Reshma Kewalramani. Vertex

A deal-hun­gry Ver­tex ush­ers in three more gene edit­ing pro­grams as it clos­es out har­bin­ger CRISPR deal

Back in 2015, Ver­tex made a splash in the nascent CRISPR gene edit­ing world by wa­ger­ing $105 mil­lion to part­ner with one of its pi­o­neers — then-pri­vate start­up CRISPR Ther­a­peu­tics — to gen­er­ate up to six new treat­ments for ge­net­ic dis­eases.

Fast for­ward four years, the duo has ac­com­plished the his­toric feat of dos­ing their ex vi­vo ther­a­py, CTX001, in pa­tients with be­ta tha­lassemia, and Ver­tex has clear­ly sig­naled its in­tent on go­ing big­ger in­to gene edit­ing tech. As they wrap up the re­search part of their col­lab­o­ra­tion, the big biotech is tap­ping three more CRISPR pro­grams for its pipeline, which has bal­looned in re­cent weeks with new drugs in­tro­duced from new pacts.

Jeff Lei­den

In a brief up­date tucked in­to its Q3 re­port, Switzer­land-based CRISPR Ther­a­peu­tics not­ed that Ver­tex has ex­er­cised its op­tions to li­cense three ad­di­tion­al tar­gets, in­clud­ing the cys­tic fi­bro­sis trans­mem­brane con­duc­tance reg­u­la­tor (CFTR) gene and two oth­ers. That trig­gered a $30 mil­lion up­front pay­ment — $10 mil­lion each — and each project can earn as much as $410 mil­lion in mile­stones.

Mu­ta­tions in the CFTR gene is the cul­prit for cys­tic fi­bro­sis, a se­ri­ous dis­ease char­ac­ter­ized by the buildup of thick mu­cus in the lungs. Ver­tex’s port­fo­lio of ap­proved ther­a­pies, in­clud­ing the lat­est break­through triplet Trikaf­ta, work by tin­ker­ing with the re­sult­ing mu­tant pro­teins.

The CRISPR ap­proach, on the oth­er hand, aims to re­pair the de­fects (and there are hun­dreds of vari­a­tions) at their roots.

In all Ver­tex has ex­er­cised op­tions on five pro­grams, a spokesper­son con­firmed: In ad­di­tion to be­ta tha­lassemia, they are al­so test­ing CTX-001 in sick­le cell dis­ease. Pa­tient en­roll­ment is on­go­ing for both Phase I tri­als, and CRISPR re­mains in­volved in the de­vel­op­ment.

And they are not done yet. In June Ver­tex un­veiled a $245 mil­lion ac­qui­si­tion of Ex­on­ics, UT South­west­ern in­ves­ti­ga­tor Er­ic Ol­son’s gene-edit­ing start­up laser-fo­cused on Duchenne mus­cu­lar dy­s­tro­phy, and ex­pand­ed its al­liance with CRISPR Ther­a­peu­tics to cov­er DMD as well as my­oton­ic dy­s­tro­phy type 1. The ex­pan­sion cost $175 mil­lion up­front.

Those deals were the har­bin­ger of a shop­ping spree in which Ver­tex al­so picked up a stem-cell-based po­ten­tial cure for Type 1 di­a­betes and se­cured ac­cess to a small mol­e­cule plat­form for drug­ging RNA — all be­fore CEO Jeff Lei­den is set to hand the ba­ton to CMO Resh­ma Ke­wal­ra­mani, mov­ing to the chair­man post.

Now that Ver­tex has es­sen­tial­ly con­quered CF — both on the reg­u­la­to­ry and re­im­burse­ment fronts, hav­ing fi­nal­ly struck a deal with Eng­land’s health sys­tem — Ke­wal­ra­mani’s tenure will be de­fined by the new tech­nolo­gies and dis­ease ar­eas that she choos­es to pri­or­i­tize.

She will be do­ing so with a top team that has a bal­anced gen­der mix, fol­low­ing the pro­mo­tions of Car­men Boz­ic, for­mer­ly EVP of glob­al med­i­cines de­vel­op­ment and med­ical af­fairs, to CMO and Nia Tat­sis to head of reg­u­la­to­ry.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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The lat­est Cin­derel­la sto­ry in on­col­o­gy ends with a sud­den rout as up­dat­ed da­ta dis­play spooks in­vestors

NextCure’s turn as the Cinderella of cancer-focused biotechs was short-lived.
Just a few days after its shares $NXTC zoomed up more than 250% on some very early stage results in a SITC abstract, a more complete analysis over the weekend spiked the hype and left investors in high dudgeon as the stock price collapsed back towards earth Monday.
The focus at NextCure is centered on NC318, an antibody that is intended to shut down the immunosuppressive Siglec-15 — or S-15 — target. After adding a small group of patients to the readout, investigators circled 2 clinical responses, a complete and partial response, along with 4 stable disease cases in non-small cell lung cancer.

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Te­va spin­out rais­es $85M in IPO; No­var­tis beefs up gener­ics unit with $440M deal

→ After Teva spinout 89bio recently announced that its IPO was being held up, the company is back in the game offering 5,304,687 shares at a price of $16 per share. The company has raised $84.9 million IPO in gross proceeds and will be listed under the ticker symbol $ETNB. BofA Securities, SVB Leerink and RBC Capital Markets are the joint book-running managers for the offering. Oppenheimer & Co is the co-manager for the offering.
→ Looking to amp up its presence in Japan’s hospitals, Novartis has struck a deal to buy out Aspen’s portfolio of generics in the world’s third largest healthcare market. The pharma giant is paying $440 million for Aspen’s Japanese subsidiary.
→ Novartis said tropifexor, a non-bile acid FXR agonist, has scored on several key biomarkers of NASH in a Phase IIb trial, including reductions in hepatic fat, alanine aminotransferase and body weight compared to a placebo at 12 weeks.

Break­through sta­tus and promise of a speedy re­view ar­rives for Op­di­vo/Yer­voy com­bi­na­tion as Bris­tol-My­ers bites at Bay­er

Its frontline and single-agent aspirations have been set back, but Bristol-Myers Squibb just took a big step forward in its efforts to apply its checkpoint inhibitor Opdivo to liver cancer. The FDA has granted breakthrough status and priority review to a combination, second-line treatment.

The designation is for Opdivo (nivolumab) in combination with Yervoy (ipilimumab),  for treating advanced hepatocellular carcinoma (HCC), the most common form of liver cancer. The PD-L1 drug was already approved as a single-agent, second-line treatment for HCC. A PDUFA date was set for March 10, 2020 — just 4 months from now.

Third time un­lucky: Lipocine's lat­est quest to mar­ket their oral testos­terone drug snubbed again by FDA

Lipocine’s latest attempt at securing approval for its oral testosterone drug has fizzled yet again.

The Utah-based drug developer on Monday said the FDA has spurned its marketing application, indicating that some efficacy data on the drug, Tlando, was not up to scratch to treat male hypogonadism, a condition characterized by low production of the hormone testosterone, which is responsible for maintaining muscle bulk, bone growth, and sexual function.

UP­DAT­ED: De­cry­ing 'ar­bi­trary and capri­cious' ac­tion, Re­genxBio sues FDA over clin­i­cal holds on gene ther­a­py

When RegenxBio disclosed that the FDA had placed a partial clinical hold on one of its lead gene therapies, execs outlined several customary next steps: continuing assessment and monitoring, delaying a related IND filing, and working with the FDA to address the matter.

As it turned out, they were planning something much less mundane. Two days after announcing the hold in its Q3 update, RegenxBio filed a lawsuit seeking to set it aside, the FDA Law Blog noted.

Roche's SMA chal­lenge to Bio­gen's Spin­raza fran­chise looms larg­er with piv­otal win

Roche has just landed a crucial advance in scoring a come-from-behind win on the spinal muscular atrophy field, giving Novartis and Biogen a run for their money.

The update was brief, but Roche said risdiplam hit the primary endpoint in the placebo-controlled pivotal SUNFISH trial, meeting the threshold for change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment. The results, which is the second, confirmatory portion of a two-part study, involved 180 patients with type 2 or 3 spinal muscular atrophy between 2 and 25 years old.

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Roche steers Gazy­va in­to a new PhI­II pro­gram af­ter com­bo shows promise in lu­pus nephri­tis study

Roche is working on putting together a late-stage study for its monoclonal antibody Gazyva in patients with severe kidney disease associated with lupus after a combination approach helped patients in a mid-stage study.

The 125-patient NOBILITY trial evaluated Gazyva, combined with standard-of-care treatment mycophenolate mofetil or mycophenolic acid and corticosteroids, versus standard treatment alone. The combo met the main goal of inducing a statistically superior complete renal response (CRR) of 40% at week 76, versus 18% in patients given standard treatment, Roche said.