On a roll, Ver­tex racks up im­pres­sive PhI­II da­ta for VX-659 com­bos in cys­tic fi­bro­sis

Ver­tex $VRTX has come up with a stack of pos­i­tive Phase III da­ta points for their cys­tic fi­bro­sis triple in­clud­ing the ex­per­i­men­tal VX-659. And its R&D group says they can ride these re­sults all the way to an FDA ap­proval.

Right now we’re just get­ting the pri­ma­ry end­point read­out, which is im­pres­sive. VX-659 is be­ing cred­it­ed with a 14% place­bo-ad­just­ed im­prove­ment from base­line in FEV1 for pa­tients with one F508del mu­ta­tion and one min­i­mal func­tion mu­ta­tion, with a clear score on the p val­ue (p<0.0001). 

In a sep­a­rate Phase III pa­tients ho­mozy­gous for the F508del mu­ta­tion al­ready tak­ing Symdecko saw an av­er­age 10% im­prove­ment with VX-659 added on.

Resh­ma Ke­wal­ra­mani

Now Ver­tex CMO Resh­ma Ke­wal­ra­mani says they will wait for the Phase III num­bers on their oth­er big add-on, VX-445, in Q1 be­fore de­cid­ing on the reg­u­la­to­ry strat­e­gy as they look to ex­pand their hold on CF.

Of par­tic­u­lar note: These new da­ta points are right in line with Phase II re­sults, of­fer­ing some clear par­al­lel sup­port for their com­bi­na­tion to take to reg­u­la­tors. 

The da­ta “live up to the hype,” not­ed Stifel’s Paul Mat­teis, high­light­ing the close com­par­i­son be­tween the last two tri­als.

While de­tails (as ex­pect­ed) are lim­it­ed be­yond the pri­ma­ry end­point, from press re­lease there’s noth­ing to nit­pick (in our view) for what has been reaf­firmed as a trans­for­ma­tion­al drug reg­i­men in “het-min” CF pa­tients, a pop­u­la­tion that rep­re­sents a mul­ti-bil­lion dol­lar op­por­tu­ni­ty with no tar­get­ed ther­a­pies ap­proved to­day.

Michael Yee at Jef­feries al­so joined the cheer­ing sec­tion.

In ho­mozy­gous which is a mar­ket al­ready well pen­e­trat­ed by Orkam­bi and Symdeko, this triple of ‘659 is meant to sig­nif­i­cant­ly im­prove FEV ef­fi­ca­cy from a cur­rent +4% to +14%, which is a huge im­prove­ment and ben­e­fit for pa­tients and will even­tu­al­ly re­place Orkam­bi/Symdeko over time and cre­ates a huge bar­ri­er to en­try giv­en the over­all ef­fi­ca­cy/safe­ty pro­file of ‘659 in this pop­u­la­tion.

Those views helped Ver­tex shares gain about 4% in ear­ly trad­ing Tues­day.

Ver­tex has been sur­round­ed by ri­vals look­ing to carve off a piece of the block­buster mar­ket that the Boston biotech has been carv­ing out for it­self. In sev­er­al cas­es, we’ve seen com­peti­tors stum­ble with mixed or poor da­ta. Pro­teosta­sis has man­aged to es­cape that fate, with some pos­i­tive ear­ly-stage num­bers of its own. But Ver­tex has been dom­i­nant in CF for years now, and ap­pears de­ter­mined to stay well in the lead.

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)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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Andrew Allen, Gritstone CEO (Gritstone via website)

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.

On Tuesday, the biotech revealed plans to start clinical testing of an experimental Covid-19 vaccine — in tandem with NIAID — that can also target other coronaviruses, with the goal of preventing future pandemics should SARS-CoV-2 prove difficult to cure with current vaccines. Then, on Wednesday morning, Gritstone licensed lipid nanoparticle technology from Genevant Sciences to develop what it’s calling “self-amplifying RNA vaccines” against Covid-19.

Bris­tol My­ers Squibb gets re­view date for Op­di­vo com­bo in gas­tric can­cer, look­ing to over­turn Keytru­da's 3-year lead

The past two months have been tough for Bristol Myers Squibb and its checkpoint inhibitor Opdivo after setbacks in lung and brain cancers. But in the battle against Merck’s Keytruda, any success matters — and now Bristol could be looking at a quick approval for Opdivo in an unmatched indication.

The FDA will launch a speedy review of a combination of Bristol Myers Squibb’s Opdivo and chemotherapy to treat first-line patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer or esophageal adenocarcinoma, the drugmaker said Wednesday. The agency set an action date of May 25 for the application.

Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

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.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Covid-19 claims an­oth­er PDU­FA vic­tim as Glax­o­SmithK­line push­es back planned PD-1 roll­out

Bristol Myers Squibb isn’t the only pharma giant that’s been standing in the FDA’s waiting line for site inspections.

GlaxoSmithKline is telling us today that their H2 2020 PDUFA deadline for the PD-1 drug dostarlimab — picked up in its Tesaro buyout — was pushed back due to a delay in the manufacturing site inspection needed for a regulatory decision. And that is forcing the company to revise its timeline.

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Artist rendering of the Assembly Square site in Somerville, MA (BioMed Realty)

Bio­Med Re­al­ty snaps up in­no­va­tion cam­pus site with­in earshot of pricey and bustling Boston biotech hub

On the short list of the premier biotech hubs in the world, the Boston area has transformed into a home for innovation — and ridiculously high rent. Now, a real estate firm is seeking tenants for a major site in neighboring Somerville with more than enough elbow room.

Snapped up by BioMed Realty, the land — which consists of an existing 162,000 square-foot office building and a 7.5 acre site — will serve as an “innovation space” for a variety of research, technology and life science tenants, the real estate company said in a press release. Financial terms weren’t disclosed.

Ugur Sahin, BioNTech CEO (AP Images)

Covid-19 roundup: BioN­Tech of­fers da­ta show­ing Pfiz­er-part­nered vac­cine pro­tects against vari­ant; No­vavax at­trib­ut­es re­spon­si­bil­i­ty for PhI­II de­lay to OWS

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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