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.

UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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UP­DAT­ED: The FDA sets a reg­u­la­to­ry speed record, pro­vid­ing a snap OK for Ver­tex's break­through triplet for cys­tic fi­bro­sis

The FDA has approved Vertex’s new triplet for cystic fibrosis at a record-setting speed.

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IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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That $335M JV Bay­er set up on CRISPR/Cas9? They’re let­ting the biotech part­ner car­ry on

Bayer committed $300 million to set up a joint venture on CRISPR/Cas9 tech with CRISPR Therapeutics $CRSP. But they’re handing off control now to the smaller biotech while retaining a couple of opt-ins for programs nearing an IND.

Bayer $BAY made much of the fact that they were going all-in on gene editing when they did their deal 3 years ago with CRISPR Therapeutics, which pitched $35 million in on their end. This was the cornerstone of their plan to set up new JVs that could make some serious leap forwards in hot new R&D spaces. Now CRISPR will have full management control of Casebia as they pursue programs in hemophilia, ophthalmology and autoimmune diseases.
Samarth Kulkarni, the CEO at CRISPR, made it sound like a natural progression.

J&J's block­buster Ste­lara wins US ap­proval for ul­cer­a­tive col­i­tis

J&J’s Stelara, which is set to be in the top ten list of blockbusters come 2025, is now cleared by the FDA for use in ulcerative colitis (UC), an inflammatory disease of the large intestine.

The biologic targets interleukin (IL)-12 and IL-23 cytokines, which are known to play a key role in inflammatory and immune responses. Stelara, which generated about $4.7 billion in the first nine months of 2019, is a key player in the crowded marketplace of drugs to treat autoimmune disorders such as psoriasis, rheumatoid arthritis and Crohn’s disease. AbbVie’s star therapy, Humira, continues to dominate, despite its looming patent cliff in the United States, while others including J&J’s $JNJ own anti-IL23 Tremfya, Lilly’s $LLY anti-IL-17 Taltz and AbbVie’s $ABBV recently approved anti-IL-23 antibody Skyrizi carve out a slice of market share.

Drug com­pa­nies reach $260M set­tle­ment just ahead of opi­oid tri­al; Oys­ter Point set terms for $85M IPO

→ Hours before the first federal opioid trial was set to begin, three drug distributors and an opioid manufacturer agreed to a $260 million agreement settlement, the Wall Street Journal was the first to report. The deal — which will see McKesson, Cardinal Health and AmerisourceBergen pay $215 million to Summit and Cuyahoga counties, and Teva deal out $35 million in cash and addiction treatments — does not resolve the pending, nationwide litigation that may result in a settlement worth upwards of $40 billion. Negotiators in that case, brought by 2,300 tribes, counties and cities nationwide and led by several states’ attorneys general, worked through much of Friday without success. Josh Stein, the attorney general for North Carolina, said they were trying to put together a $48 billion deal.

GSK of­floads two vac­cines in $1.1B deal as it works to re­vive the pipeline

GlaxoSmithKline is leaving the deep dark woods and its viruses behind.

GSK has agreed to divest its vaccines for rabies, RabAvert, and tick-born encephalitis vaccine, Encepur, to Bavarian Nordic, part of the company’s broader efforts to narrow its pipeline and focus on oncology and immunology.

The deal is worth up to nearly $1.1 billion, with a $336 million upfront payment. GSK acquired the vaccines from Novartis as part of an exchange for their late-stage oncology programs in 2015 under former chief Sir Andrew Witty.

Pfiz­er gets some en­cour­ag­ing PhI­II news on a fran­chise sav­ior, but is a dos­ing ad­van­tage worth the $295M up­front?

Close to 3 years after Opko tried to defend itself as shares tumbled on the news that its long-acting growth hormone had failed to outperform a placebo, the Pfizer partner $PFE is back. And this time they’re pitching Phase III data that demonstrate their drug is non-inferior — or maybe a tad better — than their well-known but fading standard in the field.
The comparator drug here is Genotropin, which earned a marginal $142 million for Pfizer last year — down 9% from the year before. Approved 24 years ago, biosimilars are now in development that Pfizer would like to stay out in front of. The market leader here is Norditropin, a growth hormone from Novo Nordisk that uses the same basic ingredient as Genotropin, which the Danish company sells with a kid-friendly self-injectable pen. That would also present some big competition if the new therapy from Opko/Pfizer makes it to the market.
The new data, says researchers, underscore that a weekly injection of somatrogon performed as well or slightly better than Genotropin (somatropin) in young children with growth hormone deficiency. Investigators tracked height velocity at 10.12 cm/year, edging out the older drug’s 9.78 cm/year. That 0.33 difference may not prove compelling to payers, though, who have been known to overlook dosing advantages in favor of lower costs.
That message may have weighed on the stock reaction this morning, with a 30%-plus hike $OPK giving way to more marginal gains.
Back in late 2016, Opko had to defend itself against a devastating Phase III setback as their initial late-stage trial failed against a sugar pill. Opko later blamed that setback on outliers in the study, though it wasn’t able to expunge the failure.

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As­traZeneca's Farx­i­ga scores FDA nod to cut risk of hos­pi­tal­iza­tion for heart fail­ure in di­a­bet­ics

While the FDA recently spurned an application to allow AstraZeneca’s blockbuster drug Farxiga for type 1 diabetes that cannot be controlled by insulin, citing safety concerns — the US regulator has endorsed the use of the SGLT2 treatment to reduce the risk of hospitalisation for heart failure in patients with type-2 diabetes and established cardiovascular disease or multiple CV risk factors.