The Med­i­cines Com­pa­ny touts fresh ev­i­dence that their PC­SK9si can durably bat down LDL

John Maraganore, Al­ny­lam

The ex­ec­u­tive crew at The Med­i­cines Com­pa­ny say that they re­main on track with an ear­ly-stage PC­SK9 that re­li­ably knocked down lev­els of bad cho­les­terol, clear­ing one more hur­dle in a long jour­ney aimed at de­liv­er­ing a new ther­a­py that can beat out the pi­o­neers in the field with a drug that will on­ly need to be ad­min­is­tered two, three or four times a year.

There’s noth­ing new about PC­SK9 drugs, de­signed to slash LDL. Am­gen and the Re­gen­eron/Sanofi team de­liv­ered back-to-back ther­a­pies that do the same thing. But The Med­i­cines Com­pa­ny be­lieves it can roll up the mar­ket with a new and bet­ter ap­proach in-li­censed from Al­ny­lam that will be much eas­i­er to re­main com­pli­ant on.

Clive Mean­well, The Med­i­cines Com­pa­ny

The top-line in­ter­im analy­sis show­ing that PC­SK9si was con­tin­u­ing to look ef­fec­tive at the 90-day mar­ket — along with an ab­sence of safe­ty is­sues — stoked fresh hopes that the biotech com­pa­ny could be on­to some­thing. And the com­pa­ny is promis­ing an even more ad­vanced peak at the da­ta at the big AHA meet­ing next month.

“Mgmt cites the da­ta as “ro­bust” and that the LDL curves are ‘tight,'” telegraphed Ever­core ISI’s Umer Raf­fat Tues­day morn­ing.  “Com­pa­ny plans on head­ing in­to an ag­gres­sive Ph 3.”

That was en­cour­ag­ing enough for in­vestors to dri­ve shares of The Med­i­cines Com­pa­ny $MD­CO up 6% this morn­ing.

Last year, The Med­i­cines Com­pa­ny CEO Clive Mean­well and Al­ny­lam CEO John Maraganore out­lined plans for a years-long ef­fort aimed at dis­rupt­ing what is wide­ly ex­pect­ed to be­come a large mar­ket for these PC­SK9 cho­les­terol drugs. And Maraganore has told End­points that he be­lieves their drug is a sure-fire block­buster in the mak­ing.

Med­i­cines Com­pa­ny CEO Clive Mean­well had this to say to­day:

“These com­pelling in­ter­im da­ta af­firm PC­SK9si’s high­ly-com­pet­i­tive pro­file and val­i­date PC­SK9si’s game-chang­ing po­ten­tial. We look for­ward to pre­sent­ing re­sults from the ORI­ON-1 study, in­clud­ing Day 180 fol­low-up for up to 200 pa­tients, at the AHA meet­ing on No­vem­ber 15, 2016.”

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

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Hal Barron, GSK R&D chief (GSK via YouTube)

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CEO Stephen Yoder (Pieris)

Pieris fi­nal­ly vaults FDA hold on next-gen sol­id tu­mor hunter, clear­ing the path for mid-stage tri­al

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Neumann had joined Verastem after leaving bluebird bio, which surprisingly split into two companies last week, one in oncology and one in rare diseases. It’s not yet clear to where Neumann is headed next, but he noted in a statement that Verastem’s data and strategy were “truly exciting.”

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A month after filing the IND application for its human extracellular matrix designed to regenerate knee cartilage, Histogen has hit a roadblock.

The FDA on Tuesday verbally notified the San Diego-based biotech that it was placing a clinical hold on the planned Phase I/II clinical trial of HST-003 due to pending CMC information and additional questions needed to complete their review.

Histogen had planned to test the safety and efficacy of implanting hECM within microfracture interstices and related cartilage defects to regenerate that cartilage in conjunction with a microfracture procedure. The company said in a press release that it expects to receive written notice of the clinical hold from the FDA by Feb 12.

Janet Woodcock and Joshua Sharfstein (AP, Images)

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It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

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Grit­stone con­tin­ues Covid-19 push with deal to de­vel­op 'self-am­pli­fy­ing RNA' vac­cines, as shares con­tin­ue bal­loon­ing

Gritstone Oncology has had a big week, and it’s only Wednesday.

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Ugur Sahin, BioNTech CEO (AP Images)

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Ugur Sahin and his team at BioNTech have proffered more evidence that their Pfizer-partnered Covid-19 vaccine can protect people from a much-feared variant of SARS-CoV-2.

Colloquially known as the UK variant, the B.1.1.7 lineage triggered alarms because it appeared more transmissible. Among a series of mutations on its spike protein — the key antigen that all frontrunners in the vaccine race targeted — N501Y was of particular concern because it’s located on the receptor-binding site.

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