FDA will con­vene ad­comm for Bris­tol-My­er­s' Re­blozyl in MDS; FDA grants 'or­phan' tags to a trio of drugs

Cel­gene and Ac­celeron‘s Re­blozyl (lus­pa­ter­cept) earned an OK from the FDA last month to treat ane­mia in adults who re­quire reg­u­lar trans­fu­sions, but still had a pend­ing re­view with reg­u­la­tors about us­ing the drug for myelodys­plas­tic syn­dromes (MDS). Now, Bris­tol-My­ers — which fi­nal­ly com­plet­ed its $74 bil­lion takeover of Cel­gene — and Ac­celeron said the FDA’s On­co­log­ic Drugs Ad­vi­so­ry Com­mit­tee will meet lat­er this month to dis­cuss their sup­ple­men­tal bi­o­log­ics li­cense ap­pli­ca­tion to to cov­er the treat­ment of adult pa­tients with very low- to in­ter­me­di­ate-risk MDS-as­so­ci­at­ed ane­mia who have ring sider­ob­lasts and re­quire red blood cell (RBC) trans­fu­sions. The PDU­FA date is set for April 4, 2020.

David Meek­er’s KSQ Ther­a­peu­tics — which earned a hefty $80 mil­lion from a fi­nanc­ing round last year — has en­list­ed cell en­gi­neer­ing ser­vice provider Max­Cyte to help de­vel­op adop­tive cell ther­a­pies for PD-1 re­frac­to­ry sol­id tu­mors. Un­der the terms of agree­ment, KSQ will be us­ing Max­Cyte’s tech to ad­vance their en­gi­neered tu­mor-in­fil­trat­ing lym­pho­cyte (eTIL) pro­grams.

→ The FDA has grant­ed Swiss-based APR Ap­plied Phar­ma Re­search (APR) or­phan drug des­ig­na­tion for its in­ves­ti­ga­tion­al drug, APR-TD011 — a hy­po­ton­ic acid-ox­i­diz­ing so­lu­tion con­tain­ing hypochlor­ous acid in a sprayable form — for the treat­ment of epi­der­mol­y­sis bul­losa (EB).

Tetra Bio-Phar­ma, a cannabi­noid-de­rived drug dis­cov­ery and de­vel­op­ment com­pa­ny, has re­ceived or­phan drug des­ig­na­tion from the FDA for delta-9-tetrahy­dro­cannabi­nol (THC) in the treat­ment of he­pa­to­cel­lu­lar car­ci­no­ma.

Adas­tra Phar­ma­ceu­ti­calszotiraci­clib has been des­ig­nat­ed by the FDA as an or­phan drug for glioma. The drug is cur­rent­ly un­der­go­ing two sep­a­rate Phase Ib clin­i­cal tri­als in glioblas­toma and the com­pa­ny ex­pects re­sults for both in 2020.

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.

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.

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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Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Xen­cor, UCLA to part­ner for new ther­a­peu­tic an­ti­bod­ies; Schrödinger ex­pands part­ner­ship with Google Cloud

Seeking to build on the 20 clinical-stage drug candidates already in its pipeline, California oncology and autoimmune disease-focused biotech Xencor announced on Thursday a partnership with nearby UCLA and the university’s Technology Development Group.

The two will collaborate to develop novel therapeutic antibodies, pairing novel targets proposed by scientists at UCLA with Xencor’s XmAb technology platforms that engineer monoclonal antibodies to create new protein structures. No financial terms of the agreement were disclosed, but any selected drug candidates resulting from the partnership will use a framework of “predefined terms to enter sponsored research agreements and potential license agreements,” the organizations said.

S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

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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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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Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.