My­ovan­t's uter­ine fi­broid drug looks com­pet­i­tive in PhI­II — but can they van­quish mighty Ab­b­Vie?

Vivek Ra­maswamy’s My­ovant $MY­OV has close­ly matched its pos­i­tive first round of Phase III da­ta for their uter­ine fi­broid drug re­l­u­golix, set­ting up a head-to-head ri­val­ry with phar­ma gi­ant Ab­b­Vie as the lit­tle biotech steers to the mar­ket with a planned fil­ing in Q4.

Vivek Ra­maswamy

Here’s how My­ovant plans to pre­vail over the Ab­b­Vie $AB­BV em­pire.

In the study, 71.2% of women re­ceiv­ing once-dai­ly re­l­u­golix com­bi­na­tion ther­a­py achieved the clin­i­cal re­sponse they were look­ing for, com­pared to on­ly 14.7% in the con­trol arm. The da­ta com­fort­ably re­flect­ed the same out­comes in the first Phase III — 73.4% of women re­ceiv­ing once-dai­ly oral re­l­u­golix com­bi­na­tion ther­a­py achieved the re­spon­der cri­te­ria com­pared with 18.9% of women re­ceiv­ing place­bo — which will re­as­sure reg­u­la­tors that they are get­ting the care­ful­ly ran­dom­ized da­ta that qual­i­fies for the FDA’s gold stan­dard for suc­cess.

The suc­cess here, though, has to be com­pared with Ab­b­Vie’s elagolix, ap­proved last year for en­dometrio­sis and al­so on track for a date with the FDA on uter­ine fi­bro­sis — a con­di­tion that caus­es heavy men­stru­al bleed­ing among a por­tion of the pa­tients who have fi­broids. Ab­b­Vie’s Phase II­Is pro­duced clin­i­cal im­prove­ments for 76% and 68.5% of pa­tients.

Charles As­ch­er-Walsh Mount Sinai

A cou­ple of months ago, re­searchers pro­vid­ed an out­line of elagolix side ef­fects, though, that gave ex­perts in the field cau­tion about just how wide­ly sought af­ter the drug may be. In ad­di­tion to the loss of bone den­si­ty, the side ef­fects al­so in­clud­ed a pro­file sim­i­lar to menopause, with hot flash­es and night sweat. That could per­suade all but the most se­ri­ous cas­es to avoid the go­nadotropin-re­leas­ing hor­mone (GnRH) an­tag­o­nist.

“Women are will­ing to put up with [fi­broids] for years, so the idea of tak­ing a med­ica­tion that will have sig­nif­i­cant side ef­fects I don’t think is of­ten that pop­u­lar. [Symp­toms] are not as dra­mat­ic as the de­bil­i­tat­ing pain from en­dometrio­sis,” Charles As­ch­er-Walsh of Ic­ahn School of Med­i­cine at Mount Sinai told Med­Page To­day.

My­ovant, mean­while, said that there were no sig­nif­i­cant dif­fer­ences in terms of bone min­er­al den­si­ty with a sim­i­lar ef­fect in terms of hot flash­es — 5.6% for the drug ver­sus 3.9% for the place­bo.

Cowen’s Phil Nadeau sized it up and likes My­ovant’s chances of carv­ing out a block­buster fran­chise:

We think that to­day’s da­ta from LIB­ER­TY 2 cor­rob­o­rates the LIB­ER­TY 1 re­sults and con­firms that re­l­u­golix+add-back ther­a­py has a com­pet­i­tive pro­file with sim­i­lar ef­fi­ca­cy, some­what bet­ter tol­er­a­bil­i­ty, and im­proved con­ve­nience com­pared to Ab­b­Vie’s Orilis­sa. There­fore we con­tin­ue to be­lieve that re­l­u­golix has $1B+ sales po­ten­tial in uter­ine fi­borids.

Draw­ing dis­tinc­tions be­tween the two could be tough in this mar­ket for a com­pa­ny that doesn’t have near the same kind of com­mer­cial punch as Ab­b­Vie. But My­ovant has a com­pet­i­tive pro­file. In­vestors bid their shares up 10% this morn­ing in some­thing less than an en­thu­si­as­tic em­brace for the ri­val­ry ahead — then saw that melt away to a 1% drop in the ear­ly af­ter­noon.

So­cial im­age: Vivek Ra­maswamy at the US-Chi­na Bio­phar­ma In­no­va­tion and In­vest­ment Sum­mit in Shang­hai on Oc­to­ber 23, 2018; Cred­it: End­points News, Pharm­Cube

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

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.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,900+ biopharma pros reading Endpoints daily — and it's free.

Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll