My Experience at the EHA 2023 Congress as a Patient

When you go to such a large congress, like the EHA 2023 celebrated in Frankfurt, the most pleasant thing is to meet all your friends, meet new people and listen to the presentations.

That’s what I did from Wednesday till Sunday. It is somehow tiring but exciting, and you come home with many excellent and exciting news.

Photos credits: Own elaboration (1, 2) and EHA/Marco Kovič (3-6)

With this short message, I would like to inform you about the most crucial development we can see in our MPN world, which shows that a lot is happening and if everything goes well, within a few years, we should have a relatively large number of new treatments and a few of them already in the market, at least in some countries.

This is not a complete list, but my highlights of what I find most interesting as a patient and MPN patient advocate. Let's start:

GSK/Momoletinib

This company acquired SIERRA ONCOLOGY some time ago. The product is expected to be approved soon for MF patients, especially those with anemia resistant to standard treatments. Early data indicate better outcomes than other JAK2 inhibitors. GSK is vigorously marketing the product and pursuing patient feedback, which is encouraging and significant. We are delighted to collaborate with them.

MORPHOSYS/Pelabresib

This company absorbed Constellation about two years ago. Pelabresib is a pan-BET inhibitor that decreases the inflammatory response in the MFP by modifying the microenvironment. MorphoSys is a very patient-oriented company that is transparent and active on social media. Working with them and bringing the patient's opinions to them is a pleasure.

MSD (Merck)/Bomedemstat

MSD completed the acquisition of Imago at the end of last year and gained access to the innovative Bomedemstat drug. The product is an LSD1 inhibitor that shows promising results in ongoing clinical trials for essential thrombocythemia. We have maintained a strong partnership with them for a long time and have enjoyed productive, transparent and positive collaboration.

Kartos Therapeutics/Navtematlin in combination with Ruxolitinib

Kartos Therapeutics is a promising pharmaceutical company that develops Navtematlin, an oral MDM2 inhibitor. Navtematlin has shown remarkable efficacy in treating myelofibrosis when combined with ruxolitinib, a JAK inhibitor. The company recently hired Prof. Serge Verstovsek, a renowned expert in myeloproliferative neoplasms, which boosted its reputation and value in the market.

Protagonist/Rusfertide (PTG-300)

Finally, a future product, hopefully, for PV. Rusfertide is a targeted therapy for the uncontrolled erythrocytosis in polycythemia vera because is a novel hepcidin mimetic that acts on the effects of hepcidin on erythropoiesis. This drug has demonstrated an enormous decrease in the frequency and number of phlebotomies that are a frequent cause of iron-deficiency symptoms in PV patient. The company behind Rusfertide is small but passionate, with a culture of openness and collaboration. I enjoy working with them very much.

CTI/Pacritinib

FDA-approved product. Swedish SOBI has just acquired CTI, one more merger in the pharmaceutical industry market. Right now, it's hard to say how things will play out. Great people are working at CTI, and let's hope the merger goes smoothly. Pacritinib is a cytoreduction product. The product is indicated in MF patients with low platelet count, where it has demonstrated efficacy and safety by not worsening the low platelet count that unfortunately occurs in some MF patients, a condition that limits the application of other JAK2 inhibitors.

BMS/Fedratinib (inrebic)

Due to the merger with Celgene, BMS is selling this approved product. I don't have much experience now; we should know more in about a year - also a cytoreductive product. Fedratinib is a JAK2V617F inhibitor especially recommended for those patients who become resistant to ruxolitinib. After a temporary withdrawal by the FDA, it is now fully accepted as a suitable JAK2 inhibitor.

Menarini/Kartyo Pharmaceutical

Menarini is a large and well-known pharmaceutical company in Italy. They have a marketing deal with Kartyo Pharmaceutical, which is developing a treatment for MF.

Once approved, Menarini will be a strong and professional partner for Kartyo Pharmaceuticals.

That's all my friends, I just wanted to share my thoughts on EHA Frankfurt 2023 with our community.

One thing that stood out to me was how the speakers addressed the patient's perspective and the impact of symptoms on quality of life. So far, minor points like Allylburden and Portalveine also appear in the talks.

I hope this post gives you some insight into the latest research and the potential benefits of new therapies for patients with MPN. I look forward to hearing your feedback and comments!

Peter Loffelhardt