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.

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.