Fre­quen­cy fol­lows Astel­las pact with $62M round to bankroll PhII of lead re­gen­er­a­tive drug

A few days ago Fre­quen­cy Ther­a­peu­tics got some high pro­file en­dorse­ment on its small mol­e­cule strat­e­gy for re­gen­er­a­tive med­i­cine, when Astel­las of­fered $80 mil­lion up­front to part­ner on their lead drug. As it turns out, the Japan­ese phar­ma wasn’t the on­ly one in­trigued by the plat­form and up­com­ing Phase II study of FX-322.

David Lucchi­no

Per­cep­tive Ad­vi­sors has jumped in to lead a $62 mil­lion Se­ries C, which al­so drew Deer­field Man­age­ment, RTW In­vest­ments and Mizuho Se­cu­ri­ties Prin­ci­pal In­vest­ment. Po­laris, Tai­wa­nia, Ax­il and Co­Bro al­so dou­bled down on their ex­ist­ing bet.

Con­sid­er­ably larg­er than pre­vi­ous rounds — which to­taled $147 mil­lion — the new fi­nanc­ing will fund an im­por­tant tri­al to tease out FX-322’s ef­fects on sen­sorineur­al hear­ing loss, hav­ing re­cent­ly wrapped a safe­ty and tol­er­a­bil­i­ty test, ac­cord­ing to CEO David Lucchi­no.

“In the fu­ture, we may eval­u­ate our ap­proach in oth­er hear­ing in­di­ca­tions and we con­tin­ue to study a broad ar­ray of dis­ease ar­eas where our plat­form may have ap­pli­ca­tions,” the com­pa­ny said.

First de­vel­oped by MIT’s Bob Langer and Jeff Karp, Fre­quen­cy’s plat­form cen­ters on dis­patch­ing small mol­e­cule drugs to ac­ti­vate prog­en­i­tor cells, push­ing them to dif­fer­en­ti­ate in­to tar­get cells (hair cells in the in­ner ear in the case of FX-322). By tin­ker­ing with lo­cal cells that are pre-pro­grammed to serve the pur­pose, the biotech be­lieves it can of­fer a safer and more ef­fi­ca­cious al­ter­na­tive to stem cell ther­a­pies.

Bill Chin

That can lead to some am­bi­tious new tar­gets, in­clud­ing dis­eases of the mus­cle, gas­troin­testi­nal tract, skin and bone. Fre­quen­cy is al­so look­ing to bring a dis­cov­ery pro­gram fo­cused on re­myeli­na­tion in mul­ti­ple scle­ro­sis in­to the clin­ic.

He’s stay­ing mum about the ex­act de­sign of the up­com­ing hear­ing loss tri­al for now. But if the scant de­tails from the Phase I/II read­out were any hint, the team led by CMO Bill Chin could be look­ing for more signs that the drug can im­prove au­diom­e­try and word scores.

So­cial im­age: Small mol­e­cules trig­ger­ing prog­en­i­tor cells to dif­fer­en­ti­ate. Fre­quen­cy via YouTube

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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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.

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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 with any issues.

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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.

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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