TG Ther­a­peu­tic­s' shares jump as um­bral­is­ib wins FDA's break­through sta­tus

About a month af­ter the pub­li­ca­tion of en­cour­ag­ing re­sults from an ear­ly-stage study eval­u­at­ing J&J’s Im­bru­vi­ca in com­bi­na­tion with its ex­per­i­men­tal drug um­bral­is­ib, TG Ther­a­peu­tics on Tues­day said the drug has been grant­ed the FDA’s break­through ther­a­py sta­tus for use in pa­tients with mar­gin­al zone lym­phoma (MZL), a type of can­cer with no specif­i­cal­ly ap­proved ther­a­pies.

The New York-based com­pa­ny’s stock $TGTX jumped about 12% in pre-mar­ket trad­ing.

The des­ig­na­tion was award­ed on the ba­sis of in­ter­im da­ta from a co­hort of MZL pa­tients, who have un­der­gone at least one pri­or ther­a­py, in an on­go­ing mid-stage study — UNI­TY-NHL — test­ing um­bral­is­ib, which is a PI3K delta in­hibitor. Re­sults from the co­hort are ex­pect­ed by the mid­dle of the year. MZL com­pris­es slow grow­ing B-cell non-Hodgkin lym­phomas that orig­i­nate in the mar­gin­al zone of lym­phoid tis­sue.

Once a hot prospect in can­cer R&D, the PI3K path­way has in­spired mul­ti­ple drug pro­grams but seen lim­it­ed de­vel­op­men­tal suc­cess due to study fail­ures, safe­ty con­cerns and dura­bil­i­ty is­sues. The first ever PI3K in­hibitor, Gilead’s Zy­delig, was ap­proved by the FDA in 2014, and car­ries a boxed warn­ing high­light­ing its po­ten­tial­ly dead­ly side ef­fects. Since then, the US agency has giv­en the nod to oth­er such drugs in­clud­ing Bay­er’s Aliqopa. A pletho­ra of PI3K in­hibitors are al­so in de­vel­op­ment.

TG’s um­bral­is­ib is al­so cur­rent­ly in late-stage de­vel­op­ment in com­bi­na­tion with the com­pa­ny’s oth­er ex­per­i­men­tal drug, ubli­tux­imab, for pa­tients with hema­to­log­ic ma­lig­nan­cies.

Why it Works: Man­u­fac­tur­ing a Vac­cine in a Mul­ti-Prod­uct Fa­cil­i­ty.

COVID-19 launched the pharmaceutical industry to the frontline in the battle against the fast-spreading global pandemic. The goal: distribute a safe, effective vaccine as quickly as possible. Major players in the vaccine market needed to partner with contract development and manufacturing organizations (CDMOs) to achieve the goal of mass vaccine quantities under expedited timelines. With CDMOs stepping up to play a critical role in the vaccine manufacturing process, multi-product CDMO facilities took the spotlight. Partnerships quickly formed as the race to save lives and fight a pandemic was on.

Merck CEO Rob Davis

Mer­ck emerges as lead bid­der in po­ten­tial Ac­celeron buy­out with deal pos­si­ble this week — re­port

With rumors swirling about a potential buyout of biotech Acceleron and its lead PAH drug sotatercept, market watchers have been keeping close tabs on industry movers and shakers due up for an expensive bolt-on. According to a new report, it appears Merck may be the one.

Merck is in “advanced talks” on a deal to acquire Cambridge, MA-based Acceleron in what previous reports pegged as a potential $11 billion buyout, the Wall Street Journal reported Monday. A deal could come as early as this week, according to the Journal.

Habib Dable, Acceleron CEO

Days of heat­ed ru­mors cul­mi­nate in a re­port that Ac­celeron is in ad­vanced buy­out talks

Days of frothy rumors about possible M&A discussions at Acceleron were capped late Friday with a Bloomberg report asserting that the biotech company is in advanced talks for an $11 billion buyout deal.

Bloomberg was unable to identify any bidders in the deal, but speculation has been running rampant that the surging value of Acceleron stock had to be the result of leaks around the auction of the company. As of early Monday morning, we’re still awaiting the final word.

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Alexander Lefterov/Endpoints News

The coro­n­avirus vac­cine that the world for­got could still help save it

Back at the beginning of the pandemic — back when we still called the virus “novel” and a single case in Washington state could make headlines — there emerged the story of the coronavirus vaccine that the world forgot.

It was an allegory for our pandemic ill-preparedness. At a time when the world had been caught so flat-footed, there were a pair of scientists who had seen the crisis coming, lab-coated Cassandras with an antidote if only the world had listened sooner.

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Ash Shehata, KPMG

As sup­ply chain wor­ries ease up, Big Phar­ma CEOs have a new top con­cern: re­cruit­ing and keep­ing em­ploy­ees

As the industry goes through a boom, Big Pharma’s CEOs seem to have realized one thing above the rest: It’s time to reward the people doing the leg work.

KPMG, a Big Four accounting network, has compiled a business confidence report for pharma CEOs for the past few years, and while 2020’s report had top executives soul-searching for answers and predictions amidst a chaotic time, it appears that some normalcy, coupled with the skyrocketing growth of the industry, has restored confidence levels at the top.

Safe­ty fears force Pfiz­er to change piv­otal DMD gene ther­a­py tri­al pro­to­col

As one of the biggest players in an increasingly packed gene therapy space, Pfizer has taken an early lead over specialists like Sarepta in taking a Duchenne muscular dystrophy (DMD) candidate into late-stage testing. But new safety fears have led Pfizer to scale back that trial, cutting out patients with certain genetic mutations.

Pfizer has amended its enrollment protocol for a Phase III test for gene therapy fordadistrogene movaparvovec in DMD after investigators flagged severe side effects tied to specific mutations, according to a letter the drugmaker sent to Parent Project Muscular Dystrophy, a patient advocacy group.

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Kevin Boyle, Ziopharm Oncology CEO

Zio­pharm lays off half its staff af­ter man­u­fac­tur­ing is­sues force de­lays

New Ziopharm CEO Kevin Boyle has only been at the biotech for less than a month, but he’s already engineering major changes.

Late Monday afternoon, Ziopharm announced a sweeping restructuring that involved eliminating more than half its workforce and 60 jobs in total. The layoffs were necessary to help the penny stock player refocus its attention on advancing its TCR program, which saw a setback earlier this year after “unforeseen delays” at a contract manufacturer, Ziopharm said.

Albert Bourla, Pfizer CEO (John Thys, Pool via AP Images)

Covid-19 roundup: Pfiz­er/BioN­Tech sub­mit vac­cine da­ta to FDA for younger chil­dren; Doc­tors kept pre­scrib­ing hy­drox­y­chloro­quine

Pfizer and BioNTech said Tuesday they submitted to FDA positive data from a Phase II/III trial of their Covid-19 vaccine in children aged 5 to less than 12 years old.

A formal EUA submission for the vaccine in these children is expected to follow “in the coming weeks,” the companies said in a statement.

The trial of 2,268 healthy participants aged 5 to less than 12 years old showed the vaccine was safe and elicited robust neutralizing antibody responses using a two-dose regimen of 10 μg doses, which is one-third the dose that’s administered to adults.

From left to right: Mark Springel, Kristina Wang, Lin Ao, Soufiane Aboulhouda

George Church, his stu­dents, and top VCs go na­tion­wide with a biotech train­ing camp

One night last fall, Floris Engelhardt sat down in her Boston apartment and logged onto a Zoom call, armed with a comic and a vague idea about starting a biotech.

Engelhardt was joining a student-run “match night.” A postdoc at MIT’s Bathe BioNanoLab, where researchers use DNA and RNA like Lego blocks for nanometer-sized structures, Engelhardt wanted to find real-world applications for her work. She sketched out — literally — a plan to use DNA origami, a decade-old technique for precisely folding DNA, to make therapies.

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