crafted a new startup out of pieces from a pair of fledglings in the VC’s nest. And a prominent Roche veteran who ran one of the biotechs won’t be making the next leg of the journey.

The new company is called Sonata Therapeutics, which is picking up the work that Inzen was doing related to the cellular microenvironment and combining with Flagship’s Cygnal Therapeutics, which came out of stealth more than 3 years ago and put Pearl Huang — the BeiGene founder and former Roche SVP — at the helm.

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Patty Murray (D-WA) (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usually reserved for making decisions on drug applications or enforcing what Congress stipulates, the FDA is now dipping its toe into the wild world of congressional politics as it attempts to fix a major court decision that could have a chilling effect on rare disease R&D.

The case in question from last October saw a US appeals court overturn a prior FDA court win, saying that the agency never should’ve approved a rare disease drug because a previously approved but more expensive drug with the same active ingredient has orphan drug exclusivity barring such an approval.

Peter Marks (Greg Nash/Pool via AP)

Even FDA's Pe­ter Marks is wor­ried about the com­mer­cial vi­a­bil­i­ty of gene and cell ther­a­pies

When bluebird bio’s gene therapy to treat beta thalassemia won European approval in 2019, the nearly $2 million per patient price tag for the potential cure seemed like a surmountable hurdle.

Fast forward two years later, and bluebird has withdrawn Zynteglo, the beta thal drug, along with the rest of its gene therapy portfolio from Europe, which the company said is generally unwilling to pay a fair price for the treatment.

Pri­cy in­halers re­main ex­pen­sive due to de­vice tweaks that keep com­peti­tors at bay, re­searchers find

New research published in Health Affairs today highlights the way in which the FDA’s inhaler regulations have rewarded incremental adjustments to older products, thereby enabling companies to skirt around cheaper competition.

A DC appeals court clerk and researchers from Harvard and the University of Calgary dug through all the patents and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020, finding that of the 62 inhalers approved, 53 (or 85%) were brand-name products, with a median of 16 years of protection from generic competition.