Mi­cro­cap MEI joins the long line­up of can­cer bio­phar­mas to land a ‘break­through’

Fi­nan­cial prowess is no pre­req­ui­site to a break­through drug des­ig­na­tion at the FDA these days. Four years af­ter San Diego-based MEI Phar­ma land­ed an HDAC in­hibitor for a batch of stock and promised mile­stones, the mi­cro­cap biotech qual­i­fied for the in­side track at the FDA.

That was good for a 24% spike in its stock price $MEIP in pre­mar­ket trad­ing this morn­ing, as its mar­ket cap sat at a mere $46 mil­lion.

Just last Feb­ru­ary, the stock was flirt­ing with pen­ny stock ter­ri­to­ry. It’s moved out of that range in the months since.

The BTD is for pra­ci­no­s­tat, an HDAC in­hibitor used in com­bi­na­tion with azac­i­ti­dine for pa­tients with new­ly di­ag­nosed cas­es of acute myeloid leukemia (AML). It’s in­tend­ed for the el­der­ly or pa­tients who are un­fit for in­ten­sive chemother­a­py. And MEI says they al­so nailed down an agree­ment with reg­u­la­tors on their Phase III de­sign.

MEI is­sued 500,000 shares of stock to do the deal for pra­ci­no­s­tat four years ago, bag­ging the drug from S*BIO in Sin­ga­pore.

The com­pa­ny has a num­ber of ri­vals in the HDAC are­na. Syn­dax, now run by ex-As­traZeneca R&D chief Brig­gs Mor­ri­son, has a lead HDAC drug. As­traZeneca, mean­while, is col­lab­o­rat­ing with Mi­rati on an­oth­er HDAC, while HUYA Bio­science land­ed rights to an HDAC last March.

MEI tout­ed mid-stage da­ta of a me­di­an over­all sur­vival rate of 19.1 months and a com­plete re­sponse rate of 42% (21 of 50 pa­tients) in a re­cent study. In­ves­ti­ga­tors say the sin­gle arm study com­pared well to a Phase III study of azac­i­ti­dine, which showed a me­di­an over­all sur­vival of 10.4 months with azac­i­ti­dine alone and a CR rate of 19.5% in a sim­i­lar pa­tient pop­u­la­tion.

MEI CEO Daniel Gold

The FDA has hand­ed out a long line­up of BTDs since be­gin­ning the pro­gram. It’s in­tend­ed to pro­vide an in­side track at the agency for com­pa­nies de­vel­op­ing po­ten­tial­ly land­mark ther­a­pies, mak­ing it eas­i­er for com­pa­nies to get an ac­cel­er­at­ed ap­proval. The on­col­o­gy group has been par­tic­u­lar­ly will­ing to is­sue break­through des­ig­na­tions, which have mul­ti­plied like rab­bits.

“With this des­ig­na­tion, the FDA rec­og­nizes that our pre­lim­i­nary clin­i­cal da­ta demon­strate that Pra­ci­no­s­tat may re­sult in a sub­stan­tial im­prove­ment in the lives of AML pa­tients over avail­able ther­a­py,” not­ed MEI CEO Daniel Gold. “We have worked close­ly with the FDA to get to this point and now fo­cus on ex­e­cut­ing our Phase III study and bring­ing Pra­ci­no­s­tat to mar­ket as quick­ly and ef­fi­cient­ly as pos­si­ble.”


How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

Soon after becoming the darling of the VC crew and clearing the bar on a $4 billion valuation, Ginkgo — a synthetic biotech player out to reprogram cells with industrial efficiency — has now struck a deal to go public in the latest leviathan SPAC that sets its pre-money valuation at $15 billion. In one swift vault, Ginkgo will combine with Harry Sloan’s Soaring Eagle Acquisition Corp. and leap into the public markets.

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FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

A sign that the FDA’s non-Covid-related processes are beginning to normalize: The release of six guidelines from the International Council of Harmonisation.

Years in development, the ICH documents offer an international perspective on drug development, with these latest guidelines covering everything from recommendations to support the classification of drug substances, featured in the M9 guidance, to standards for nonclinical safety studies for pediatric medicines in the S11 guideline.

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Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

A recent court filing raises new questions about how major pharma companies like Sanofi, GlaxoSmithKline, and Boehringer Ingelheim have dealt with a lawsuit related to recalls of certain over-the-counter heartburn drugs due to the presence of a potentially cancer-causing substance found in them.

More than 70,000 people who took Sanofi’s Zantac and other heartburn drugs containing ranitidine, which have been recalled over the past two years, have sued the manufacturers, including generic drugmakers, and other retailers and distributors as part of a consolidated suit before US District Court Judge Robin Rosenberg in Florida.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

UP­DAT­ED: Bio­gen push­es in a fresh stack of chips and starts prep­ping a glob­al R&D game plan af­ter watch­ing the cards turn on ear­ly throm­bolyt­ic da­ta

After patiently steering through a decade-long journey for its early-stage clinical work, a small Tokyo biotech has clinched a deal to out-license its lead thrombolytic agent to US heavyweight Biogen — which sees a potentially game-changing impact on the clot-busting field after taking a careful look at some upbeat Phase IIa data.

Three years after Biogen anted up $4 million to gain an option on the drug from TMS, the big US biotech is making a small bet to beef up its stroke portfolio. The BD team inked a deal to go ahead and grab rights to the drug for $18 million, with another $335 million in milestone cash on the table for a successful outcome.

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Chris Garabedian (Xontogeny)

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

When Perceptive Advisors and startup accelerator Xontogeny initially teamed up on an early-stage VC round in 2019, the partners hoped to prove their investments could be a force multiplier for early-stage companies. Now, with that proof of concept behind them, the pair have closed a second VC round worth more than double the money.

Dubbed PXV Fund II and headed by Xontogeny CEO and former Sarepta head Chris Garabedian, the $515 million fund will target 10 to 12 early-stage preclinical companies with Series A rounds in the $20 million to $40 million range with opportunities for Series B follow-ups. The oversubscribed fund is bringing the band back with initial investors from PXVI as well as new investors that include “top-tier” asset managers, endowments, foundations, family offices, and individual investors.

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A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

As the pandemic constrained the FDA’s ability to travel for onsite manufacturing inspections, the agency increasingly turned to requesting records to fill the gap, even for hundreds of US-based facilities.

FDA explains in its guidance on manufacturing inspections during the pandemic that the agency can request records (not to be confused with the FDA’s remote interactive evaluations) directly from facilities “in advance of or in lieu of” certain onsite inspections. Companies are legally required to fulfill those requests because a denial may be considered limiting an inspection, which could lead to the FDA deeming a drug made at that site to be adulterated.

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Stephen Squinto, Gennao Bio CEO (Gennao)

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er

With his name already behind a rare disease success story in Alexion, Stephen Squinto was looking for a great story to drive him to jump back into the biotech game. He found that in a fledging non-viral gene therapy company, and now he’s got a few backers on board as well.

On Tuesday, Gennao Bio launched with a $40 million Series A co-led by OrbiMed and Logos Capital with participation by Surveyor Capital. The biotech, which is looking to use its cell-penetrating antibody platform to deliver nucleic acid “payloads” during into the nucleus, had to rush for its initial series — and had a name change along the way.

Alvotech takes Ab­b­Vie to court over al­leged patent 'mine­field' sur­round­ing megablock­buster Hu­mi­ra

AbbVie has so far been successful in shooing away competition to its megablockbuster Humira, deploying a number of patents and settlements to keep biosimilars off the US market until 2023. But one Icelandic drugmaker doesn’t want to wait — and on Tuesday, it filed a lawsuit challenging what it called a patent “minefield.”

Alvotech has accused AbbVie of trying to “overwhelm” and “intimidate” it with “an outrageous number of patents of dubious validity,” according to court documents. The company is currently seeking approval for its Humira copycat AVT02, which AbbVie says would infringe upon 62 patents.