The newest, $86M Third Rock start­up chas­es the tiny bi­o­log­i­cal ma­chines in­side of you

It took 23 years from the iso­la­tion of the gene for cys­tic fi­bro­sis to the ap­proval of the first drug to tar­get it, and an­oth­er 7 for a drug that could treat the vast ma­jor­i­ty of CF pa­tients. Third Rock Ven­ture’s lat­est start­up thinks they can build sim­i­lar drugs a whole lot faster.

MO­MA Ther­a­peu­tics has raised $86 mil­lion to in­ves­ti­gate and drug a class of en­zymes known as mol­e­c­u­lar ma­chines. These pro­teins in­clude every­thing from en­zymes in­volved in DNA re­pair to the trans­port pro­teins that go awry in cys­tic fi­bro­sis — over 400 dif­fer­ent types, by MO­MA’s count. And yet, MO­MA con­tends, they have been over­looked to date, with re­searchers both fail­ing to un­der­stand them as a co­he­sive group and fail­ing to em­ploy sys­tem­at­ic ways of find­ing and drug­ging them.

Ver­tex’s CF drugs and pro­ton-pump in­hibitors, both of which go af­ter ma­chines, are red her­rings, MO­MA wrote in a blog post ac­com­pa­ny­ing the launch: Their suc­cess is a sign of both the field’s po­ten­tial and bio­phar­ma’s over­all fail­ure.

“Not on­ly do these med­i­cines fur­ther un­der­score the ther­a­peu­tic po­ten­tial of these en­zymes,” MO­MA wrote, “they al­so il­lus­trate bio­phar­ma’s rudi­men­ta­ry ap­proach­es to pros­e­cut­ing them — near­ly all were dis­cov­ered from nat­ur­al prod­ucts, through serendip­i­ty or with­out a sys­tem­at­ic ap­proach to in­ter­ro­gat­ing the bio­chem­istry of the pro­teins them­selves.”

Reid Hu­ber

Al­though the young biotech has yet to an­nounce any tar­gets, the blog post points em­pha­sizes the role of these mol­e­c­u­lar ma­chines in can­cer and rare dis­eases. Like most Third Rock star­tups, it will be led by a firm part­ner: in this case, Reid Hu­ber, who most re­cent­ly served as CSO of In­cyte. And the com­pa­ny’s R&D ef­forts will be led by Blue­print vet­er­ans Christoph Lengauer and Tim Guzi, who step in as CSO and SVP of drug de­vel­op­ment, re­spec­tive­ly.

The com­pa­ny’s ap­proach comes large­ly out of the Howard Hugh­es Med­ical In­sti­tute, where Dorothee Kern, Eva No­gales and Jo­hannes Wal­ter col­lab­o­rat­ed. A fourth founder, Third Rock en­tre­peneur-in-res­i­dence Timur Yusufzai, will head up pro­tein sci­ences at the biotech.

Dorothee Kern

Mol­e­c­u­lar ma­chines are, as the New York Times once put it, the world’s small­est me­chan­i­cal de­vices. Sci­en­tists have just be­gun syn­the­siz­ing ar­ti­fi­cial ones, but hun­dreds of dif­fer­ent types of these puny mo­tors are run­ning in the body at every minute, break­ing down and build­ing DNA, fer­ry­ing salt and wa­ter in­to and out of cells, forc­ing mus­cles to re­lax and con­tract.

Nor­mal­ly, these mo­tors func­tion by shift­ing be­tween iso­forms — dif­fer­ent struc­tures with the same chem­i­cal make­up. But a sin­gle mu­ta­tion, such as those seen on the CFTR mol­e­c­u­lar ma­chine in cys­tic fi­bro­sis, can act like a faulty valve, caus­ing var­i­ous de­grees of mal­func­tion. Tu­mors, mean­while, can some­times de­pend heav­i­ly on one of these mo­tors to grow.

Their struc­tur­al changes are their weak­ness, Hu­ber told End­points News. Al­though all pro­teins change shape, mol­e­c­u­lar ma­chines do so at an un­par­al­leled scale. MO­MA will search for those ma­chines with ge­nom­ic da­ta and try to use small mol­e­cules to in­ter­vene, po­ten­tial­ly cor­rect­ing mal­func­tion in rare dis­eases and crip­pling tu­mors.

Think of it like the dif­fer­ence be­tween walk­ing to the mail­box at the end of the dri­ve­way vs. get­ting in a car and dri­ving to Ohio — mol­e­c­u­lar ma­chines are the lat­ter. They open, they close, they twist, they slide, they un­wind on a mas­sive scale — all in or­der to pro­duce work,” Hu­ber wrote in an email. “This unique de­pen­dence on such large changes in shape pro­vides the key op­por­tu­ni­ty for MO­MA Tx — if we can dis­rupt those changes with small mol­e­cules, we may be able to dis­cov­er nov­el med­i­cines and do it in a sys­tem­at­ic way across the en­tire class.”

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

(L-R) Dr. Robert Redfield, director of the Centers for Disease Control and Prevention; Dr. Anthony Fauci, director of the National Institute for Allergy and Infectious Diseases; Adm. Brett P. Giroir, Assistant Secretary for Health; and Dr. Stephen M. Hahn, commissioner of the Food and Drugs Administration prepare to testify at a hearing of the House Committee on Energy and Commerce on Capitol Hill on June 23, 2020 in Washington, DC. The committee is investigating the Trump administration's response to the COVID-19 pandemic. (Photo by Kevin Dietsch-Pool/Getty Images)

Trump neuters FDA's at­tempt to strength­en vac­cine guide­lines, call­ing it 'ex­treme­ly po­lit­i­cal'

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

Jim Roberts and Brian Finrow (Lumen Bioscience)

With a $4M fed­er­al grant, Lu­men jumps in­to the Covid-19 treat­ment race

It’s been less than a month since Lumen Bioscience announced a $16 million Series B to engineer spirulina — a nutrient-packed super food — for diseases like traveler’s diarrhea, norovirus and C. difficile colitis. And now, the biotech has pulled in another $4 million to do the same for Covid-19.

The approach is quite similar to other gastrointestinal targets the company is pursuing, co-founders and Brian Finrow and Jim Roberts said. The Seattle-based company is working on a camelid antibody cocktail to combat GI infection common among Covid-19 patients. In a study published in the American Journal of Gastroenterology, a majority of Covid-19 patients showed GI and respiratory symptoms, and 25% had only GI symptoms.

CEO Markus Warmuth (Monte Rosa)

Monte Rosa rakes in $96M Se­ries B as it pre­pares 'mol­e­c­u­lar glue' plat­form for IND-en­abling stud­ies

About four months after completing an extension to its Series A, Monte Rosa Therapeutics is putting its next foot forward with another heap of cash.

The Boston-based biotech is back with $96 million in Series B financing with a goal to get its lead program ready for IND-enabling studies by the end of the year. Though Monte Rosa is keeping its specific target a secret for now, the company has been researching how to utilize its protein degradation technology in breast cancer and non-small cell lung cancer, among other areas.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Gene Wang, Immetas co-founder and CEO (file photo)

Im­metas Ther­a­peu­tics nabs $11M Se­ries A to nar­row their bis­pe­cif­ic work tar­get­ing in­flam­ma­tion in age-re­lat­ed dis­eases

How does a biotech celebrate its two-year anniversary? For Immetas Therapeutics, it’s with an $11 million Series A round and a game plan to fight age-related disease.

Co-founders Gene Wang and David Sinclair came together years ago around the idea that inflammation is the ultimate process driving age-related illnesses, including cancer. The duo launched Immetas in 2018 and packed the staff with industry experts. Wang, who says he’s always had an entrepreneurial spirit, has held lead roles at Novartis, GSK, Bristol Myers Squibb and Merck. He’s worked on blockbuster drugs like Humira, Gardasil, Varubi and Zolinza. And now, he’s channeling that spirit as CEO.