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.

George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.

Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.

Randy Schatzman, Bolt CEO (Bolt Biotherapeutics)

Bolt Bio­ther­a­peu­tics nabs $93.5M to push Provenge in­ven­tor's new idea deep­er in the clin­ic

A cancer-fighting concept from the inventor of the first cancer vaccine is nearing prime time, and its biotech developer has received a significant new infusion of cash to get it there.

Bolt Biotherapeutics announced a $93.5 million Series C round led by Sofinnova Investments and joined by more than 9 others, including Pfizer Ventures and RA Capital Management. That money will go toward pushing the San Francisco biotech’s platform of innate immune-boosting warheads through its first trial on metastatic solid tumors and into several more.

Josh Cohen, Justin Klee

Armed with pos­i­tive ALS da­ta, Amy­lyx scores $30M in fresh fund­ing to com­plete Alzheimer's PhII

Four years after announcing themselves to the biotech world with a new idea for drugging neurodegeneration, backing by the late Henri Termeer and $5 million from Morningside Venture, the young entrepreneurs at Amylyx are back for round 2.

Morningside continued to lead the $30 million Series B, with participation from Termeer’s widow, Belinda, and other unnamed investors. Having celebrated a topline Phase II win for its lead program in amyotrophic lateral sclerosis, Amylyx expects the cash to fund talks with regulators as well as a separate trial for the same drug in Alzheimer’s — for which they had just finished enrolling.

An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.

Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.

New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

Anthony Johnson, Goldfinch Bio CEO (Goldfinch via YouTube)

Gilead-part­nered Goldfinch Bio lands $100M as it piv­ots to clin­i­cal stage biotech

A year after landing $109 million in cash for an early-stage discovery deal with Gilead, Goldfinch Bio has scored a similar bounty from investors, raising $100 million in the biotech’s first financing round since its 2016 launch.

The new round, led by Eventide Asset Management and joined by 8 other firms, confirms that last year’s Gilead deal began a new, busier, more vocal era for the company. After launching out of Third Rock with $55 million and a plan to tackle kidney disease, they focused on preclinical research and made few material announcements. But the latest press release is full of plans, including their first Phase II study and a new therapy scheduled to enter the clinic next year.