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.

“It’s a pro­tein that we’ve found to be es­sen­tial in me­di­at­ing the ac­tiv­i­ty of a tran­scrip­tion fac­tor, one that the field has tried to find med­i­cines for for quite a while,” CEO Markus War­muth told End­points News. “Be­cause of the breadth of can­cers that are de­pen­dent on that, it could be very broad.”

Wednes­day’s round was led by Ais­ling, with Ver­sant and New En­ter­prise As­so­ci­ates par­tic­i­pat­ing once again. New in­vestors in­clud­ed HBM, Cor­morant, GV, Amzak Health, Cas­din Cap­i­tal, Six­ty De­gree Cap­i­tal and Cam­bridge As­set Man­age­ment.

Monte Rosa’s plat­form re­volves around de­vel­op­ing “mol­e­c­u­lar glues” that can re­pro­gram ubiq­ui­tin lig­as­es cen­tral to pro­tein degra­da­tion. By tak­ing con­trol of this process, the biotech hopes to redi­rect the body’s nat­ur­al degra­da­tion ef­forts and elim­i­nate the pro­teins al­to­geth­er rather than mere­ly in­hib­it their func­tions.

His­tor­i­cal­ly, War­muth said, many drugs have at­tempt­ed to fol­low the in­hi­bi­tion method, but that re­quires tar­gets to have an ac­tive site. Monte Rosa’s mol­e­c­u­lar glues al­low the biotech to go af­ter tar­gets that don’t have such sites, es­pe­cial­ly when it comes to the no­to­ri­ous­ly dif­fi­cult area of tran­scrip­tion.

“Part of the ex­cite­ment around our plat­form is we can fi­nal­ly go very sys­tem­at­i­cal­ly af­ter tran­scrip­tion, with few ex­cep­tions,” War­muth said. “Try­ing to go af­ter the in­ter­ac­tion be­tween a tran­scrip­tion fac­tor and DNA has been in­her­ent­ly dif­fi­cult, and so this mol­e­c­u­lar glue-based plat­form that we have re­al­ly of­fers up that op­por­tu­ni­ty.”

War­muth com­pared Monte Rosa’s ap­proach with that of PRO­TACs, which he de­scribed as a scaf­fold, with one part bind­ing to the ubiq­ui­tin lig­ase and the oth­er to the tar­get of in­ter­est, con­nect­ed to a hy­dropho­bic link­er that unites those two paths. Though that makes the mol­e­cules rel­a­tive­ly big, this helps it in­duce changes with­in the lig­as­es that ul­ti­mate­ly dri­ve the dis­ease-fight­ing func­tion.

The best known ex­am­ple of a glue-based de­grad­er is lenalido­mide, the mul­ti­ple myelo­ma chemother­a­py drug made by Cel­gene, War­muth said. In this ap­proach, two tran­scrip­tion fac­tors are glued to­geth­er to the lig­ase and trans­formed in­to the “per­fect shape” to be de­stroyed in help­ing treat the can­cer.

Pro­tein degra­da­tion it­self isn’t all that new, but mon­ey has poured in­to the area re­cent­ly and es­pe­cial­ly this year. Back in March, the biotechs Kymera and Nurix com­bined to raise $220 mil­lion on back-to-back days, and both went pub­lic over the sum­mer. Cur­rent­ly, on­ly Arv­inas’ pro­tein de­graders, an an­dro­gen re­cep­tor-tar­get­ing drug for prostate can­cer and an es­tro­gen re­cep­tor-tar­get­ing drug for breast can­cer, have reached the clin­ic, get­ting in­to hu­man test­ing in 2019.

But what War­muth hopes sep­a­rates Monte Rosa from the pack is the spe­cif­ic fo­cus on their glue-based tech­nol­o­gy. De­spite keep­ing in­for­ma­tion about their lead pro­gram — as well as the rest of their pipeline — close to the chest, War­muth ex­pressed con­fi­dence in the plat­form and the team his com­pa­ny has as­sem­bled.

“The rea­son why we’re fo­cus­ing on it is re­al­ly this promise of now go­ing af­ter pre-clin­i­cal­ly val­i­dat­ed tar­gets, things we’ve al­ways want­ed to go af­ter but have es­caped be­cause there’s no drug­gable site,” War­muth said. “That’s re­al­ly what dis­tin­guish­es us from the field sci­en­tif­i­cal­ly.”

In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

Adam Koppel and Jeffrey Schwartz, Bain

Bain ex­ecs Adam Kop­pel and Jef­frey Schwartz line up $125M for their first blank check deal as Wall Street con­tin­ues to em­brace biotech

Adam Koppel and Jeffrey Schwartz have jumped into the blank check game, raising $125 million for a stock listing in search of a company.

Their SPAC, BCLS Acquisition Corp, raised $125 million this week, with a line on $25 million more as it scouts for a biotech in search of money and a place on Wall Street.

The two principals at Bain Life Sciences have been on a romp since they set up the Bain operation 4 years ago. Their S-1 spells out a track record of 22 deals totaling $650 million for the life sciences group, which led to 9 IPOs.

Covid-19 roundup: An mR­NA play­er gets a boost out of the lat­est round of an­i­mal da­ta; Phase­Bio pulls the plug on treat­ment tri­al

The big tell for CureVac $CVAC is coming up with a looming early-stage readout on their mRNA Covid-19 vaccine in the clinic. But for now they’ll make do with an upbeat assessment on the preclinical animal data they used to get into the clinic.

Researchers for the German biotech say they got the high antibody titers and T cell activation they were looking for, lining up a hamster challenge to demonstrate — in a simple model — that the vaccine could protect the furry creatures. Like the other mRNA vaccines, the drug sends instructions to spur cells to decorate themselves with the distinctive spike on the virus to elicit an immune response.

Chi­nese in­vestors wa­ger $105M on an IPO-bound biotech look­ing to push RNAi as main­stream can­cer ther­a­py

Shortly after Sirnaomics brought in a $47 million Series C for its small interfering RNA pipeline last year, Patrick Lu — the founder, president and CEO — was asked to outline the scientific advances that will be necessary to make better drugs out of RNA tech.

“The next step in the evolution of RNAi as a leading therapeutic will be the ability to safely target organs outside the liver such as lung, brain, etc,” he had offered. “This will revolutionize disease treatments if the industry can demonstrate similar data sets for non-liver targets as we have seen in liver-based diseases.”