As uter­ine race with Ab­b­Vie heats up, My­ovant eyes FDA ap­proval with tri­al re­sults from prostate can­cer

My­ovant has long had a se­cret weapon in its uter­ine ri­val­ry with Ab­b­Vie: Men.

Lynn Seely My­ovant

While the small Swiss biotech has jock­eyed with the Illi­nois-based gi­ant for a foothold in the en­dometrio­sis and uter­ine fi­broid ther­a­py mar­ket, the com­pa­ny has been de­vel­op­ing the same lead com­pound, re­l­u­golix, for use in one of the most com­mon can­cers for the uterus-less: prostate can­cer. To­day, My­ovant is out with pos­i­tive topline re­sults from its big Phase III tri­al on the go­nadotropin-re­leas­ing hor­mone (GnRH) an­tag­o­nist. They say they’ve reached every pri­ma­ry and sec­ondary end­point with p val­ues less than .0001.

“It works quick­ly,” CEO Lynn Seely told End­points News. “It’s an oral pill and it sup­press­es hor­mone pro­duc­tion and when you stop it, your hor­mones come back and you have the po­ten­tial for im­proved qual­i­ty of life.”

My­ovant’s stock is up 95% pre­mar­ket, from $6.06 to $11.80 per share

The tri­al test­ed a re­l­u­golix arm along­side a stan­dard-of-care, le­upro­lide, arm in their abil­i­ty to sup­press testos­terone in pa­tients with ad­vanced prostate can­cer. Testos­terone fu­els prostate can­cer’s growth. The pri­ma­ry end­point was the per­cent­age of men who reached and main­tained med­ical cas­tra­tion, or less than 50 nanograms of testos­terone per deciliter, over 48 weeks.

Re­l­u­golix achieved that in 96.7% of pa­tients — safe­ly clear­ing the FDA’s stat­ed bench­mark for ap­proval of 90%, Seely said. For Japan­ese and Eu­ro­pean ap­proval, they need­ed to show non-in­fe­ri­or­i­ty and did so, with their le­upro­lide arm show­ing on­ly an 88% re­sponse rate.

The re­sults set up an FDA ap­pli­ca­tion in Q2 of 2020 and Eu­ro­pean and Japan­ese ap­pli­ca­tions short­ly there­after, adding to the com­pa­ny’s im­mi­nent US ap­pli­ca­tion for uter­ine fi­broid ther­a­py.

The un­der­ly­ing mech­a­nism be­hind the two ther­a­pies is es­sen­tial­ly in­versed. Le­upro­lide is a GnRH ag­o­nist and de­sen­si­tizes the re­cep­tor by hit­ting it re­peat­ed­ly. Re­l­u­golix blocks it di­rect­ly.

My­ovant’s pitch cen­ters around the speed at which the drug worked, its pill form — rather than le­upro­lide, which is in­ject­ed every three months — and the ta­per­ing of some side ef­fects, which can be lengthy when it comes to testos­terone sup­pres­sion, in­clud­ing hot flash­es, fa­tigue, loss of mus­cle mass, sex­u­al dys­func­tion and de­pres­sion. Doc­tors have some­times giv­en “drug hol­i­days” to man­age the clin­i­cal ben­e­fit against the costs.

“Low­er­ing the testos­terone is bet­ter for treat­ing prostate can­cer,” Seely said. “But it’s not great for the man.”

The ad­verse event ra­tio for both drugs was around 93%, but Seely not­ed a low­ered rate of ma­jor car­dio­vas­cu­lar events in the re­l­u­golix group, 2.9% com­pared to 6.2%. The biotech al­so said hor­mone lev­els re­turned to nor­mal 90 days af­ter treat­ment in the re­l­u­golix group while some­times re­main­ing re­duced at that point or longer for le­upro­lide pa­tients — al­though they did not an­nounce spe­cif­ic num­bers.

So why isn’t Ab­b­Vie, My­ovant’s uter­ine ri­val, pur­su­ing a prostate treat­ment? Seely’s best guess is that it’s be­cause Ab­b­Vie’s GnRH an­tag­o­nist’s half-life is much short­er than My­ovant’s. Ab­b­Vie re­quires high­er dos­es in their uter­ine treat­ments than My­ovant gives — which would mean even high­er dos­es in the prostate ther­a­pies, she said.

The ques­tion, though, is if le­upro­lide is still large­ly ef­fec­tive and the side ef­fects most­ly the same, will doc­tors pre­scribe a new pill in­stead?

“I think the biggest con­cern is that le­upro­lide … has been in the field for decades,” she said. “Some peo­ple ques­tion if on­col­o­gists and urol­o­gists will change their prac­tice.”

Rev­o­lu­tion Med shoots for $100M+ IPO — and di­vulges some se­crets about that Warp Dri­ve buy­out

Biotech investors who like to wager on the race to the front of the KRAS market now have a new team to consider.

Revolution Medicines, which extended its reach on RAS with a deal to acquire Warp Drive Bio about 18 months ago, filed their S-1 in search of $100 million-plus. And they gave up a few secrets in the process.

The main clinical claim to fame that Revolution has centers on the SHP2 inhibitor RMC-4630, partnered with Sanofi back in the summer of 2018 — just after John Reed was named the incoming R&D chief. We already knew that the pharma giant handed over $50 million in cash plus a commitment of hundreds of millions more to align itself with Revolution as it makes a fresh foray into oncology. Now we know that Sanofi is also footing 80% of Revolution’s R&D bill on the program, while setting up a smorgasbord of $235 million in development milestones and $285 million in commercial bonuses.

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Turn­ing the cor­ner on treat­ing the root cause of sick­le cell dis­ease

Early in my career, as a medical resident, I saw first-hand the enormous challenges faced by children and adults with sickle cell disease (SCD), a genetic blood disorder that historically has lacked adequate treatment options. People living with this life-long disease are mainly those with ancestors from sub-Saharan Africa, as well as people of Hispanic, South Asian, Southern European and Middle Eastern descent. These patients suffer from devastating physical symptoms, including progressive, eventually fatal, organ damage and excruciating pain. In addition, they encounter emotional, mental and social burdens – non-physical aspects of living with SCD that also take a serious toll on patients and their caregivers.

Olivier Brandicourt, AP Images

#JPM20 ex­clu­sive: Olivi­er Brandi­court fol­lows the Big Phar­ma CEO path to pri­vate eq­ui­ty, join­ing Black­stone ahead of a mam­moth fund de­but

Nick Galakatos Blackstone

Seven months after Olivier Brandicourt’s surprise “early retirement” from Sanofi, he’s back in the game, this time taking meetings at JP Morgan to discuss his new role at Blackstone, where he’s quietly begun work with Nick Galakatos and the life sciences crew.

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Video Re­play: End­points at #JPM20 — news­mak­ers on deal­mak­ing, pric­ing and man­u­fac­tur­ing

On Monday, we held our fourth annual #JPM event — and the team hit a key milestone that I’d like to share with the entire Endpoints News audience: We live-streamed the conversation and had nearly triple the number of executives watching online than we had in the sold-out crowd of 320.

For a media company on a mission to connect the biopharma world in bigger and better ways, we’re proud of how we were able to extend the reach of our franchise event. Paid subscribers were given access to the stream in real time, and now, two days later, we’re opening it up to everyone in this post.

Endpoints@JPM: (left to right) Steve Pearson, Nick Leschly, Bari Talente, Stephen Ubl, John Carroll

#JPM20: 'The NPV is al­ways wrong.' Take­da preps an­oth­er spin­out — this time on psych

Editor’s Note: Endpoints News is reporting live from #JPM20 after kicking things off with an action-packed event, which you can replay here. What follows is a stream of tidbits we have collected while wandering around Union Square in San Francisco. Check back in throughout the week for updates by John Carroll and Jason Mast.

SAN FRANCISCO — A year ago Takeda CEO Christophe Weber and R&D chief Andy Plump arrived at JP Morgan right on the heels of closing their big Shire buyout. Now they’re back after shaking up the portfolio, boosting R&D spending by about 50% to $4.5 billion and adjusting the pipeline — a task which isn’t quite finished yet.

Nick Leschly at Endpoints News' panel at the 2020 JP Morgan Healthcare Conference. Credit: Jeff Rumans

At #JPM20, two CEOs, two rad­i­cal­ly dif­fer­ent ther­a­pies, and a fight to chase down sick­le cell

SAN FRANCISCO – Few CEOs tell a story better than bluebird’s Nick Leschly.

He cuts a Jeff Bezos figure on stage at the Colonial Room, the JP Morgan presentation hall for A-list biotechs: lean and bald, fast-talking and vest-wearing. He explains in simple language, apologizing when he has to brush on the data. It helps that he has a good story to tell.

“We treated them one time,” Leschly tells a packed crowd, gesturing to the slide behind him. “Look what happened.”

The slide shows 9 horizontal bars studded with diamonds. Each bar, he explained, represented a sickle cell patient, and each diamond represented a severe medical event, such as a pain crisis. The diamonds stud one side – before the therapy – and vanish on the other, afterward.

“A 99% reduction in these events — this is a functional cure for sickle cell disease,” Leschly says. “This is unprecedented data.”

Upstairs and an hour later, Ted Love stands before a narrow conference room in his suit and polka-dot tie. Love, the CEO of Global Blood Therapeutics, is a 60-year-old physician. His voice trails off at the end of sentences, and the story he tells is less compelling. There are no cured patients.

“This is the first drug that addresses the root cause of sickle cell disease,” Love says, speaking in front of a slide showing a white pill bottle for GBT’s new drug Oxbryta. “Right in the label, it says that this drug inhibits polymerization.”

In the 60 years after scientists discovered the cause of sickle cell, almost no treatments emerged, even as the condition debilitated hundreds of thousands of Americans, most of them black or Hispanic. But the last few years have seen a resurgence of interest as new technologies have made the disease seem newly beatable.

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Neon Ther­a­peu­tics makes one last re­treat, sell­ing it­self cheap in a bar­gain base­ment M&A deal

Crushed by weak data for what had been their lead drug, Neon Therapeutics is being bought for parts this morning.

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Mark Pruzanski

#JPM20: Af­ter a year of NASH col­laps­es, all eyes on two biotechs

SAN FRANCISCO – It’s not quite Dewey defeats Truman, but Goldman Sachs calling 2019 “The Year of NASH” may well go down in the annals of worst biotech predictions.

Goldman Sachs slapped the label on weeks before 2019’s JP Morgan conference, projecting that long-discussed treatments for the obesity-driven condition suspected to lurk in millions of Americans would begin to bear fruit and investors would move accordingly. That did not quite happen.

“If you look at 2019, it was just a string of disappointing news,” Pascal Prigent, CEO of NASH-focused biotech Genfit, told Endpoints News in an interview.

The Year of NASH, or nonalcoholic steatohepatitis, became a year of NASH failures. Gilead failed two large Phase III trials. CymaBay went from a $1 billion company to a $100 million company after they found their drug was killing patients’ liver cells. Cirius withdrew an $86 million IPO bid after a disastrous readout. Industry-wide, there were few acqusitions in a market often projected to be worth $35 billion.

Gilead, after dominating the NASH discussion at the 2019 JPM, gave one quick mention to the program in their 2020 presentation before pivoting to other drugs.

“As promising as some of the mechanisms looked in earlier stages, when push comes to shove in large study settings, they just haven’t proven out,” Mark Pruzanski, CEO of the NASH-focused biotech Intercept, told Endpoints in an interview.

As biotech turns from 2019, the failures have refocused eyes away from Gilead and back toward two startups, both facing key events in the coming months: Intercept, which first alerted investors to NASH at JPM 2014, and the France-based Genfit.

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Piaoyang Sun (ALAMY)

Chi­na’s fourth rich­est man takes a back seat at the top drug­mak­er he built

After spending the past 30 years transforming Jiangsu Hengrui Medicines from just another manufacturer of dirt cheap over-the-counter pills to a top 25 global biopharma player, Piaoyang Sun is taking a step back.

The billionaire is handing over the chairman role to Zhou Yunshu, his right-hand man and president of the company, Hengrui said in a statement. But Sun will stay on the board and continue to lead the strategy committee.