Aus­tralian cell ther­a­py com­pa­ny Mesoblast joins forces with Ger­many's Grü­nen­thal to tack­le low back pain

While the FDA re­views a rolling ap­pli­ca­tion for its ex­per­i­men­tal stem cell ther­a­py for acute graft ver­sus host dis­ease (GVHD), Aus­tralia’s Mesoblast has tied up Ger­many’s pain spe­cial­ist Grü­nen­thal to de­vel­op a treat­ment for chron­ic low back pain caused by de­gen­er­a­tive disc dis­ease.

Un­der the deal, Grü­nen­thal will gain ex­clu­sive com­mer­cial­iza­tion rights to the drug, MPC-06-ID, for Eu­rope and Latin Amer­i­ca. Mesoblast, which is test­ing the drug in a US late-stage study that is ex­pect­ed to read­out next year, is get­ting $150 mil­lion in up­front and mile­stone pay­ments, in ad­di­tion to po­ten­tial roy­al­ties. Cu­mu­la­tive mile­stone pay­ments could sur­pass $1 bil­lion, the com­pa­nies said on Mon­day.

A sin­gle in­jec­tion of the drug, which is be­ing de­vel­oped for use in pa­tients who have not ben­e­fit­ed from ex­ist­ing op­tions, re­sult­ed in over a three-fold in­crease, ver­sus place­bo, in suc­cess­ful­ly achiev­ing a com­pos­ite goal in a 100-pa­tient mid-stage study. The end­point con­sist­ed of 50% im­prove­ment in low back pain and 15 point im­prove­ment in func­tion at both 12 and 24 months with no treat­ment or sur­gi­cal in­ter­ven­tions at the treat­ed lev­el through 24 months. Over­all, 37% of pa­tients treat­ed with MPC-06-ID com­pared with 10% in the con­trol group met the com­pos­ite end­point over two years, Mesoblast said.

In the Unit­ed States, the FDA has amped up its scruti­ny of stem cell clin­ics promis­ing won­der cures with­out a shred of ev­i­dence.  Mesoblast is putting its tech­nol­o­gy — which is based on mes­enchy­mal lin­eage adult stem cells (rare cells found in the vicin­i­ty of blood ves­sels that pro­mote tis­sue re­pair and mod­u­late im­mune re­spons­es) — through the clin­ic to test its vi­a­bil­i­ty across a va­ri­ety of con­di­tions such as GVHD, heart fail­ure, pain, rheuma­toid arthri­tis and Crohn’s dis­ease. The com­pa­ny’s off-the-shelf GVHD cell ther­a­py, Tem­cell, has been ap­proved in Japan (and is li­censed by part­ner JCR Phar­ma­ceu­ti­cals in the re­gion).

MPC-06-ID is de­signed as an in­tra-dis­cal in­jec­tion — en­gi­neered to de­gen­er­ate in­ter­ver­te­bral discs to al­low the mes­enchy­mal lin­eage adult stem cells (MLCs) to se­crete bio­mol­e­cules in­volved in en­hanced mi­gra­tion and pro­lif­er­a­tion of in­ter­ver­te­bral disc prog­en­i­tor cells, in a bid to strength­en the load-bear­ing func­tion of the disc by im­prov­ing its sta­bil­i­ty and en­hanc­ing its wa­ter con­tent, while al­so re­duc­ing in­flam­ma­tion and pain.

Grü­nen­thal and Mesoblast have agreed on an over­all de­vel­op­ment plan for MPC-06-ID to meet Eu­ro­pean reg­u­la­to­ry re­quire­ments, and the two part­ners will ac­cord­ing­ly work on a late-stage de­sign for Eu­rope. The re­sults of the Eu­ro­pean and US tri­als are ex­pect­ed to sup­port reg­u­la­to­ry ap­provals in both re­gions, the com­pa­nies said.

Among key char­ac­ter­is­tics of Mesoblast’s tech­nol­o­gy is that the com­pa­ny’s prod­ucts are fer­tile for sig­nif­i­cant ex­pan­sion in cul­ture, Mesoblast chief ex­ec­u­tive Sil­viu Ites­cu not­ed. “These prop­er­ties fa­cil­i­tate their use as al­lo­gene­ic, or ‘off-the-shelf,’ ther­a­peu­tics with well-de­fined re­lease cri­te­ria and batch-to-batch re­pro­ducibil­i­ty that meet strin­gent reg­u­la­to­ry re­quire­ments.”

These are some of the chal­lenges faced by the first gen­er­a­tion of cell ther­a­pies which are au­tol­o­gous CAR-T ther­a­peu­tics — No­var­tis’ $NVS Kym­ri­ah and Gilead’s $GILD Yescar­ta which se­cured FDA ap­proval in 2017.  These prod­ucts re­quire the man­u­fac­tur­ers to first iso­late cells from the pa­tient, ma­nip­u­late them in the lab by adding chimeric anti­gen re­cep­tors to di­rect T cells to snuff out can­cer cells and then re-in­fuse them back in­to the pa­tient. This process re­quires enor­mous man­u­fac­tur­ing heft — a chal­lenge that has trig­gered rel­a­tive­ly mut­ed sales for Kym­ri­ah, with No­var­tis strug­gling to meet de­mand.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Af­ter 4 years of furor, the FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. And this time they plan to squash it

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition.

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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UP­DAT­ED: Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug — and shares rock­et up

There’s no public data yet, but Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

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Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

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Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.

Sor­ren­to shrugs off an anony­mous pri­vate eq­ui­ty group’s $1B of­fer to buy the com­pa­ny

San Diego-based Sorrento Therapeutics isn’t going the M&A route — at least not today.

The biotech caused quite a stir when it put out word a few weeks ago that an unidentified private equity group was bidding a billion dollars-plus for the company. The news drove a quick spike in the company’s share price as investors hooked up for the ride — that didn’t happen.

The update sparked a 5% drop in the share price $SRNE ahead of the bell. It’s now trading just above $4, without any evidence that the $7 price looked like it was firm.