A new play­er in the search for an oral hered­i­tary an­gioede­ma pill has re­ceived a ma­jor boost and show of con­fi­dence.

Phar­varis an­nounced $66 mil­lion in Se­ries B fi­nanc­ing for its B2 re­cep­tor an­tag­o­nist pill, near­ly tripling the fund­ing At­tune Phar­ma­ceu­ti­cals re­cent­ly re­ceived in Se­ries B for its own hered­i­tary an­gioede­ma (HAE) pill. The mon­ey will fu­el the com­pa­ny’s Phase I tri­al on its lead com­pound, PHA121, on healthy vol­un­teers. The first sub­jects have al­ready been dosed.

The news comes three-and-a-half months af­ter BioCryst’s great an­gioede­ma fiz­zle. Al­though the Durham out­fit’s oral drug BCX7353 tech­ni­cal­ly passed a Phase III tri­al, the drug failed to cut the at­tack rate by at least 50%, the cut­off at which Wall Street an­a­lysts con­sid­ered the drug mar­ketable, and the biotech’s stock tum­bled.

Hered­i­tary an­gioede­ma, a po­ten­tial­ly life-threat­en­ing ge­net­ic dis­or­der, af­fects 1 in every 50,000 peo­ple. It is char­ac­ter­ized by episodes of ex­treme swelling across the body. It can cause vom­it­ing and nau­sea when man­i­fest in the gut and can even be fa­tal if it ap­pears in the air­ways and re­stricts breath­ing.

Sev­er­al ther­a­pies and in­jec­tions ex­ist to curb the at­tacks, but last year Shire’s Takhzy­ro (lanadelum­ab) emerged as one of the hottest and most ef­fec­tive drugs, con­tribut­ing to an 87% re­duc­tion in mean HAE at­tack fre­quen­cy in a Phase III tri­al (ver­sus a 0% re­duc­tion in the place­bo group) that ush­ered it to­ward mar­ket. The drug is on track to be­come a block­buster. Clair­voy­ant An­a­lyt­ics projects $1.15 bil­lion in sales by 2022.

But be­cause Takhzy­ro is an in­jec­tion, the search is still on for an oral med­ica­tion that can curb the dis­or­der at sim­i­lar rates. BioCryst con­tin­ues to de­vel­op its oral drug, while At­tune Phar­ma­ceu­ti­cals re­mains in the ear­ly stage.

HAE is caused by low lev­els of or dys­func­tion­ing C1 es­terase in­hibitor pro­tein. Most avail­able ther­a­pies work by in­hibit­ing C1 es­terase, ful­fill­ing the func­tion of the miss­ing pro­tein. But Phar­varis’ Ph121 works like the FDA-ap­proved icat­i­bant in­jec­tion, which func­tions as an an­tag­o­nist of bradykinin B2 re­cep­tors – i.e. it at­tacks the mol­e­cules that di­rect­ly cause the swelling. The founders of Phar­varis in­clude an in­ven­tor of icat­i­bant and a for­mer mem­ber of the lead­er­ship at Jeri­ni AG, the bio­phar­ma­ceu­ti­cal that sells the in­jec­tion.

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.

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.

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.