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.

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.

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.

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.

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.

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.