Fi­bro­Gen, As­traZeneca score Chi­na ap­proval for ane­mia drug ahead of piv­otal US da­ta

It may be the first time a multi­na­tion­al phar­ma­ceu­ti­cal com­pa­ny As­traZeneca $AZN could be­gin sell­ing a po­ten­tial block­buster med­i­cine in Chi­na, be­fore the Unit­ed States or Eu­rope. Fi­bro­Gen $FGEN and the British drug­mak­er have se­cured Chi­na ap­proval for their oral drug, rox­adu­s­tat, for ane­mic pa­tients with chron­ic kid­ney dis­ease who are de­pen­dent on dial­y­sis.

The drug, which stim­u­lates the pro­duc­tion of red blood cells by mim­ic­k­ing the ef­fect of high al­ti­tude in hu­mans, will be launched in the sec­ond half of next year un­der the Chi­nese brand name: ài ruì zhuó 爱瑞卓. While Fi­bro­Gen will take care of man­u­fac­tur­ing, As­traZeneca is in charge of com­mer­cial­iza­tion. Ap­proval for a larg­er pool of pa­tients — who are not de­pen­dent on dial­y­sis — is ex­pect­ed some­time in 2019, af­ter Chi­nese reg­u­la­tors com­plete an in­spec­tion of tri­al sites, the San Fran­cis­co-based com­pa­ny said on Mon­day.

The ap­proval could open up an es­ti­mat­ed $300-500 mil­lion sales op­por­tu­ni­ty, not­ed Jef­feries’ Michael Yee. “For Chi­na, FGEN has re­ceived $43 mil­lion from AZN al­ready and is en­ti­tled to up to $334 mil­lion of ad­di­tion­al pay­ments, much of which could be paid to FGEN this quar­ter,” he wrote. Over­all, Fi­bro­Gen has rough­ly $900 mil­lion in glob­al reg­u­la­to­ry mile­stones out­stand­ing, the ma­jor­i­ty of which should be re­al­ized over the next 12-18 months, not­ed Leerink’s Ge­of­frey Porges.

Over the past few years, Chi­na’s ver­sion of the FDA in­tro­duced a slate of re­forms to en­tice drug de­vel­op­ers, in­clud­ing over­turn­ing a rule that re­quired drug­mak­ers to con­duct sep­a­rate tri­als in Chi­na be­fore ap­provals were sanc­tioned. How­ev­er, Rox­adu­s­tat — which could even­tu­al­ly be used to treat mil­lions af­flict­ed with CKD in Chi­na — was giv­en pri­or­i­ty re­view by Chi­nese health reg­u­la­tors, who agreed to eval­u­ate the mar­ket­ing ap­pli­ca­tion on a rolling ba­sis, af­ter the drug per­formed well in two piv­otal stud­ies in Chi­na.

Porges es­ti­mat­ed rox­adusat will gen­er­ate sales of $9 mil­lion in Chi­na next year – but ex­pects that num­ber will climb to a hefty $1 bil­lion by 2025.

In the Unit­ed States, the drug is ex­pect­ed to be sub­mit­ted for re­view in the first half of next year, af­ter piv­otal tri­als are com­plet­ed. Top-line ef­fi­ca­cy da­ta from a US tri­al are ex­pect­ed in the com­ing weeks, fol­lowed by key MACE safe­ty da­ta in March/April 2019, Yee not­ed.

Fi­bro­Gen, which orig­i­nal­ly de­vel­oped the drug, has part­nered with As­traZeneca since 2013 to de­vel­op and sell the treat­ment in re­gions in­clud­ing the Unit­ed States, Chi­na, Aus­tralia, New Zealand and South­east Asia. Astel­las is work­ing with Fi­bro­Gen to do the same in ar­eas such as Japan, Eu­rope, the Mid­dle East and South Africa. An ap­pli­ca­tion to mar­ket rox­adu­s­tat in Japan was sub­mit­ted in Oc­to­ber.

Fi­bro­Gen and As­traZeneca are locked in a race with Ake­bia $AK­BA, whose ex­per­i­men­tal drug vadadu­s­tat has a sim­i­lar mech­a­nism of ac­tion. But piv­otal vadadu­s­tat da­ta are not ex­pect­ed un­til 2020. The dom­i­nant drugs for anaemia in the Unit­ed States are red-blood-cell boost­ing ery­thro­poiesis-stim­u­lat­ing agents (ESA) from Am­gen $AMGN.

Healthy kid­neys pro­duce a hor­mone called ery­thro­poi­etin (EPO), which prompts the bone mar­row to make red blood cells that car­ry oxy­gen through­out the body. If dis­eased or dam­aged, kid­neys are un­able to make enough EPO, re­sult­ing in few­er blood cells, and even­tu­al­ly anaemia.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.