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.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll