Tar­get­ing un­der­ly­ing cause of PAH, Cam­bridge spin­out Mor­phogen-IX rais­es about $23M in Se­ries B

Back in the year 2000, a ma­jor mu­ta­tion in fa­mil­ial pul­monary ar­te­r­i­al hy­per­ten­sion (PAH) was dis­cov­ered in the re­cep­tor for BMP9 — and for years sci­en­tists in­ves­ti­gat­ed how this mu­ta­tion caus­es PAH and whether that in­for­ma­tion could be used to treat the de­bil­i­tat­ing dis­ease. This cul­mi­nat­ed in a man­u­script in Na­ture Med­i­cine in 2015, and served as the foun­da­tion for the com­pa­ny Mor­phogen-IX — which on Tues­day said it had raised £18.4 mil­lion ($23.2 mil­lion) in a Se­ries B round of fi­nanc­ing. 

Nick Mor­rell

The name Mor­phogen-IX (pro­nounced mor­phogenix) em­anates from BMP9, which is a bone mor­pho­genet­ic pro­tein. The mon­ey will be used to sup­port for­mal pre­clin­i­cal de­vel­op­ment of the com­pa­ny’s lead can­di­date — dubbed MGX292 — and the ini­ti­a­tion of hu­man stud­ies by 2021, the com­pa­ny said.

MGX292 is a pro­tein en­gi­neered vari­ant of the nat­u­ral­ly oc­cur­ring BMP9. The ap­proach was pi­o­neered by the British Heart Foun­da­tion-fund­ed lab­o­ra­to­ry of Pro­fes­sor Nick Mor­rell at the Uni­ver­si­ty of Cam­bridge, who now serves as the com­pa­ny’s CEO.

“The ge­net­ic val­i­da­tion for the use of BMP9 in PAH is over­whelm­ing.  We al­so dis­cov­ered that mu­ta­tions in BMP9 it­self cause PAH ear­li­er this year,” Mor­rell told End­points News.

PAH is a rare, pro­gres­sive dis­ease char­ac­ter­ized by nar­row­ing and oblit­er­a­tion of small pul­monary ar­ter­ies, re­sult­ing in a se­vere el­e­va­tion in ar­te­r­i­al pres­sure and ul­ti­mate­ly right heart fail­ure. Mor­tal­i­ty rates re­main high de­spite cur­rent­ly li­censed ther­a­pies that tar­get vaso­con­stric­tion. In cer­tain pa­tients, the dis­ease has a ge­net­ic cause that re­duces the pro­tec­tive func­tion of the BMP9 pro­tein in blood, and some 25% of pa­tients with id­io­path­ic and fa­mil­ial PAH car­ry mu­ta­tions in com­po­nents of the BMP9 sig­nal­ing path­way, the com­pa­ny es­ti­mat­ed.

“The prob­lem is that the dis­ease is not char­ac­terised by vaso­con­stric­tion — it is the re­sult of pro­found en­dothe­lial dys­func­tion and struc­tur­al al­ter­ations in the vas­cu­lar wall. The mor­tal­i­ty in PAH re­mains 50% at 5 years de­spite com­bi­na­tions of the avail­able drugs. There is thus a huge un­met med­ical need for drugs that are tru­ly dis­ease mod­i­fy­ing that tar­get the mol­e­c­u­lar and cel­lu­lar path­ways in­volved in the dis­ease, ex­em­pli­fied by BMP9,” Mor­rell said.

David Grainger

The biotech, which was spun out of Cam­bridge En­ter­prise, raised £1.5 mil­lion in a Se­ries A round back in 2015. This lat­est round of fund­ing was led by Medicxi and in­clud­ed the par­tic­i­pa­tion of Cam­bridge In­no­va­tion Cap­i­tal and Cam­bridge En­ter­prise.

“The pow­er of hu­man ge­net­ics iden­ti­fy­ing the cen­tral­i­ty of BMP9 in PAH, to­geth­er with struc­ture-dri­ven en­gi­neer­ing to cre­ate a pro­tein that can be ad­min­is­tered safe­ly, places Mor­phogen-IX in a world-lead­ing po­si­tion to de­vel­op the first agent ca­pa­ble of halt­ing, or even re­vers­ing, the progress of this ter­ri­ble dis­ease.  We are work­ing with many of world’s lead­ing ex­perts in PAH to get MGX292, al­ready the sub­ject of patent ap­pli­ca­tions, in­to the clin­ic as quick­ly as pos­si­ble,” said David Grainger, chair­man of the Mor­phogen-IX board and chief sci­en­tif­ic ad­vi­sor at Medicxi, in a state­ment.

For Mor­rell, the tran­si­tion from sci­en­tist to CEO has been in­ter­est­ing. “I am 50% CEO and 50% aca­d­e­m­ic. As com­pa­ny CEO I get to lead sci­ence that re­al­ly mat­ters to pa­tients with PAH, as well as lead­ing on BD and com­mer­cial stuff that I find re­al­ly fas­ci­nat­ing (and chal­leng­ing!)” he said.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,000+ biopharma pros reading Endpoints daily — and it's free.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,000+ biopharma pros reading Endpoints daily — and it's free.

Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,000+ biopharma pros reading Endpoints daily — and it's free.

Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,000+ biopharma pros reading Endpoints daily — and it's free.

Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

UP­DAT­ED: With loom­ing ‘apoc­a­lypse of drug re­sis­tance,’ Mer­ck’s com­bi­na­tion an­tibi­ot­ic scores FDA ap­proval on two fronts

Merck — one of the last large biopharmaceuticals companies in the beleaguered field of antibiotic drug development — on Wednesday said the FDA had sanctioned the approval of its combination antibacterial for the treatment of complicated urinary tract and intra-abdominal infections.

To curb the rise of drug-resistant bacteria and maintain the efficacy of the therapy, Recarbrio (and other antibacterials) — the drug must be used to treat or prevent infections that are proven or strongly suspected to be caused by susceptible gram-negative bacteria, Merck $MRK said.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,000+ biopharma pros reading Endpoints daily — and it's free.