Dai­ichi scores in PhI­II study of AML drug, beat­ing chemo and build­ing its case for world­wide ap­provals

An AML drug picked up by Dai­ichi Sankyo in its Am­bit buy­out has out­shone chemother­a­py in a late-stage tri­al, set­ting up the Japan­ese drug­mak­er to file for world­wide ap­provals.

An­toine Yver

The drug, called quizar­tinib, was test­ed in a Phase III study against re­lapsed or re­frac­to­ry acute myeloid leukemia (blood and bone mar­row can­cer) — par­tic­u­lar­ly in pa­tients who have the FLT3-ITD mu­ta­tion. This dan­ger­ous (but fair­ly com­mon) mu­ta­tion af­fects one in four AML pa­tients, giv­ing them a worse over­all prog­no­sis, in­clud­ing more re­laps­es and an in­creased risk of death fol­low­ing re­lapse. There are no ap­proved treat­ments.

The ther­a­py met its pri­ma­ry end­point of pro­long­ing over­all sur­vival com­pared to sal­vage chemother­a­py in pa­tients with the FLT3-ITD mu­ta­tion af­ter first-line treat­ment. Dai­ichi didn’t share any da­ta in its press re­lease, but said re­sults would be pre­sent­ed at an up­com­ing sci­en­tif­ic con­fer­ence.

“Sin­gle agent quizar­tinib is the first FLT3 in­hibitor to show a sig­nif­i­cant im­prove­ment in over­all sur­vival com­pared to cy­to­tox­ic chemother­a­py in a ran­dom­ized Phase III study of pa­tients with re­lapsed/re­frac­to­ry AML with FLT3-ITD mu­ta­tions, a very ag­gres­sive form of the dis­ease with lim­it­ed treat­ment op­tions,” said An­toine Yver, ex­ec­u­tive vice pres­i­dent and glob­al head of Dai­ichi Sankyo’s on­col­o­gy R&D, in a state­ment. ” We look for­ward to work­ing with reg­u­la­to­ry au­thor­i­ties world­wide to po­ten­tial­ly bring quizar­tinib to pa­tients as quick­ly as pos­si­ble.”

The com­pa­ny said it will use the da­ta to sup­port its oth­er on­go­ing Phase III tri­al of the drug. That tri­al is called QuAN­TUM-First, and it’s eval­u­at­ing quizar­tinib in com­bi­na­tion with in­duc­tion and con­sol­i­da­tion chemother­a­py as well as a main­te­nance ther­a­py.

Picked up in the $410 mil­lion buy­out of San Diego-based Am­bit Bio­sciences in 2014, quizar­tinib has been grant­ed fast track des­ig­na­tion by the FDA, as well as or­phan drug des­ig­na­tion by the FDA and EMA. The com­pa­ny said these re­cent Phase III re­sults will form the ba­sis of reg­u­la­to­ry sub­mis­sions for quizar­tinib world­wide.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Covid-19 roundup: Mer­ck­'s $356M sup­ply deal on hold as FDA asks for more da­ta; FDA ap­proves Pfiz­er/BioN­Tech vac­cine stor­age at stan­dard freez­er temps

Merck is pushing back plans to supply the US government with a Covid-19 drug after the FDA asked for more data to support an emergency use authorization.

The antibody, MK-7110, had looked promising in a Phase III study conducted by OncoImmune before Merck came along and bought the biotech for $425 million. At the interim analysis, investigators looked at data from 203 patients and concluded that a single dose of the drug cut the risk of death or respiratory failure by more than 50% among severe patients. And those taking the drug had a 60% higher chance of improvement in clinical status compared to placebo.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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CEO Fred Aslan (Artiva)

NK cell ther­a­py play­er Arti­va makes some more noise, pulling in $120M Se­ries B less than a month af­ter Mer­ck deal

Not even one month after Big Pharma took notice of Artiva when Merck signed a collaboration worth nearly $2 billion in milestones, the off-the-shelf NK cell biotech already has its next big fundraise.

Artiva returns from the venture well Friday with a $120 million Series B round, money they will use to get their first program into the clinic and to file INDs for another two candidates. The raise marks the latest development in a rapidly expanding footprint for Artiva, which, in addition to the Merck deal last month, has now raised almost $200 million since its Series A last June.

Fatty liver conceptual image, 3D illustration showing fatty liver silhouette made from micrograph of liver steatosis (Shutterstock)

The path to NASH: un­der­stand­ing the role of se­vere obe­si­ty in a com­plex, mul­ti-sys­tem dis­ease

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to senior editors Kyle Blankenship and Amber Tong.

We often think a person’s transition from a healthy to a diseased state is binary. But that’s often not the case. In reality, the onset of a disease is not something that occurs overnight, and the majority lie on a continuum that is impacted by a multitude of factors. Some of these factors are in a patient’s control. Others are not.

This is the case in nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH), two of the most complex diseases that “live” on this proverbial continuum. The clinical onset of NAFLD — and ultimately NASH — is a complex process that is closely related to obesity, insulin resistance and impaired adipose tissue metabolism.