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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.

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Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.

John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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