Af­ter BioCryst fiz­zle, a new oral HAE play­er emerges

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.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

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. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

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.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Mer­ck helps bankroll new part­ner Themis' game plan to fin­ish the chikun­gun­ya race and be­gin on­colyt­ic virus quest

As Themis gears up for a Phase III trial of its chikungunya vaccine, the Vienna-based biotech has closed out €40 million ($44 million) to foot the clinical and manufacturing bills.

Its heavyweight partners at Merck — which signed a pact around a mysterious “blockbuster indication” last month — jumped into the Series D, led by new investors Farallon Capital and Hadean Ventures. Adjuvant Capital also joined, as did current investors Global Health Investment Fund, aws Gruenderfonds, Omnes Capital, Ventech and Wellington Partners Life Sciences.