High­land who? Biotech gets a $200M war chest to crack the US AD­HD mar­ket

Craig Lewis, Iron­shore Phar­ma­ceu­ti­cals

At sev­er­al points last year, Toron­to-based High­land Ther­a­peu­tics spot­light­ed da­ta from a pair of piv­otal Phase III stud­ies of Ben­jor­na (HLD-200) which they say pro­vid­ed clear ev­i­dence that a night­ly dose of the drug upped the per­for­mance of kids with AD­HD from morn­ing through night. Now, star­ing down a Ju­ly 30 PDU­FA date with the FDA, the low-pro­file biotech has raised $200 mil­lion to fund the com­mer­cial ramp-up, con­fi­dent that they’re now a few short steps from shak­ing up a ma­jor mar­ket.

The mon­ey comes from Mor­gan Stan­ley, which sold se­nior se­cured notes due in 2024 for a whol­ly owned sub­sidiary of High­land’s — Iron­shore Phar­ma­ceu­ti­cals & De­vel­op­ment. In­vestors are gam­bling that the biotech can make the dif­fi­cult switch from drug de­vel­op­er to com­mer­cial­iza­tion group, while fac­ing off against heavy­weight play­ers in the field, with plans to mar­ket the drug to a big mar­ket through­out the US.

In the sec­ond Phase III study of 161 kids aged 6 to 12, in­ves­ti­ga­tors said that they had tracked an av­er­age 44% im­prove­ment in symp­toms com­pared to a place­bo. But it’s the full-day ben­e­fit they tout­ed the hard­est. Their “be­fore school” rat­ing achieved a 59% im­prove­ment, mak­ing it eas­i­er for the kids to get ready for their day, with­out a lot of parental help. In the evening, there was a 44% im­prove­ment.

That’s key for the field, where lead­ers like Shire have been work­ing on new sec­ond-gen drugs that can of­fer a sin­gle dai­ly ther­a­py with con­sis­tent ben­e­fits.

Just days ago Shire had its long-de­layed SHP465 ac­cept­ed for a re­view at the FDA. Orig­i­nal­ly filed way back in 2006, but sub­se­quent­ly re­ject­ed, the drug has the same ac­tive in­gre­di­ent as the now gener­ic Adder­all XR, but re­for­mu­lat­ed, in­ves­ti­ga­tors say, to make it a once-dai­ly ther­a­py, boost­ing its ef­fi­ca­cy from 12 hours to 16 hours.

Just what pay­ers may thing about these drugs, though, has yet to be seen. In­sur­ers have honed their skills at keep­ing mem­bers on cheap gener­ic drugs, re­gard­less of dos­ing ben­e­fits. And these fol­lowup drugs may face a tough fight.

High­land, though, be­lieves it’s on the verge of a big break­through.

Craig Lewis, pres­i­dent of Iron­shore Phar­ma­ceu­ti­cals Inc., said:

“As a sci­ence-based, pa­tient- cen­tric or­ga­ni­za­tion, we be­lieve HLD200 has the po­ten­tial to be­come the stan­dard of care in the treat­ment of AD­HD.

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 help@endpointsnews.com with any issues.

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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

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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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.