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.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

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But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP)

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

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Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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Silviu Itescu, Mesoblast CEO

Mesoblast sends in im­proved po­ten­cy as­say, look­ing to re­sub­mit to FDA on acute graft-ver­sus-host dis­ease drug

In 2020, the FDA rejected Mesoblast’s remestemcel-L, or Ryoncil, its lead candidate for pediatric acute graft-versus-host disease (aGVHD) that didn’t respond to steroids. The FDA raised a number of concerns, first objecting to Mesoblast’s single arm, open-label trial, though regulators struggled to describe how a randomized trial would work, since pediatricians and parents were reluctant to put children with aGVHD in a placebo arm.

Johnny Stilou, Scandion Oncology acting CEO

Scan­dion's shares fall af­ter on­col­o­gy biotech re­ports PhII fail

Danish biotech Scandion Oncology posted some Phase II results on Friday, and investors were none too pleased.

The biotech reported topline results from the second part of an ongoing Phase II trial called CORIST. The study was investigating Scandion’s lead candidate SCO-101 in 25 patients as a combination treatment with FOLFIRI chemotherapy in metastatic colorectal cancer. And so far, the study did not meet the biotech’s primary endpoint: tumor reduction of at least 30%.

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Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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