Hookipa rais­es $60M to fund im­munother­a­py R&D; Roche bags Io­n­is op­tion for $45M; Re­gen­eron fights back against No­var­tis' Eylea ri­val

→ Hookipa Biotech has raised $60 mil­lion (€50 mil­lion) in an over­sub­scribed Se­ries C round to help push its lead pro­gram, a vac­cine for in­fec­tions caused by a genus of virus­es called cy­tomegalovirus, through Phase II clin­i­cal tri­als. The new round was led by an un­named blue chip U.S. pub­lic in­ven­st­ment fund, and was joined by Gilead as a strate­gic in­vestor. Oth­er new in­vestors in­clude HBM Part­ners, Hill­house Cap­i­tal, and Sirona Cap­i­tal. Hookipa al­ready had sev­er­al ex­ist­ing in­vestors on board in­clud­ing Boehringer In­gel­heim’s ven­ture arm and Take­da Ven­tures, which par­tic­i­pat­ed in the round. Oth­er than the cy­tomegalovirus pro­gram (called HB-101), the Vi­en­na-based com­pa­ny is al­so hop­ing to take its ther­a­py for head and neck squa­mous cell car­ci­no­ma to Phase I tri­als. Hookipa plans to ex­pand its tech plat­form in­to oth­er dis­ease ar­eas, in­clud­ing prostate can­cer.

Io­n­is start­ed the day with the news that its part­ner Roche has ex­er­cised the op­tion to li­cense IO­N­IS-HT­TRx for Hunt­ing­ton’s dis­ease, hand­ing the com­pa­ny $IONS $45 mil­lion in cash. Roche made the de­ci­sion — which makes the gi­ant $RHH­BY re­spon­si­ble for all de­vel­op­ment and com­mer­cial ac­tiv­i­ties of the drug — in light of re­sults from a Phase I/IIa place­bo-con­trolled, dose es­ca­la­tion study. A deal years in the mak­ing, the pact with Roche dates back to 2013, when Io­n­is was still known as Isis. To be sure, HD is not at the top of Io­n­is ex­ecs’ minds, who are busy in bat­tle with their hered­i­tary AT­TR amy­loi­do­sis block­buster con­tender in­ot­ersen. But they didn’t mind tout­ing the da­ta from this deal-seal­ing study, ei­ther: “For the first time, a drug has low­ered the lev­el of the tox­ic dis­ease-caus­ing pro­tein in the ner­vous sys­tem, and the drug was safe and well tol­er­at­ed,” said lead in­ves­ti­ga­tor Sarah Tabrizi in a state­ment.

→ The FDA has ac­cept­ed Re­gen­eron’s ap­pli­ca­tion for a 12-week reg­i­men of wet AMD drug Eylea and set a PDU­FA date for Au­gust 2018. The progress puts the com­pa­ny in a bet­ter po­si­tion to com­pete with No­var­tis’ fast-ap­proach­ing Eylea ri­val RTH258 (brolu­cizum­ab), should the dos­ing sched­ule be ap­proved. No­var­tis $NVS is large­ly bank­ing on RTH258’s 12-week reg­i­men will win over a big slice of the Wet AMD mar­ket, as Re­gen­eron’s flag­ship ther­a­py Eylea is cur­rent­ly an 8-week reg­i­men. There’s a lot at stake, with Re­gen­eron $REGN tak­ing in $953 mil­lion in US Eylea sales in Q3 this year. The news is like­ly a re­lief for Re­gen­eron, which has un­der­gone two back-to-back com­bo tri­al flops with Eylea. Ear­li­er this year, the drug in com­bi­na­tion with nes­vacum­ab failed to beat a so­lo ver­sion of Eylea. And more than a year ago, its an­ti-PDFG an­ti­body rin­u­cum­ab com­bined with Eylea failed a Phase II tri­al for age-re­lat­ed mac­u­lar de­gen­er­a­tion.

→ In an­oth­er at­tempt to run ahead of ri­vals Iron­wood and Syn­er­gy, Arde­lyx an­nounced to­day that it has out-li­censed tena­panor for $12 mil­lion up­front to Shang­hai-based Fo­s­un Phar­ma, which has bought the ex­clu­sive rights to de­vel­op and com­mer­cial­ize the lead prod­uct in Chi­na. Ad­di­tion­al mile­stones to­tal up to $113 mil­lion; the agree­ment al­so cov­ers tiered roy­al­ty pay­ments on net sales rang­ing from the mid-teens to 20%. Where­as the fo­cus is on the hot­ly con­test­ed field of ir­ri­ta­ble bow­el syn­drome with con­sti­pa­tion (IBS-C) and hy­per­phos­phatemia re­lat­ed to chron­ic kid­ney dis­ease, Fo­s­un has al­so ac­quired the rights to com­mer­cial­ize tenepanor for oth­er in­di­ca­tions ap­proved in the US. The Fre­mont, CA-based biotech came out in Oc­to­ber with new PhI­II da­ta that proved more im­pres­sive than the round re­leased in May. Clear­ly, Fo­s­un shared the en­thu­si­asm of US in­vestors. “Tena­panor’s po­ten­tial as a dif­fer­en­ti­at­ed treat­ment for both car­diore­nal and GI dis­eases is im­pres­sive,” said Yi­fang Wu, pres­i­dent and CEO of Fo­s­un’s epony­mous par­ent com­pa­ny. “The da­ta gen­er­at­ed by Arde­lyx to-date gives us con­fi­dence that tena­panor could be a lead­ing treat­ment op­tion for both ther­a­peu­tic ar­eas, of­fer­ing unique ad­van­tages to pa­tients.”

With ad­di­tion­al re­port­ing by Brit­tany Meil­ing.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.