Apel­lis grabs $60M to pay for a loom­ing PhI­II show­down with Alex­ion's Soliris

Back­ing up da­ta from small but promis­ing stud­ies for their ri­val ther­a­py to Soliris, Louisville-based Apel­lis has round­ed up a $60 mil­lion round to pay for a late-stage show­down with Alex­ion $ALXN.

Hav­ing tried and failed to pull off an IPO in chilly mar­ket wa­ters, this E round in­cludes some top ven­ture play­ers — who may be re­cal­cu­lat­ing the biotech’s shot at a re­peat ef­fort on the IPO front. Sec­toral As­set Man­age­ment led the round with Sofinno­va, Vi­vo Cap­i­tal, F-Prime Cap­i­tal Part­ners, cer­tain in­vest­ment funds ad­vised by Clough Cap­i­tal Part­ners, and ven­Bio Se­lect join­ing as new in­vestors. Ex­ist­ing in­vestors Morn­ing­side Ven­tures, Cor­morant As­set Man­age­ment, ven­Bio Glob­al Strate­gic Fund, and Ep­i­darex Cap­i­tal al­so par­tic­i­pat­ed in the fi­nanc­ing.

It’s no co­in­ci­dence that a lit­tle more than a month ago Apel­lis re­port­ed out pos­i­tive re­sults from two tiny stud­ies of 3 and 6 pa­tients suf­fer­ing from parox­ys­mal noc­tur­nal he­mo­glo­bin­uria (PNH). In 3 pa­tients nev­er treat­ed with Soliris, in­ves­ti­ga­tors re­port­ed that all of them ex­pe­ri­enced a quick cor­rec­tion on a key bio­mark­er for lac­tate de­hy­dro­ge­nase, or LDH. In 6 pa­tients not re­spond­ing well to Soliris, the av­er­age he­mo­glo­bin lev­el was brought up an av­er­age of 36%, LDH was cor­rect­ed and trans­fu­sions dropped from 3.4/month on eculizum­ab monother­a­py to 0.3/month when APL-2 was added to eculizum­ab. And the biotech raised no un­usu­al red flags on the safe­ty side.

Apel­lis says the fresh cash will fund the Phase III pro­gram for their drug while the com­pa­ny is al­so prep­ping the re­lease of re­sults from a Phase I study in au­toim­mune he­molyt­ic ane­mia and a Phase II study in ge­o­graph­ic at­ro­phy, an ad­vanced form of age-re­lat­ed mac­u­lar de­gen­er­a­tion.

Drawn to a mar­ket dom­i­nat­ed by one of the world’s most ex­pen­sive ther­a­pies, Apel­lis isn’t alone in work­ing on a drug that could re­place Alex­ion’s big mon­ey­mak­er. Ra Phar­ma­ceu­ti­cals and Akari have been on the trail, while Alex­ion has al­so been mak­ing ad­vances with a sec­ond-gen prod­uct for their key mon­ey­mak­er.

“Da­ta gen­er­at­ed to date with APL-2 across a range of clin­i­cal in­di­ca­tions sup­port our be­lief that C3 in­hi­bi­tion has great po­ten­tial to de­liv­er nov­el and com­mer­cial­ly suc­cess­ful treat­ments,” com­ment­ed Cedric Fran­cois, founder and CEO of Apel­lis. “This group of top tier in­vestors sup­ports our vi­sion of de­vel­op­ing APL-2 to its full po­ten­tial, and of pro­vid­ing a range of dif­fer­en­ti­at­ed treat­ments to pa­tients with se­ri­ous un­met med­ical needs,” he added. “The re­main­der of this year will see sig­nif­i­cant mile­stones for Apel­lis.”

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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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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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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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.

Sofinno­va-backed Abi­vax touts longer term mid-stage da­ta in ul­cer­a­tive col­i­tis

Two months after Abivax convinced Sofinnova to bankroll several mid-stage studies of its lead drug — ABX464 — with a €12 million stock purchase, the Paris-based biotech has rolled out more data on the anti-inflammatory molecule for all investors to see.

In a Phase IIa maintenance study involving 22 patients with moderate to severe ulcerative colitis who have been failed by previous treatments, 12 achieved clinical remission as assessed by endoscopy. But since only 19 completed the full one-year trial, 16 of whom had an endoscopy, investigators scored the remission rate at 75%. Although there’s no comparator arm, execs were pleased with improvements over an initial two-month, placebo-controlled induction phase by a number of measures ranging from remission to Mayo score and a fecal biomarker.

Alex­ion clinch­es aHUS ap­proval for Ul­tomiris as the clock ticks on Soliris con­ver­sion

Alexion has racked up a second approval for Ultomiris, the successor therapy to Soliris, as its mainstay blockbuster therapy faces a patent review process that could drastically shorten its patent exclusivity.

The FDA OK for atypical hemolytic uremic syndrome (aHUS) on Friday was widely expected after Alexion posted a full slate of positive Phase III data in January. But regulators also flagged concerns about serious meningococcal infections, slapping a black box warning on the label and mandating a REMS.