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.

Fangliang Zhang, AP Images

UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Por­tion of Neil Wood­ford’s re­main­ing in­vest­ments, in­clud­ing Nanopore, sold off for $284 mil­lion

It’s been precisely one year and one day since Neil Woodford froze his once-vaunted fund, and while a global pandemic has recently shielded him from the torrent of headlines, the fallout continues.

Today, the California-based patent licensing firm Acacia Research acquired the fund’s shares for 19 healthcare and biotech companies for $284 million.  Those companies include shares for public and private companies and count some of Woodford’s most prominent bio-bets, such as Theravance Biopharma, Oxford Nanopore and Mereo Biopharma, according to Sky News, which first reported the sale. It won’t include shares for BenevelontAI, the machine learning biotech once valued at $2 billion.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Mer­ck wins a third FDA nod for an­tibi­ot­ic; Mereo tack­les TIG­IT with $70M raise in hand

Merck — one of the last big pharma bastions in the beleaguered field of antibiotic drug development — on Friday said the FDA had signed off on using its combination drug, Recarbrio, with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. The drug could come handy for use in hospitalized patients who are afflicted with Covid-19, who carry a higher risk of contracting secondary bacterial infections. Once SARS-CoV-2, the virus behind Covid-19, infects the airways, it engages the immune system, giving other pathogens free rein to pillage and plunder as they please — the issue is particularly pertinent in patients on ventilators, which in any case are breeding grounds for infectious bacteria.