Poised to launch its first mar­ket­ing ef­fort, Al­ny­lam makes a big leap in­to CNS dis­eases and Alzheimer's

With its lead RNAi drug poised for an his­toric ap­proval, Al­ny­lam $AL­NY is now lay­ing plans to cre­ate a pipeline of CNS drugs that will tar­get some of the tough­est dis­eases that face re­searchers to­day — in­clud­ing Alzheimer’s, the Night King of dis­eases.

Kevin Fitzger­ald

Al­ny­lam re­searchers are fol­low­ing up on a ro­dent study where they say they were able to de­liv­er a small in­ter­fer­ing RNA drug — or siR­NA —in­to the brain and spinal cord area. Us­ing in­trathe­cal in­jec­tion — cir­cum­vent­ing the blood-brain bar­ri­er — the biotech says that they were able to durably si­lence a dis­ease tar­get gene tran­script, of­fer­ing proof of prin­ci­ple ev­i­dence that they could suc­ceed where a long queue of con­tenders have failed.

Us­ing new tech­nol­o­gy, Al­ny­lam says the plan now is to se­lect its first CNS can­di­date lat­er this year, with a grow­ing ef­fort to go af­ter a string of tough-to-beat dis­eases, in­clud­ing Alzheimer’s, Hunt­ing­ton’s, and Parkin­son’s.

“We have now ap­plied our learn­ings, in­clud­ing ad­di­tion­al chem­istry ad­vances, to en­able de­liv­ery of siR­NAs be­yond the liv­er to the CNS, where there are a large num­ber of un­met needs well suit­ed for RNAi ther­a­peu­tics. As we be­gin to ad­vance our CNS pipeline, ini­tial ef­forts are fo­cused on ge­net­i­cal­ly val­i­dat­ed CNS tar­gets, use of bio­mark­ers for ini­tial proof-of-con­cept, and dis­ease set­tings with high un­met need and a de­fin­able path to reg­u­la­to­ry ap­proval and pa­tient ac­cess,” said Kevin Fitzger­ald, the se­nior VP of re­search at Al­ny­lam. 

Alzheimer’s in par­tic­u­lar has proven to be the tough­est tar­get in bio­phar­ma. Mer­ck’s re­cent fail­ure with a BACE1 in­hibitor led the phar­ma gi­ant to con­clude that that ap­proach — elim­i­nat­ing the flow of amy­loid be­ta in­to the brain — will like­ly not work in symp­to­matic pa­tients. Their re­searchers al­so con­ced­ed that the whole amy­loid be­ta the­o­ry could be noth­ing but a fic­tion that drove bil­lions of dol­lars of mis­spent re­search in­vest­ments.

The fo­cus now has shift­ed more to­ward pre-symp­to­matic pa­tients as well as com­bi­na­tion ther­a­pies go­ing af­ter more than one tar­get at once. And the FDA has in­di­cat­ed its will­ing­ness to pro­vide new guide­lines that fo­cus more on bio­mark­ers of the dis­ease to prove a drug’s abil­i­ty to dri­ve im­proved cog­ni­tion for pa­tients.

While a host of ma­jor play­ers like Pfiz­er and As­traZeneca and GSK have re­treat­ed from the field in the wake of mul­ti­ple fail­ures, leav­ing few or no projects in their wake, new play­ers like Cel­gene and the start­up De­nali have been get­ting in­to the Alzheimer’s are­na. You can now in­clude one more big play­er which plans to make a dif­fer­ence.

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

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”

CDC’s Robert Redfield, NIAID’s Anthony Fauci, Admiral Brett Giroir at HHS, and FDA’s Stephen Hahn prepare to testify at a House hearing on June 23 (Getty)

'Ex­treme­ly po­lit­i­cal' — Trump neuters FDA's at­tempt to strength­en vac­cine EUA

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.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust of the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Laura Shawver (Silverback Therapeutics)

Fol­low­ing a hefty Se­ries B, Sil­ver­back Ther­a­peu­tics quick­ly pulls in $85M for 'an im­por­tant growth phase'

Months after reeling in a $78 million Series B round, Silverback Therapeutics has hooked an even larger Series C.

The Seattle-based company announced Wednesday that it netted $85 million from a slate of new and previous investors. The quick boost could be a sign that an IPO is on the way.

In an email, Silverback CEO Laura Shawver told me she was “not able to provide any additional comments about Silverback” beyond what was shared in the company’s news release. In the prepared statement, she said the company is at “an important growth phase.”

Covid-19 roundup: Op­er­a­tion Warp Speed's 7th vac­cine is live at­ten­u­at­ed; Small biotech touts big suc­cess where gi­ants have failed

Operation Warp Speed is stacking its vaccine portfolio with a “TBD” new candidate: a live attenuated vaccine that can be administered in a single dose, potentially as an oral formulation rather than an injection.

Sound familiar?

That could be because the unannounced candidate appears to match the profile of an inoculation being developed by Merck, according to Bloomberg, which first reported the development based on a presentation by Moncef Slaoui.

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On­ly five months af­ter a Se­ries A launch, Taysha goes pub­lic with $157M IPO

As has been the trend in 2020, Taysha Gene Therapies has become the latest biotech to make a quick ascent from a small, privately-funded company to enjoying its very own Nasdaq ticker.

The Dallas-based biotech raised $157 million for its IPO after pricing shares at $20 apiece Thursday, the high-point of its expected range. Initially pegging $100 million in financing, Taysha offered a little less than 8 million shares and will trade under the $TSHA symbol.

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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.