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The tireless pace of change in hemato-oncology was showcased in Orlando.




The pace of progress in hematology has increased enormously in the last few years, making the annual American Society of Hematology (ASH) congress in early December one of the biggest stages for biopharma to unveil its most exciting advances.

Attending this gigantic event and keeping up with the most compelling data presentations is a taxing business; so much so that ‘powernap sleep pods’ were introduced this year in Orlando to allow attendees to recharge their batteries before returning to the fray.

The congress ran over four days (7-10 December) and has just concluded for another year. Here’s a rundown of just some of the highlights.

1. Bispecifics Take Center Stage
The biggest buzz in Orlando was around new results from a broad range of bispecific antibodies which are set to challenge CAR-Ts across blood cancer indications.

The first group are CD20-CD3 bispecific agents which are being developed for late-stage hard-to-treat lymphoma patients – including some already failed by CAR-Ts.  The stand-outs showcased at ASH were Roche’s mosunetuzumab and CD20-TCB, Regeneron’s REGN1979 and Xencor Inc.’s Xmab13676.

Meanwhile in multiple myeloma, there is a great swathe of bispecific candidates in the pipeline. Regeneron Pharmaceuticals Inc.’s REGN5458 and BMS’ CC-93269 both impressing with early stage data at the congress, with BMS’ candidate standing out thanks to overall response and complete remission rates to rival CAR-Ts in heavily treated and relapsed patients.

The promise of bispecifics has always been that they could achieve the same level of complete response seen in CAR-Ts, but without the severe side-effects (most notably cytokine release syndrome, CRS) seen in a large proportion of patients. However data presented at ASH showed bispecifics also bring with them risk of severe side-effects.

Analysts at Bernstein concluded that this meant there was “no free lunch” when it comes to treating patients with these potent new agents.  The Bernstein note concluded: “It is unclear which of the two modalities (CAR-T or BsAb) will win in the end – presumably the one that makes the biggest strides forward first; things do look better for CAR-T post ASH than prior to ASH.”

This final suggestion of potential superiority of CAR-Ts was based on studies presented on two stand-out next generation CAR-Ts: BMS’ liso-cel (JCAR017) and Johnson & Johnson’s JNJ4528, which both showed later onset of CRS side-effects than first generation CAR-Ts.

2. Janssen Steals Thunder In BCMA-Targeting CAR-T
The frontrunners to be first-to-market with a CAR-T therapy targeting BCMA in relapsed and refractory multiple myeloma are Bristol-Myers Squibb Co. and bluebird with bb2121.  The partners presented their latest KarMMA data on bb2121 just ahead of ASH, and will submit it to regulators in the first half of 2020. But while this data exceeded expectations, the readout from J&J and Legend Biotech’s  JNJ-4528 performed even better, producing an unheard of 100% response rate in all 29 patients on the CARTITUDE-1 Phase I/II trial.

3. More Progess In AML
Neglected for decades, acute myeloid leukemia (AML) has seen a rush of new treatments reach the market in the last few years, with lots more in the pipeline. Small biotech Forty Seven Inc. saw its share price double based on Phase I data for its magrolimab in myelodysplastic syndrome (MDS) and AML. This was based on an overall response rate of 64% and a complete response rate of 41% in AML, and even better figures in MDS. Meanwhile BMS provided data from a Phase III trial of oral maintenance therapy CC-486, improving median overall survival to 24.7 months compared to 14.8 months for placebo. BMS plans to file with regulators in the first half of 2020.

4. Calquence Shines In BTK inhibitor Battle
Competitors to AbbVie and J&J’s Imbruvica (ibrutinib) are crowding in on the blockbuster in B-cell cancers.  ASH saw lots of data from the Bruton’s tyrosine kinase (BTK) inhibitor class, and AstraZeneca has scored a major hit with Calquence (acalabrutinib) in first line CLL. There are plenty of other challengers, including ARQ-531 from ArQule, which was snapped up by Merck for $2.7bn during the congress. (Also see "AstraZeneca's Calquence Steps Up in First-Line CLL" - Scrip, 10 Dec, 2019.)

5. Sanofi Success In Niche Anemia Therapy
The Paris-headquartered company has scored a hit with a Phase III single arm trial of sutimlimab in cold agglutinin disease (CAD), a rare autoimmune hemolytic anemia. The first-in-class C1s inhibitor met the primary and secondary endpoints by rapidly improving anemia and fatigue symptoms. The CAD market is small, but analysts see greater opportunity in its use in immune thrombocytopenic purpura, where the therapy is now in Phase I. (Also see "Sanofi’s Sutimlimab Shows Promise In Rare Blood Disorder" - Scrip, 22 Nov, 2019.)

6. Darzalex Riding High
While it’s a cliché to say that competition is intense in hemato-oncology, one drug is making the most of having the field to itself. Janssen and Genmab AS’s Darzalex (daratumumab) remains the only CD38 targeting treatment for multiple myeloma on the market since it was launched in 2015, and is on course to hit revenues of $3.6bn next year.

New data presented in Orlando showed it powering ahead, providing evidence of greatly extended overall survival in frontline treatment of myeloma and in relapsed and refractory patients. A number of CD38-targeting competitors in the pipeline, including Sanofi’s isatuximab which is expected to gain approval in 2020, but Darzalex’s lead is unassailable. (Also see "Sanofi Poised To File Isatuximab In Multiple Myeloma, Going Up Against J&J's Darzalex" - Scrip, 5 Feb, 2019.)

7. Scientist’s Travel Ban Condemned
Last but by no means least is the hottest topic on the #ASH19 Twitter hashtag during the conference. The tweet features a sign taped to a stand where a poster presentation should have been. It states: “We regret that we are unable to present our data, since the current US travel bans prohibited the presenting scientist to the US and attend the conference.”

The lead researcher in question is Ehsan Bahrami, an Iranian postdoctoral researcher at the HelmholtzZentrum in Munich.

Many physicians responding with shock and disappointment at the news, reflecting widespread opposition to the travel ban which the Trump administration introduced for a small number of blacklisted nations since 2017.

One of those commenting was US hematologist Lori Rosenstein, who tweeted: “Sad for science and for these researchers. While it is called the “American” Society of Hematology, it truly is an international forum”.

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