Al­ler­gan/Ed­i­tas fi­nal­ly kick off CRISPR tri­al — mark­ing first in vi­vo test of the gene-edit­ing tech­nol­o­gy

More three years af­ter Al­ler­gan $AGN dipped its toes in­to the world of CRISPR/Cas9 gene edit­ing by en­list­ing Ed­i­tas $ED­IT as a part­ner — the com­pa­nies are now off to the races with a tri­al in pa­tients with a rare eye dis­ease.

This tri­al marks the first in­stance of us­ing the tech­nol­o­gy to ed­it DNA with­in the body.

Mean­while, part­ners Ver­tex $VRTX and CRISPR Ther­a­peu­tics $CR­SP kick­start­ed a tri­al ear­li­er this year that em­ployed the tech­nol­o­gy to en­gi­neer a gene-edit­ed stem cell ther­a­py to treat pa­tients suf­fer­ing from se­vere he­mo­glo­binopathies, a group of blood dis­or­ders. Sang­amo Ther­a­peu­tics $SG­MO has test­ed its old­er zinc fin­ger tech­nol­o­gy in pa­tients with two lyso­so­mal stor­age dis­eases, but a set­back dif­fused en­thu­si­asm for the pro­gram — but the com­pa­ny bounced back with en­cour­ag­ing ear­ly da­ta on its he­mo­phil­ia pro­gram part­nered with Pfiz­er.

Al­ler­gan and Ed­i­tas had orig­i­nal­ly ex­pect­ed to file an ap­pli­ca­tion to test their ther­a­py in hu­mans by the end of 2017, but man­u­fac­tur­ing trou­ble pushed that time­line to Oc­to­ber 2018. To get the FDA en­dorse­ment, Ed­i­tas fought off a stiff patent chal­lenge — and like its peers — bat­ted back per­sis­tent ques­tions about off-tar­get ef­fects to ad­e­quate­ly con­vince the reg­u­la­tor it was armed and ready to make the jump from an­i­mal stud­ies to hu­mans.

The tri­al, chris­tened Bril­liance, will eval­u­ate their ex­per­i­men­tal ther­a­py AGN-151587 (ED­IT-101) for the treat­ment of Leber con­gen­i­tal amau­ro­sis 10 (LCA10), a rare, in­her­it­ed form of blind­ness caused by mu­ta­tions in the CEP290 gene.

LCA is a group of in­her­it­ed reti­nal de­gen­er­a­tive dis­or­ders caused by mu­ta­tions in at least 18 dif­fer­ent genes, and are con­sid­ered the most com­mon cause of in­her­it­ed child­hood blind­ness, oc­cur­ring in two to three per 100,000 live births glob­al­ly. The most com­mon form of the dis­ease, LCA10, ac­counts for ap­prox­i­mate­ly 20% to 30% of all LCA pa­tients.

Ka­trine Bosley Ed­i­tas

Spark Ther­a­peu­tics’ ap­proved gene ther­a­py Lux­tur­na, which de­liv­ered via a vi­ral vec­tor, is tai­lored to treat an­oth­er rare eye dis­ease due to mu­ta­tions in both copies of the RPE65 gene. The FDA nod sparked Roche’s $4.3 bil­lion takeover of the com­pa­ny, al­though the deal is yet to be con­sum­mat­ed.

The Bril­liance study is set to en­roll 18 pa­tients — and in­clude up to five co­horts and eval­u­ate three dos­es of the drug. Each pa­tient will re­ceive a sin­gle dose of AGN-151587 ad­min­is­tered via an eye in­jec­tion aimed at pho­tore­cep­tor cells in the reti­na.

Ed­i­tas’ se­nior team has seen up­heaval in the past year. In Jan­u­ary, CEO Ka­trine Bosley, who led the com­pa­ny for near­ly five years, an­nounced her ex­it and sev­ered ties with the board, with­out ex­pla­na­tion. Chief fi­nan­cial of­fi­cer An­drew Hack an­nounced his de­ci­sion to leave by March, back in De­cem­ber 2018, and chief med­ical of­fi­cer Ger­ald Cox jumped ship at the end of last year.

Be­set by set­backs, Al­ler­gan’s Brent Saun­ders made the best of a bad sit­u­a­tion by agree­ing to be swal­lowed by Ab­b­Vie $AB­BV in an eye-wa­ter­ing $63 bil­lion deal ear­li­er this year. The US drug­mak­er was swayed by the po­ten­tial of Al­ler­gan’s block­buster Botox fran­chise to sta­bi­lize its rev­enue as its $20 bil­lion cash cow Hu­mi­ra hur­tles to­wards a patent cliff in the Unit­ed States.

So­cial im­age: Shut­ter­stock

Spe­cial re­port: Meet 20 ex­tra­or­di­nary women who are su­per­charg­ing bio­phar­ma R&D

Even though many biopharma leaders have come together in recent years to address its gender gap, the consensus is clear: We still have a long way to go.

Companies this year were 2.5 times more likely than last year to have a diversity and inclusion program in place, according to a recent BIO survey, but women are still largely absent from executive roles. Getting women to enter the industry isn’t the problem — studies show that they represent just under half of all biotech employees around the world. But climbing through the ranks can be challenging, as women still report facing stereotypes, and, unfortunately, harassment.

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Op­ti­miz­ing Oral Drug De­liv­ery us­ing Zy­dis® Oral­ly Dis­in­te­grat­ing Tablet Tech­nol­o­gy to Ad­dress Pa­tient Chal­lenges

KEY POINTS

Patients prefer oral dosing, but swallowing tablets can be a challenge for many patients.
The Zydis® orally disintegrating tablet (ODT) platform addresses challenges associated with oral dosing, expanding benefits for patients and options for healthcare providers.
A strong growth trajectory is expected for ODTs given therapeutic innovation and continued technology development.

Many patients prefer conventional tablets for the administration of medications, but some geriatric and pediatric patients and those with altered mental status and physical impairments find swallowing tablets to be difficult. Orally disintegrating tablets (ODTs), which dissolve completely without chewing or sucking, offer a patient-friendly dosage form for the administration of small-molecule drugs, peptides and proteins. With the potential for multiple sites of drug absorption, often faster onset action for the active pharmaceutical ingredient (API), and potentially greater bioavailability, ODTs are an attractive option for drug developers considering first-to-market formulations or product line extensions of existing drugs with compatible API. In this report, we look at how innovation in the industry-leading Zydis ODT platform is expanding oral formulation options and bringing benefits to patients.

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Geoffrey Porges (SVB Leerink)

The 2022 wave com­ing? Top an­a­lyst says Big Phar­ma will have more than $1T avail­able to sat­is­fy its grow­ing ap­petite for biotech M&A

All through this year you could practically feel the frustration of the biotech investor class as M&A activity continued to drag behind expectations — or desires. Buyouts of public companies provide the essential juice for keeping stocks lively, and there’s been a notable lack of juice in 2021.

So is all that about to change, big time?

SVB Leerink’s Geoffrey Porges, a longtime student of biotech M&A, thinks so. In a lengthy analysis he put out last week, Porges totted up the cash flow of the major pharmas and determined that there was a good long list of industry buyers who would have around a half trillion dollars of cash to play with in 2022. Leverage that up with added debt and you could get that deal cache to $1.6 trillion.

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Mer­ck pumps the brakes on two more PhI­II tri­als for its lead an­ti-HIV drug

After trial investigators flagged a drop in immune cell counts that an external committee determined was related to treatment last month, Merck has been pausing HIV-related Phase II and III trials ever since.

On Monday, the biopharma company announced it’s pausing enrollment in two of its Phase III trials evaluating its leading anti-HIV drug candidate, which is the once-monthly, oral islatravir.

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Bolt Bio CEO Randy Schatzman

Bolt Bio goes bust as in­vestors boo sin­gle re­sponse in ear­ly test against HER2-ex­press­ing tu­mors

Bolt Bio’s BDC-1001, an antibody conjugate drug designed to amp up the body’s innate immune system response to tumors, posted a single partial response in a Phase I/II study in patients with HER2-expressing solid tumors after a year of dosing. Just 13 of 40 evaluable patients showed any signs of “clinical activity,” the biotech said Monday.

BDC-1001 links a HER2-targeting biosimilar of Herceptin with a TLR7/8 agonist, which is designed to activate myeloid cells in the innate immune system and drive tumor cytotoxicity, Bolt said. But the early results paint the picture of a drug with little effect on HER2 tumors, one of the most highly validated tumor targets in drug development.

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Biohaven CEO Vlad Coric (Photo Credit_ Andrew Venditti)

Bio­haven shakes up lead­er­ship team as it feels the heat from mi­graine com­peti­tors

As Biohaven Pharma comes off a rollercoaster pipeline year, its CEO will take on more responsibility in a full C-suite makeover.

Vlad Coric was unanimously elected to the role of chairman of the board of directors, after Declan Doogan’s retirement. Matthew Buten will take over the role of CFO after James Engelhart’s retirement, and director Michael Heffernan has been appointed lead independent director. All of the appointments are effective immediately, a company press release said.

Chen Schor, Adicet CEO

Adicet un­veils ear­ly re­spons­es for off-the-shelf drug lever­ag­ing rare T cells. Will dura­bil­i­ty hold up?

On the hunt for the next generation of “off-the-shelf” cell therapies, biotech players like Adicet Bio have looked to leverage some of the less-obvious members of the immune system as potent cancer fighters. In Adicet’s case, scarce gamma delta T cells are on the menu, and an early cut of data is showing some promise.

Adicet’s AD-001, an off-the-shelf cell therapy developed by engineering a CD20-targeting chimeric antigen receptor (CAR) onto a donor’s gamma delta T cells, posted two complete responses across four patients in an early Phase I study testing the drug in patients with heavily pretreated B cell non-Hodgkin’s lymphoma, the biotech said Monday.

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Mar­ket­ingRx Matchup: How Ab­b­Vie and Bio­haven ads rank in head-to-head mi­graine chal­lenge

Are you ready to rumble? DTC brands that is. MarketingRx is launching a new monthly feature today called MarketingRx Matchup. We’re pitting two pharma brands’ DTC advertising in the same therapeutic category against each other to find out what consumers and patients really think.

Market research company Leger is handling the polling and analysis each month, and I’ll be writing up the results — along with my own take — inside MRx on the first Tuesday of the month.

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Wendy Lund, Organon chief communications officer

Q&A: Organon chief com­mu­ni­ca­tions of­fi­cer Wendy Lund talks about the Mer­ck spin­off, women’s health and why it mat­ters

One of Wendy Lund’s earliest jobs was head of marketing at Planned Parenthood. As the youngest person on its management team, she introduced them to emerging new technologies, and in return, she learned the importance of fighting for what you believe in.

Now as chief communications officer at Organon, the women’s health company recently spun off by Merck, Lund is keeping that point top of mind. That’s in part because women’s health hasn’t been a spotlight therapy area for Big Pharma in years. Several companies have spun off, sold or at least considered selling women’s health assets to focus on “core” products.

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