#ASH17: Blue­print el­bows its way to cen­ter ring with ear­ly da­ta and a plan to seek quick FDA OK

Blue­print Med­i­cines $BPMC jumped on­to the radar at ASH on Sun­day, out­lin­ing some re­mark­able da­ta on their lead drug as the biotech lays the foun­da­tion for a reg­is­tra­tion pro­gram with the FDA. The Cam­bridge, MA-based biotech, which went pub­lic in 2015 and has a mar­ket cap that’s grown to $2.8 bil­lion, def­i­nite­ly wants to get the word out.

In­ves­ti­ga­tors for the com­pa­ny re­port­ed that their lead drug — avapri­tinib, for­mer­ly known as BLU-285 — hit an im­pres­sive 72% over­all re­sponse rate for rare cas­es of ad­vanced sys­temic mas­to­cy­to­sis and a 100% dis­ease con­trol rate.

Blue­print shares shot up 26% in pre-mar­ket trad­ing.

Re­searchers gath­er a group of 32 pa­tients with this dis­ease — char­ac­ter­ized by the pro­lif­er­a­tion of mast cells in tis­sue and or­gans. And af­ter treat­ing them with their drug, a KIT and PDGFRα in­hibitor, they record­ed a greater than 50% drop in serum tryptase in all pa­tients, which is a key bio­mark­er for the dis­ease.

Andy Bo­ral

The pa­tients with ra­di­ograph­ic scans demon­strat­ed a de­crease in spleen vol­ume. And the 25 pa­tients with bone mar­row mast cell bur­den? In that group, 21 pa­tients had at least a 50% de­crease and 15 pa­tients achieved a CR for bone mar­row mast cell bur­den.

They seem to be ea­ger to dis­cuss this ahead of the mar­ket open to­mor­row. Their con­fer­ence call is set for 6 am.

Said Blue­print Med­i­cines CMO Andy Bo­ral:

We look for­ward to en­gag­ing with glob­al reg­u­la­to­ry au­thor­i­ties in the first half of 2018 to ob­tain feed­back on a po­ten­tial reg­is­tra­tion path­way. In ad­di­tion, based on da­ta show­ing a fa­vor­able tol­er­a­bil­i­ty pro­file and strong clin­i­cal ac­tiv­i­ty from the low­est dose lev­els test­ed, we plan to ex­pand our clin­i­cal de­vel­op­ment pro­gram to ad­dress the full spec­trum of SM, in­clud­ing ad­vanced, smol­der­ing and in­do­lent forms of the dis­ease, over the course of 2018.

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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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.

UP­DAT­ED: Feds charge an­oth­er CRO staffer with fak­ing da­ta in a Glax­o­SmithK­line pe­di­atric asth­ma study

A Florida woman has been indicted as part of a clinical trial fraud scheme over a GlaxoSmithKline pediatric asthma study, the Justice Department announced Tuesday, the latest development in a case where three individuals have already pleaded guilty.

Jessica Palacio was charged with participating in a plot to falsify medical records, giving off the appearance that trial participants were making their scheduled visits to a Miami CRO and taking an experimental asthma medication as required. Palacio was also charged with lying to FDA investigators about her conduct.

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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