Joe Wiley, Amryt CEO

A biotech with a yen for pricey rare dis­ease drugs — and bar­gain base­ment shop­ping — adopts an­oth­er or­phan in lat­est M&A pact

Af­ter mak­ing it through a long, painful haul to get past a CRL and on to an FDA ap­proval last sum­mer, lit­tle Chi­as­ma has found a buy­er.

Am­ryt $AMYT, a com­pa­ny known for its ap­petite for ac­quir­ing ex­pen­sive drugs for rare dis­eases at bar­gain prices, snagged Chi­as­ma and its acromegaly drug My­capssa (oc­treotide) cap­sules in an all-stock deal — with an ex­change of 0.396 shares of Am­ryt for every share of Chi­as­ma.

The Need­ham, MA-based Chi­as­ma $CHMA launched My­capssa last Sep­tem­ber, but record­ed less than $2 mil­lion in sales for Q1. Now Am­ryt says it can carve out $50 mil­lion in an­nu­al “syn­er­gies” with the deal as it us­es its in-house group to mar­ket the drug and look for an ex­pand­ed la­bel. That may hit the staff of 84 em­ploy­ees at Chi­as­ma.

Chi­as­ma start­ed the day with a mar­ket cap of $164 mil­lion, with shares shoot­ing up 44% on news of the buy­out.

Chi­as­ma had crashed close to 5 years ago when the FDA re­ject­ed My­capssa and sent the com­pa­ny back to the draw­ing board af­ter ques­tion­ing the de­sign of their piv­otal study. Their drug is an oral ver­sion of so­mato­statin ana­logues used to treat an over­pro­duc­tion of growth hor­mones that leads to or­gan growth.

Af­ter they fi­nal­ly won their OK, the com­pa­ny rolled it out at a cost of $5,152 for a 28-day course of ther­a­py at the start­ing dose.

Am­ryt — with a mar­ket cap of $351 mil­lion — picked up some ex­tra at­ten­tion last fall when the com­pa­ny an­nounced pos­i­tive top-line re­sults for its drug Fil­su­vez for epi­der­mol­y­sis bul­losa, though it sparked plen­ty of ques­tions about a fail­ure on the sec­ondary side. The biotech is per­haps best known for their ac­qui­si­tion of Aege­ri­on, which net­ted two of the world’s most ex­pen­sive ther­a­pies: Myalept and Jux­tapid. They fol­lowed that deal by swift­ly up­ping the price by 9.99%.

The buy­er is now paint­ing in some block­buster num­bers around its new drug, though it’s vir­tu­al­ly start­ing from scratch on mar­ket­ing.

Am­ryt CEO Joe Wi­ley not­ed:

We see sig­nif­i­cant rev­enue growth op­por­tu­ni­ties for My­capssa in acromegaly and are al­so very ex­cit­ed to fur­ther de­vel­op the po­ten­tial for My­capssa in pa­tients with car­ci­noid symp­toms stem­ming from NET where we be­lieve the com­mer­cial op­por­tu­ni­ty is sig­nif­i­cant.

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.

Benjamine Liu, TrialSpark CEO

Paul Hud­son and Tri­alSpark's mu­tu­al de­sire to speed up de­vel­op­ment con­verges in three-year, six-drug goal

A unicorn startup that originally set out to hasten clinical studies for biopharma partners dug further into its revised path of internal drug development by linking arms with Sanofi in a pact that the biotech’s CEO said originated from the top.

TrialSpark and the Big Pharma on Tuesday committed to in-licensing and/or acquiring six Phase II/Phase III drugs within the next three years.

“I’ve known Paul Hudson for a while and we were discussing the opportunity to really re-imagine a lot of different parts of pharma,” TrialSpark CEO Benjamine Liu told Endpoints News, “and one of the things that we discussed was this opportunity to accelerate the development of new medicines in mutual areas of interest.”

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Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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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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Dave Marek, Myovant CEO

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

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

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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Look­ing to push CAR-T in sol­id tu­mors, Bay Area biotech goes pub­lic in SPAC flip — with slight name change

SPACs might be slowly creeping back.

Monday evening, Estrella Biopharma said it was going public via a SPAC deal with TradeUP Acquisition Corp. The deal is set to close in the first half of 2023, and if all goes as planned, the public version of Estrella — dubbed Estrella Immunopharma — will be worth around $398.5 million.

The Bay Area biotech will also get around $45.4 million in cash, and TradeUp stockholders will get around 15% stock in the public biotech.

Al Sandrock, Voyager Therapeutics CEO

Af­ter 12 months of dig­ging, Pfiz­er opts for one of two Voy­ager cap­sids for gene ther­a­py

The path at Voyager Therapeutics keeps winding, and at the 12-month deadline, Pfizer has elected to only move forward with one of two capsids out of the preclinical biotech for the Big Pharma’s AAV gene therapies.

Last October, the Big Pharma and biotech linked arms on a deal that gave Pfizer the ability to hit the gas pedal on two capsids: one for a cardiac target and the other for a rare neurologic disease target.

Take­da to pull key hy­poparathy­roidism drug from the mar­ket en­tire­ly by end of 2024 af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024.

The decision to not re-commercialize Natpara will be a blow to not only the 2,400 people who were awaiting supplies of their reliable injection since 2019, but also the additional nearly 400 people who were accessing the drugs via the company’s Special Use Program as Takeda sought to resolve these manufacturing issues over the past five years.

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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