Sen­a­tors ac­cuse Te­va, My­lan of stonewalling price fix­ing probe; Myr­i­ad Ge­net­ics shares skid on dis­mal quar­ter­ly re­sults

→ US law­mak­ers have writ­ten to Te­va $TE­VA, My­lan $MYL and pri­vate­ly-held Her­itage Phar­ma­ceu­ti­cals ask­ing for doc­u­ments as part of a sweep­ing con­gres­sion­al probe in­to gener­ic drug price hikes, ac­cus­ing the com­pa­nies of “ap­par­ent ef­forts to stonewall” the in­ves­ti­ga­tion, Reuters re­port­ed. The probe was first launched in 2014, and ear­li­er this year 44 US states filed a law­suit al­leg­ing drug price-fix­ing by the trio of drug­mak­ers. Now, sen­a­tors Eli­jah Cum­mings and Bernie Sanders are in­ves­ti­gat­ing a pur­port­ed “co­or­di­nat­ed ob­struc­tion” of the probe, cit­ed in the State’s com­plaint, the re­port said.

→ Shares of mol­e­c­u­lar di­ag­nos­tic test mak­er Myr­i­ad Ge­net­ics $MYGN tum­bled about 34% to $29.41 on Wednes­day morn­ing, af­ter the com­pa­ny post­ed dis­ap­point­ing fis­cal fourth quar­ter re­sults and ini­tial 2020 guid­ance. “MYGN once again faced a num­ber of neg­a­tive sur­pris­es in the quar­ter dri­ven by im­pact of LBM (Lab Ben­e­fit Man­agers) on its Car­ri­er test­ing busi­ness com­bined with re­cent­ly height­ened FDA scruti­ny of phar­ma­coge­nom­ic test­ing (Gen­e­Sight). MYGN con­tin­ues to dis­agree on changes re­quired to the test, and we be­lieve that will con­tin­ue to re­main an over­hang un­til re­solved,” SVB Leerink an­a­lysts wrote in a note.

→ With the brief stint at Ac­celeron be­hind him, Robert Zeldin has found his next CMO role at Roivant’s new au­toim­mune-fo­cused off­shoot, Im­muno­vant. The ap­point­ment brings Zeldin back to his roots in im­munol­o­gy and lever­ages his more re­cent ex­pe­ri­ence with clin­i­cal de­vel­op­ment to ad­vance IMVT-1401, an an­ti­body that fa­cil­i­tates the degra­da­tion of path­o­gen­ic IgG. Brad­ford Mid­dlekauff, a Medarex alum and biotech vet, is al­so join­ing Pe­te Salz­mann’s team as gen­er­al coun­sel.

→ Rare dis­ease drug de­vel­op­er Akari Ther­a­peu­tics has won fast track sta­tus from the FDA for its lead ex­per­i­men­tal drug, no­ma­co­pan, for hematopoi­et­ic stem cell trans­plant-as­so­ci­at­ed throm­bot­ic mi­croan­giopa­thy (HSCT-TMA) in pe­di­atric pa­tients. The con­di­tion, for which there are no ap­proved ther­a­pies, has an es­ti­mat­ed 80% mor­tal­i­ty rate. Akari’s shares $AK­TX jumped about 34% to $2.41 in ear­ly trad­ing. The com­pa­ny has re­cent­ly un­der­gone a pe­ri­od of up­heaval — last year, Nas­daq threat­ened to scrap its po­si­tion on the ex­change af­ter Akari failed to file its an­nu­al re­port on time, days af­ter the New York City biotech oust­ed its for­mer CEO David Horn Solomon for al­leged­ly abus­ing the com­pa­ny cred­it card.

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