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Novel Treatments Give Hope for a Transformed Transplant Community

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March 15, 2023

Improvements in graft-vs-host disease and transplant-related mortality have created a wave of hope for clinicians treating their patients with hematologic malignancies, stemming from the encouraging data seen with Orca-T and other novel therapeutics in the pipeline.

Improvements in graft-vs-host disease (GVHD) and transplant-related mortality have created a wave of hope for clinicians treating their patients with hematologic malignancies, stemming from the encouraging data seen with Orca-T and other novel therapeutics in the pipeline.

For example, at the 2023 Transplantation and Cellular Therapy Meetings, data from a single-center phase 2 trial showed that Orca-T, which is a high precision cell therapy, produced a 100% overall survival rate in 8 patients with acute leukemia, myeloproliferative neoplasms, chronic myeloid leukemia, and other hematologic cancers who underwent an allogeneic hematopoietic stem cell transplant with 7/8 non-permissive haploidentical mismatched donors.1

The immune reconstitution of Orca-T in 100 patients from an ongoing, multicenter phase 1b trial (NCT04013685) was also examined in findings presented at the 2022 ASH Annual Meeting. Investigators performed longitudinal measurements of immune reconstitution, showcasing the peripheral blood counts of platelets, white blood cells, neutrophils, lymphocytes, monocytes, T cells, B cells, and natural killer (NK) cells. Results showed that patients on Orca-T exhibited early immune reconstitution in each of the key leukocyte and lymphocyte subsets believed to manage relapse and infection.2 Researchers also observed elevated Treg frequencies. Overall, these immune reconstitution data may be correlated to the reduced occurrence and severity of acute and chronic GVHD.

In an interview with OncLive®, Lori Muffly, MD, an associate professor of medicine at Stanford University, discussed the updated findings with Orca-T in hematologic malignancies, spoke to the product’s immune reconstitution profile, and the next steps she hopes to see taken with this type of treatment and others in the pipeline.

OncLive: Orca-T has been an agent to watch for over the last few years. Updated findings were presented at the 2022 ASH Annual Meeting, as well as some at the 2023 Transplantation and Cellular Therapy Meetings. Could you highlight the topline data reported at both meetings?

Muffly: We are really excited about Orca-T in the transplant community. The data have been presented a few times now from the single-center phase 2 and multicenter phase 1b studies . In the matched setting, which has been presented multiple times, what has been shown is low rates of both acute and chronic GVHD, and incredibly low rates of transplant-related mortality.

For someone like me, who treats patients with typically adverse risk of acute leukemia, I think the most exciting part is seeing that the relapse rates appear to be not increased in any way compared with our historical tacrolimus/methotrexate GVHD prophylaxis. That is very exciting because having overall survival rates that are phenomenal, low relapse rates, and then particularly low GVHD and transplant-related mortality rates, is exactly what we’re looking for in the field of transplant.

There were some immune reconstitution data with Orca-T that were presented at the 2022 ASH Annual Meeting. What were the key takeaways from that?

One of the other nice things about the Orca-T platform, which is really a reduced T-cell dose, along with an administration of regulatory T cells, is that the infection rates are quite low, relative to historical control. One of the problems with more of a traditional T-cell depleted graft, either ex vivo or in vivo, is higher rates of infection, particularly viral infections. We have not seen that with Orca-T.

At this past year’s ASH Annual Meeting, there was a poster demonstrating in about 90 patients, at least with day 28 data, and then around 50 patients with 1 year of follow-up, the immune cell subset reconstitution. What we could see is that these patients reconstituted B cells, T cells, regulatory T cells and NK cells by 1 year, quite effectively. That probably has something to do with why we’re seeing lower rates of infection than as predicted in a T-cell deplete graft.

Putting it into context for the practicing clinician, why is immune reconstitution important and how do we use it to guide treatment decisions for patients?

With transplant, there are so many different factors that we think about—of course, a focus on the disease itself that we’re transplanting for. For example, if a patient has acute myeloid leukemia, trying to get a transplant that has the most potential for graft vs leukemia is important. We think about GVHD, because that is the adverse flip side of graft vs leukemia and identifying mechanisms of transplant that are associated with lower GVHD is important.

Then finally, the ability of the new graft to protect patients from infection and particularly opportunistic infection and viral reactivations [are what we think about]. Any 1 of those 3 components that’s missing is a big problem for our patients. If you have a transplant approach that really can take care of all 3 [components], that is really the holy grail of transplant: low relapse, low GVHD, and low infection.

Taking these data into context, what next steps would you like to see taken with this type of therapy?

I am really excited about the possibilities with Orca-T. Currently, as you probably know, there is a randomized phase 3 study, a registration trial, ongoing comparing Orca-T with historical control tacrolimus and methotrexate [and alloHSCT]. We have been very actively participating in that study. Obviously, we are hopeful to see the results and that this may lead to commercialization of this product.

We’re also really excited about Orca-T in the mismatched myeloablative setting. There was an oral abstract at the [2023 Transplantation and Cellular Therapy Meetings] presented with just under 10 patients with mismatched, unrelated donors. Those data look exciting, as well. There is a lot of opportunity for this program in the clinical space.

Now, the other thing that’s happening in the transplant community, which is very difficult to miss, is the evolution and uptake of posttransplant cyclophosphamide as GVHD prevention. We are really blessed right now that we have 2 exciting approaches going on at the same time: posttransplant cyclophosphamide and Orca-T. The field eventually will want to look at the 2 of them side by side, because they both seem to lead to low GVHD rates without an increase in disease relapse. As someone who offers these treatments to my patients, I have never been more excited about what we have going on in this field. I look forward to future studies as well.

Is there anything else that you think would be important for the practicing oncologist to know about these or any other therapies in the pipeline?

It is important for the practicing oncologist to know that as our therapeutics for blood cancers are improving, so are our approaches to bone marrow transplant. I no longer think about transplant-related mortalities of 20% to 30% when I’m talking to patients, and that’s how things started, at least when I started. I’m now hopeful that that sort of topline number is going to be more like a 5% risk [as more options become available]. While that’s still too high, it’s at least a bit more acceptable.

Also, the likelihood of developing GVHD with these newer approaches has substantially gone down. That is important for referring physicians to know because the darker days, scarier days, of transplant are behind us. This is a therapeutic that has curative potential for many, many adults out there. Many patients just are not referred. These data are important to get out there so that people in the community can realize transplant data are looking different than they used to look.

References

  1. Pavlova A, Lowsky R, Muffly L, et al. Orca-T, a high-precision cell therapy, for the treatment of hematologic malignancies in patients with 7/8 mismatch donors. Presented at: 2023 Transplantation & Cellular Therapy Meetings of ASCT and CIBMTR; February 15-19, 2023; Orlando, FL. Abstract 30.
  2. Meyer EH, Killian MS, Pavlova A, et al. Rapid immune reconstitution and elevated regulatory T cell frequencies in patients treated with Orca-T. Blood. 2022;140(suppl 1):7656-7657. doi:10.1182/blood-2022-164468

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Earlier Jakafi Treatment Shows Encouraging Outcomes in Myelofibrosis

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Published on: March 15, 2023

 

Earlier treatment with Jakafi showed promise in both improving survival and symptom burden in certain patients with myelofibrosis, though physicians often delay Jakafi treatment.

A higher percentage of patients with intermediate-2 and high-risk myelofibrosis who were introduced to Jakafi (ruxolitinib) within a year of being diagnosed saw improved survival and reduced symptom burden compared to those who were given the drug later or not at all,according to recent research that was published in the journal Cancer.

Researchers analyzed data from two clinical trials (COMFORT-I and COMFORT-II), which included 525 patients with myelofibrosis, a type of myeloproliferative neoplasm.

In this patient population, 84 patients received Jakafi within 12 months of being diagnosed, while 216 received the drug at or after 12 months from diagnosis. Additionally, 66 patients received placebo plus best available therapy within 12 months, and 159 patients received placebo plus best available therapy after 12 months.

Myelofibrosis causes a decrease in the number of normal cells that are produced in the bone marrow. As a result, the spleen starts to produce these types of cells, and often becomes inflamed — indicating potentially worsening disease and putting patients at risk for infections.

“Patients with myelofibrosis, a bone marrow cancer, often do not live as long as the general population. These patients may also have an enlarged spleen and difficult symptoms such as fatigue,” the researchers wrote.

Study findings showed that at week 48, patients who received Jakafi earlier had a higher rate of spleen volume response (meaning that their spleen shrunk) than those who were not exposed to the drug within a year of treatment (44% versus 26.9%, respectively).

Additionally, at a 240-week follow-up, 63% of patients who were given Jakafi early were still alive, compared with 57% of those who were given the drug a year or later after being diagnosed. At this time, 49.4% of patients who received placebo plus best available therapy within 12 months were alive, and 40.7% of those given placebo/best therapy at or after 12 months were alive.

The researchers also noted that on average, patients given Jakafi — regardless of whether it was within or after 12 months of diagnosis — tended to live longer than those prescribed placebo plus best available therapy.

The Food and Drug Administration approved Jakafi for intermediate- or high-risk myelofibrosis more than a decade ago, and it is recommended to be the first therapy offered for patients with the disease. However, as the study authors pointed out, Jakafi is often not the first drug given, or it is not given soon after diagnosis.

“The National Comprehensive Cancer Network guidelines for myeloproliferative neoplasms recommend (Jakafi) as a first-line treatment for patients with higher-risk (myelofibrosis),” study authors wrote. “There is a compelling rationale to treat patients with intermediate- or high-risk (myelofibrosis) with (Jakafi). Despite this, real-world treatment patterns indicate that many physicians delay or avoid (Jakafi) treatment, often in favor of hydroxyurea or watchful waiting.”

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Incyte Announces Data from Across its Oncology Portfolio will be Presented at the AACR Annual Meeting 2023

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Tue, March 14, 2023 at 6:30 PM EDT

WILMINGTON, Del., March 14, 2023–(BUSINESS WIRE)–Incyte (Nasdaq:INCY) today announced that multiple abstracts from across its oncology portfolio will be presented during the upcoming American Association for Cancer Research (AACR) Annual Meeting 2023, held April 14-19, in Orlando, Florida.

“As Incyte continues to advance research in areas where we believe we can have the biggest impact for patients, we look forward to sharing new pre-clinical and clinical data from our expansive oncology portfolio at this year’s AACR,” said Steven Stein, M.D., Chief Medical Officer, Incyte. “Notably, a plenary session will feature data on pemigatinib in previously-treated solid tumors with activating FGFR1–3 alterations, and five-year results from the L-MIND study of tafasitamab (Monjuvi®) in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) will be highlighted in a late-breaking oral presentation.”

Key abstracts from Incyte-sponsored and partner programs include:

Oral Presentation in a Plenary Session

Pemigatinib

Clinical and Translational Findings of Pemigatinib in Previously Treated Solid Tumors with Activating FGFR1–3 Alterations in the FIGHT-207 Study (Abstract #CT016. Session: Novel Biomarker-Driven Molecularly Targeted Therapy Trials. Tuesday, April 18, 2023, 10:15 a.m. – 10:30 a.m. ET)

Oral Presentation

INCB123667 (CDK2)

Development of a CDK2-Selective Small Molecule Inhibitor INCB123667 for the Treatment of CCNE1hi Breast Cancers (Abstract #1143. Session: Small Molecule Therapeutic Agents. Sunday, April 16, 2023, 3:52 p.m. – 4:07 p.m. ET)

Tafasitamab

Five-Year Safety and Efficacy of Tafasitamab in Patients with Relapsed or Refractory DLBCL: Final Results from the Phase 2 L-MIND Study1 (Abstract #CT022. Session: Novel Clinical Trials for Hematological Malignancies. Sunday, April 16, 2023, 3:20 p.m. – 3:30 p.m. ET)

Poster Presentations

INCA33890 (PD-1×TGFbR2)

INCA33890, a Novel PD-1×TGFbR2 Bispecific Antibody Conditionally Antagonizes TGF Signaling in Primary Immune Cells Co-expressing PD-1 (Abstract #2936. Session: Therapeutic Antibodies 2. Monday, April 17, 2023, 1:30 p.m. – 5:00 p.m. ET)

INCB098377 (PI3Kδ)

Discovery of INCB098377: a Potent Inhibitor of Phosphoinositide 3-Kinase-Gamma (Abstract #5162. Session: Modifiers of the Tumor Microenvironment. Tuesday, April 18, 2023, 1:30 p.m. – 5:00 p.m. ET)

LIMBER (MPN)

A Phase 1 Study of Ruxolitinib in Combination with Abemaciclib for Patients with Primary or Post-Polycythemia Vera/Essential Thrombocythemia Myelofibrosis (Abstract #CT242. Session: Phase 1 and First-in-Human Clinical Trials in Progress. Tuesday, April 18, 2023, 1:30 p.m. – 5:00 p.m. ET)

Preclinical Characterization of the BET Inhibitor, INCB057643, in Combination with Ruxolitinib for Treatment of Myeloproliferative Neoplasms (MPN) (Abstract #6274. Session: Epigenetics. Wednesday, April 19, 2023, 9:00 a.m. – 12:30 p.m. ET)

Pemigatinib

Drug Combination Screen Identifies Pemigatinib, an FGFR Inhibitor, as a Mechanism to Overcome KRASG12C Inhibitor Resistance in Lung Cancer (Abstract #412. Session: Drug Resistance in Molecular Targeted Therapies 2. Sunday, April 16, 2023, 1:30 p.m. – 5:00 p.m. ET)

Pemigatinib, an FGFR Inhibitor, Overcomes Resistance to KRASG12C Inhibitors in Mesenchymal-Like NSCLC Tumors (Abstract #430. Session: Drug Resistance in Molecular Targeted Therapies 2. Sunday, April 16, 2023, 1:30 p.m. – 5:00 p.m. ET)

P-Selectin Glycoprotein Ligand-1

P-Selectin Glycoprotein Ligand-1 Modulates the Functions of Human T Cells and Macrophages in Vitro (Abstract #6373. Session: Immune Checkpoints. Wednesday, April 19, 2023, 9:00 a.m. – 12:30 p.m. ET)

Tafasitamab

Combination of BTK Inhibitor Orelabrutinib, Anti-CD19 Antibody Tafasitamab, and IMiD Lenalidomide for the Treatment of B Cell Malignancies2 (Abstract #4013. Session: Tyrosine Kinase and Phosphatase Inhibitors 1. Tuesday, April 18, 2023, 9:00 a.m. – 12:30 p.m. ET)

Preclinical Study of CD19 Detection Methods Using Monoclonal Antibodies Post Tafasitamab Treatment1 (Abstract #6329. Session: Anticancer Immunotherapeutics. Wednesday, April 19, 2023, 9:00 a.m. – 12:30 p.m. ET)

Zilurgisertib

Clinical Trial Simulation to Inform Dose Selection of Zilurgisertib, an ALK2 inhibitor, in Patients with Anemia Due to Myelofibrosis (MF) (Abstract #CT243. Session: Phase 1 and First-in-Human Clinical Trials in Progress. Tuesday, April 18, 2023, 1:30 p.m. – 5:00 p.m. ET)

For registered attendees, the virtual meeting platform and all on-demand sessions will be available through July 19, 2023. More information regarding the AACR Annual Meeting 2023 can be found at https://www.aacr.org/meeting/aacr-annual-meeting-2023/.

About Pemazyre® (pemigatinib)
Pemazyre is a kinase inhibitor indicated in the United States for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test3. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Pemazyre is also the first targeted treatment approved for use in the United States for treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement.

In Japan, Pemazyre is approved for the treatment of patients with unresectable biliary tract cancer (BTC) with a fibroblast growth factor receptor 2 (FGFR2) fusion gene, worsening after cancer chemotherapy.

In Europe, Pemazyre is approved for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

Pemazyre is a potent, selective, oral inhibitor of FGFR isoforms 1, 2 and 3 which, in preclinical studies, has demonstrated selective pharmacologic activity against cancer cells with FGFR alterations.

Pemazyre is marketed by Incyte in the United States, Europe and Japan.

Pemazyre is a trademark of Incyte Corporation.

About Tafasitamab (Monjuvi® / Minjuvi®)
Tafasitamab is a humanized Fc-modified CD19 targeting immunotherapy. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb® engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP).

In the United States, Monjuvi® (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In Europe, Minjuvi® (tafasitamab) received conditional marketing authorization in combination with lenalidomide, followed by Minjuvi® monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT).

Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in several ongoing combination trials.

Monjuvi® and Minjuvi® are registered trademarks of MorphoSys AG. Tafasitamab is co-marketed by Incyte and MorphoSys under the brand name MONJUVI® in the U.S., and marketed by Incyte under the brand name Minjuvi® in Europe and Canada.

XmAb® is a registered trademark of Xencor, Inc.

About Jakafi® (ruxolitinib)
Jakafi® (ruxolitinib) is a JAK1/JAK2 inhibitor approved by the U.S. FDA for treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea; intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF in adults; steroid-refractory acute GVHD in adult and pediatric patients 12 years and older; and chronic GVHD after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older3.

Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi® (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.

About Incyte
Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.

Forward-Looking Statements
Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the presentation of data from Incyte’s clinical development pipeline, whether or when any development compounds or combinations will be approved or commercially available for use in humans anywhere in the world outside of the already approved indications in specific regions and Incyte’s goal of improving the lives of patients, contain predictions, estimates and other forward-looking statements.

These forward-looking statements are based on Incyte’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; the effects of the COVID-19 pandemic and measures to address the pandemic on Incyte and its partners’ clinical trials, supply chain, other third-party providers and development and discovery operations; determinations made by the U.S. FDA and other regulatory authorities outside of the United States; the efficacy or safety of Incyte and its partners’ products; the acceptance of Incyte and its partners’ products in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in Incyte’s reports filed with the Securities and Exchange Commission, including its annual report for the year ending December 31, 2022. Incyte disclaims any intent or obligation to update these forward-looking statements.

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Dr. Rampal on the Role of JAK Inhibitors in Myelofibrosis

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Mar 14, 2023

Raajit K. Rampal, MD, PhD

Raajit K. Rampal, MD, PhD, hematologic oncologist, associate attending physician, Memorial Sloan Kettering Cancer Center, discusses how the role of JAK inhibitors will continue to evolve in the treatment of patients with myelofibrosis.

patients with myelofibrosis, Rampal begins. However, it is not believed that JAK inhibitors modify the disease, and their effects can be limited in subgroups of patients, Rampal says.

Multiple JAK inhibitors are now available for the treatment of myelofibrosis, and this has allowed select patients to potentially be cycled between different agents, Rampal explains. As more data have emerged, they have been able to demonstrate how various JAK inhibitors can effect patients differently, and this may put clinicians in a position to better select certain JAK inhibitors for select patients, Rampal emphasizes.

Within the next 2 to 3 years, it is possible that the treatment landscape for patients with myelofibrosis will drastically shift, Rampal says. Multiple agents are in late phase 3 trials, and if data continue to prove positive for some or all of these treatments, there will be an influx of new treatment options entering clinical practice, Rampal notes.

Although the landscape will change, it remains unclear exactly how that will happen. One possible scenario is that trials for the drugs in development are successful, and then clinicians will need to determine if these treatments will be added on to current regimens for use in combinations, or if they should follow the use of JAK inhibitors if a patient does not have a full response, Rampal notes. These remain unanswered questions, but they will be addressed in short time as other agents begin to enter the treatment landscape, Rampal concludes.

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Myelofibrosis Market to Witness Upsurge in Growth During the Forecast Period (2022-2032)

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Published March 13, 2023

The Myelofibrosis market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Myelofibrosis pipeline products will significantly revolutionize the Myelofibrosis market dynamics.

DelveInsight’s “Myelofibrosis Market Insights, Epidemiology, and Market Forecast-2032″ report offers an in-depth understanding of the Myelofibrosis, historical and forecasted epidemiology as well as the Myelofibrosis market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Myelofibrosis market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Myelofibrosis Overview

Myelofibrosis (MF) is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar like material. It is classified as a type of chronic leukemia and belongs to a group of blood disorders called myeloproliferative diseases.

Some of the key facts of the Myelofibrosis Market Report: 

  • The Myelofibrosis market size is anticipated to grow with a significant CAGR during the study period (2019-2032)
  • In June 2022, Sierra Oncology submitted an NDA to the US FDA for momelotinib to treat myelofibrosis. The FDA has accepted the NDA and has assigned a PDUFA date of 16 June 2023
  • Vonjo, approved by the US FDA on February 28, 2022, is a kinase inhibitor used for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L
  • In various studies of European countries, annual prevalence of Myelofibrosis ranged from 0.5–9 cases per 100,000 individuals
  • Myelofibrosis is a disease that tends to affect the middle-aged and elderly population with a mean age of 60 years at diagnosis. Males are more commonly affected than females. In younger children, girls are affected twice as often as boys
  • The total prevalent population of myelofibrosis in the 7MM comprised of 39,735 cases in2021and is projected to increase during the study period (2019–2032)
  • In 2021, the highest number of prevalent cases were observed in the US. The total prevalent population of myelofibrosis in the US is 19,815 in 2021 and is projected to increase during the forecast period (2022-2032)
  • Key Myelofibrosis Companies: Pharmaxis, Keros Therapeutics, Bristol-Myers Squibb, Ascentage Pharma Group Inc., Sumitomo Pharma Oncology, Galecto Biotech AB, Actuate Therapeutics Inc, Karyopharm Therapeutics Inc, AbbVie, Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Taiga Biotechnologies, Inc., Rigel Pharmaceuticals, Celgene, Novartis Pharmaceuticals, Sierra Oncology, Inc., Incyte Corporation, Imago BioSciences, Inc., Samus Therapeutics, Inc., Constellation Pharmaceuticals, Kartos Therapeutics, Inc., NS Pharma, Inc., Nippon Shinyaku Co., Ltd., Geron Corporation, Inc., Suzhou Zelgen Biopharmaceuticals Co.,Ltd, Hoffmann-La Roche, Lynk Pharmaceuticals Co., Ltd, Chengdu Zenitar Biomedical Technology Co., Ltd, Prelude Therapeutics, Cellenkos, Jacobio Pharmaceuticals, Active Biotech, The Menarini Group,  Cyclica Inc, GAT Therapeutics, Hinova pharmaceuticals, iOnctura,  Telios Pharma, Inc., and others
  • Key Myelofibrosis Therapies: Momelotinib, Navitoclax, Parsaclisib, Reblozy, Pelabresib, PXS-5505, and others

 Key benefits of the Myelofibrosis Market report:

  1. Myelofibrosis market report covers a descriptive overview and comprehensive insight of the Myelofibrosis Epidemiology and Myelofibrosis market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan.)
  2. The Myelofibrosis market report provides insights on the current and emerging therapies.
  3. Myelofibrosis market report provides a global historical and forecasted market covering drug outreach in 7MM.
  4. The Myelofibrosis market report offers an edge that will help develop business strategies by understanding trends shaping and driving the Myelofibrosis market.

Download the report to understand which factors are driving Myelofibrosis epidemiology trends @ Myelofibrosis Epidemiological Insights 

Myelofibrosis Market  

The dynamics of the Myelofibrosis market are anticipated to change in the coming years owing to the expected launch of emerging therapies and others during the forecasted period 2019-2032.

“According to the estimates, the highest market size of Myelofibrosis was found in theUnitedStates and the least was in Spain across the 7MM. Besides, the upcoming therapies of Myelofibrosis are expected to combat the current unmet needs faced by the patients with Myelofibrosis and add to the overall growth of the Myelofibrosis market size.”

 Myelofibrosis Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Myelofibrosis Epidemiology Segmentation:

The Myelofibrosis market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:

  • Total Prevalence of Myelofibrosis
  • Prevalent Cases of Myelofibrosis by severity
  • Gender-specific Prevalence of Myelofibrosis
  • Diagnosed Cases of Episodic and Chronic Myelofibrosis

Myelofibrosis Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Myelofibrosis market or expected to get launched during the study period. The analysis covers Myelofibrosis market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Myelofibrosis Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Myelofibrosis Therapies and Key Companies

  • Momelotinib: Sierra Oncology
  • Navitoclax: AbbVie
  • Parsaclisib: Incyte
  • Reblozy: Celgene/Bristol Myers Squibb
  • Pelabresib: MorphoSys
  • PXS-5505: Pharmaxis

Myelofibrosis Market Strengths

  • Myelofibrosis is a rare form of hematologic cancer; thus, companies developing its treatment options can possess several advantages like market exclusivities, premium pricing, trial subsidy, and other benefits from the government bodies for R&D.

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Dr. Mascarenhas on the Role of Pacritinib in Myelofibrosis

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Mar 8, 2023

John Mascarenhas, MD

John Mascarenhas, MD, professor of medicine, the Icahn School of Medicine, Mount Sinai, director, the Center of Excellence for Blood Cancers and Myeloid Disorders, member, the Tisch Cancer Institute, Mount Sinai, discusses the role of pacritinib (Vonjo) in patients with myelofibrosis.

Pacritinib is JAK1-sparing, JAK2, FLT3, IRAK1, and ACVR1 inhibitor used in the treatment of patients with myelofibrosis in the setting of cytopenias at full dose to obtain spleen and symptom benefit, Mascarenhas says. In February 2022, the FDA granted an accelerated approval to pacritinib for the treatment of adult patients with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L.

The approval was based on data from the phase 3 PERSIST-1 (NCT01773187), phase 3 PERSIST-2 (NCT02055781), and phase 2 PAC203 (NCT04884191) trials, a dose-finding study, and the ongoing phase 3 PACIFICA trial (NCT03165734) is serving as a confirmatory trial following the agent’s accelerated approval, Mascarenhas expands.

Although pacritinib is approved for patients with platelets less than 50 × 109/L , PERSIST-2 also produced data in patients with a platelet count of less than 100 x 109/L. Prior to the approval of pacritinib, clinicians did not have a JAK2 inhibitor available for patients with a platelet count below that could be administered at the full dose, Mascarenhas notes. The JAK2 inhibitors fedratinib (Inrebic) and ruxolitinib (Jakafi) have labels patients with platelet counts of 50 × 109/L and greater. Since those two agents are more myelosuppressive, clinicians must attenuate the dose when treating patients with a platelet count below 50 × 109/L, reducing the spleen and symptom benefit generated by fedratinib and ruxolitinib, Mascarenhas says. Twenty 20 mg of pacritinib given twice daily can be delivered to patients with platelet counts at 20 × 109/or less and still produce spleen and symptom benefits with a favorable toxicity profile, Mascarenhas continues.

Studies have not demonstrated an increased risk of cardiovascular events with pacritinib, and data have shown reduced myelosuppression. There may be an increased risk of bleeding with pacritinib, and patients who have a known coagulopathy, have bleeding diathesis, or are receiving anticoagulation should be counseled appropriately, Mascarenhas expands. Although it is important consider the potential benefits and risks for each individual patient, pacritinib does provide an efficacious treatment option for patients with myelofibrosis with low platelet counts, Mascarenhas concludes.

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COVID-19 Vaccine Efficacy Decreases With Ruxolitinib Use in MPNs

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Mar 9, 2023

Ashling Wahner

Treatment with ruxolitinib impaired antibody responses to complete vaccination with the BNT162b2 SARS-CoV-2 vaccine in patients with myelofibrosis or polycythemia vera.

Treatment with ruxolitinib (Jakafi) impaired antibody responses to complete vaccination with the BNT162b2 SARS-CoV-2 vaccine in patients with myelofibrosis or polycythemia vera (PV), according to findings from a prospective, single-center study.1

After completing 2 doses of the vaccine, the average level of serum immunoglobulin G (IgG) neutralizing antibody levels against the receptor-binding domain portion of the spike protein of SARS-CoV-2 (anti-S) was 369.5 BAU/mL (range, 0-6901). Of the 43 patients who received 2 vaccine doses, 14 (32.6%) had anti-S antibody levels below the threshold for immunogenicity positivity, 27 had levels below 60 BAU/mL, and 28 had levels below 100 BAU/mL. Specifically, of the patients with myelofibrosis, 13 did not achieve an antibody response, and of those with PV, 1 did not achieve an antibody response.

Previously in 2020, results from the clinical trial investigating the efficacy of the BNT162b2 mRNA COVID-19 vaccine demonstrated that fully vaccinated patients gained 95% protection against the virus.2 However, this trial did not include patients from certain fragile patient populations, such as immunocompromised patients, in whom the vaccine’s protection may be lower because of their ongoing treatments and diseases.

More recent studies have shown that the vaccine is less effective in patients with hemato-oncological diseases. Previous research with the vaccine in small cohorts of patients with myeloproliferative neoplasms (MPNs) treated with ruxolitinib has shown that these patients may exhibit lower responses to the first and second doses of the vaccine, and that responses differ across MPNs. For instance, a study investigating long-term responses to the vaccine in patients with essential thrombocythemia (ET), PV, and myelofibrosis receiving ruxolitinib showed that median antibody titers were 811 BAU/mL (range, 184->2500), 274 BAU/mL (range, 1.7->2500), and 51.4 BAU/mL (range, 0.6-529), respectively, over 1 month after the second vaccine dose.3

“As little data were available in [patients with MPNs] treated with ruxolitinib who had completed the vaccination cycle (2 doses) and a third booster dose, in this study, we investigated whether these patients could reach a protective antibody level against the SARS-CoV-2 virus,” lead study author, Giuseppe A. Palumbo, MD, PhD, of the University of Catania in Italy, and colleagues, wrote in a paper of the data published in Frontiers in Oncology.1

This study evaluated 43 patients with primary myelofibrosis (n = 15), secondary myelofibrosis (n = 15), and PV (n = 13) who received ruxolitinib for their respective MPNs. All patients received both doses of the intramuscular BNT162b2 mRNA vaccine at 30 mcg per dose 3 weeks apart, per the Italian national guidelines. Patient sera samples were obtained at a median of 36 days (range, 14-53) after they received the second vaccine dose.

Of the 43 patients, 39 received the booster dose, 2 died before receiving the booster dose, and 2 refused the booster dose. Patients received their booster dose at a median of 153 days (range, 32-243) after their second dose. Sera samples were obtained from each patient before they received the booster dose, either on the same day or the day before, and at a median of 26 days (range, 11-49) following their booster dose.

All patients began ruxolitinib treatment at a median of 1236 days (range, 10-3370) before their first vaccine dose; 1414 days (range, 10-3288) for patients with myelofibrosis and 817 days (range, 53-3370) for those with PV. At the time of their first vaccine dose, patients were receiving ruxolitinib at a median of 14.7 mg twice daily (range, 2.5-25). The median ruxolitinib dose was 16.1 mg twice daily (range, 2.5-25) for patients with myelofibrosis and 11.5 mg twice daily (range, 5-15) for those with PV.

Of the patients with primary myelofibrosis, 2, 5, 7, and 1 had low-, intermediate-1–, intermediate-2–, and high-risk disease, respectively, when they received their first vaccine dose, per the Dynamic International Prognostic Scoring System (DIPSS). No patients had DIPSS score changes at the time of their booster dose. Of these patients, 13 had JAK2 V617F mutations, and 2 had CALR mutations.

Of the patients with secondary myelofibrosis, 1, 5, 5, and 4 had low-, intermediate-1–, intermediate-2–, and high-risk disease, respectively, when they received their first vaccine dose, per Myelofibrosis Secondary to PV and ET-Prognostic Model score. At the time of their booster dose, 1 patient each had progressed from low-risk to intermediate-1–risk disease and intermediate-2–risk to high-risk disease. Of these patients, 13 had JAK2V617F mutations, and 2 had CALR mutations.

Of the patients with PV, 12 had JAK2 V617F mutations and 1 had a JAK2 exon 12 mutation. All patients with PV had received hydroxyurea before switching to ruxolitinib. Seven patients switched because of hydroxyurea intolerance, and 6 patients switched because of hydroxyurea resistance per the European Leukemia Network consensus criteria.

At first vaccine dose, all patients had a median age of 69 years (range, 46-86); The median age was 72 years (range, 46-86) in patients with myelofibrosis and 64 years (range, 50-78) in those with PV. The overall median spleen size was 3 cm below the costal margin (range, 0-20), including a median of 4.6 cm (range, 0-20) in patients with myelofibrosis and 0 cm (range, 0-2) in those with PV.

This study evaluated vaccine immunogenicity by measuring the anti-S serum IgG neutralizing antibody levels. Per the World Health Organization, an anti-S value above 7 BAU was considered positive. The investigators also measured IgG against SARS-CoV-2 nucleocapsid proteins (anti-N) to determine whether patients had a prior or ongoing SARS-CoV-2 infection before or during vaccination.

Of all patients, 2 with primary myelofibrosis had anti-N IgG antibodies, indicating their exposure to SARS-CoV-2 prior to their first vaccine dose. These patients had a high post-vaccination antibody response, of 1333.9 BAU/mL and 6901.5 BAU/mL.

With 2 vaccine doses, patients achieved antibody levels at a median of 12.3 BAU/mL (range, 0-54.2). After the booster dose, patients reached a median antibody level of 272.3 BAU/mL (range, 0-1399). With the booster, 8/40 patients (20%), 7/27 with myelofibrosis and 1/12 with PV, did not achieve antibody levels above the positivity threshold. In total, 19 patients had antibody levels below 60 BAU/mL, and 24 had levels below 100 BAU/mL.

“Our study confirmed that [patients with] ruxolitinib-treated MPNs who have received 2 standard doses of BNT162b2 show a markedly impaired antibody production,” the study authors wrote. “Although the third booster dose was able to reduce the number of patients who remained fully negative, the median antibody value reached was not significantly better, and levels were far from those obtained with the same vaccine dose and schedule in normal subjects.”

The investigators recommend delaying ruxolitinib treatment in patients with myeloproliferative diseases until they have received at least 2 vaccine doses, and for patients and their caregivers to use COVID-19 risk mitigation strategies, including hygiene measures and social distancing.

References

  1. Palumbo GA, Cambria D, La Spina E, et al. Ruxolitinib treatment in myelofibrosis and polycythemia vera causes suboptimal humoral immune response following standard and booster vaccination with BNT162b2 mRNA COVID-19 vaccine. Front Oncol. Published online February 14, 2023. doi:10.3389/fonc.2023.1117815
  2. Polack FP, Thomas SJ, Kitchin N, et al. Safety and efficacy of the BNT162b2 mRNA Covid-19 vaccine. 2020;383(27):2603-2615. doi:10.1056/NEJMoa2034577
  3. Auteri G, Bartoletti D, Di Pietro C, et al. Longer-term response to SARS-CoV-2 vaccine in MPN patients: role of ruxolitinib and disease severity. Leuk Res. 2022;(5)116:106819. doi:10.1016/j.leukres.2022.106819

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CTI BioPharma Reports Fourth Quarter and Full Year 2022 Financial Results

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NEWS PROVIDED BY

CTI BioPharma Corp. 

Mar 06, 2023, 06:25 ET

– Growing physician awareness and usage of VONJO® (pacritinib) drove quarterly double-digit revenue growth –

– VONJO® net product revenue exceeded year-end goal with a total of $54 million for 2022 and $21.1 million in the fourth quarter, a 16% increase compared to the third quarter –

– ASH 2022 oral presentation featured new data on pacritinib’s ACVR1 inhibition and anemia benefit in myelofibrosis patients –

– Management to host webcast and conference call at updated time today at 8:30 a.m. ET –

SEATTLE, March 6, 2023 /PRNewswire/ — CTI BioPharma Corp. (Nasdaq: CTIC), a commercial biopharmaceutical company focused on the development and commercialization of novel targeted therapies for blood-related cancers, today reported its financial results for the fourth quarter and full year ended December 31, 2022.

“CTI is now established as a market leader in the treatment of cytopenic myelofibrosis following the accelerated approval and U.S. commercial launch of VONJO® (pacritinib) over the past year,” said Adam Craig, M.D., Ph.D., M.B.A., President, Chief Executive Officer and Interim Chief Medical Officer. “With the launch of VONJO in March 2022, we exceeded our year-end revenue goal by achieving $54 million in net sales in 2022 with strong quarter-over-quarter growth.”

“As of year-end 2022, more than 1,000 patients were treated with VONJO, which is a significant milestone for this rare disease. Importantly, we have also achieved over 90% insurance coverage with both Medicare and Commercial plans. To further increase the market penetration of VONJO, our commercial team continues to reinforce the VONJO clinical value proposition for cytopenic myelofibrosis patients leveraging peer-to-peer interactions and education. New data presented at the 64th American Society of Hematology (ASH) Annual Meeting suggests the potential to strengthen the clinical differentiation for pacritinib through its potent Activin A receptor type 1 (ACVR1) inhibitor and anemia benefit. We look forward to continuing activities focused on market expansion in 2023, which are intended to drive quarter-over-quarter net sales increases,” concluded, Dr. Craig.

2022 Key Accomplishments and Recent Highlights

  • FDA approval of VONJO (pacritinib) for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L.
  • $54 million in net sales in the first nine months following VONJO launch.
  • Over 1,000 patients commercially treated with VONJO in 2022.
  • Inclusion in the National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines in Oncology for Myeloproliferative Neoplasms, with a recommendation for VONJO as a: First-line treatment option for higher-risk myelofibrosis patients with platelet counts <50 x 109/L who are not candidates for transplant; Second-line treatment option for: patients with higher-risk myelofibrosis who are not candidates for transplant with platelet counts ≥50 x 109/L with no response or loss of response to one prior JAK inhibitor and for patients with symptomatic lower-risk myelofibrosis with platelet counts <50 x 109/L with no response or loss of response to initial treatment.
  • Oral presentation at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition highlighted pacritinib as a potent activin A receptor type 1 (ACVR1) inhibitor with significant anemia benefit in patients with myelofibrosis.
  • On February 7, 2023, VONJO was granted seven years of orphan-drug exclusive approval by the FDA for treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 x109/L. The seven-year exclusive approval began on February 28, 2022.

Fourth Quarter and Full Year 2022 Financial Results

  • Net Product Sales: Net product sales of $21.1 million and $53.9 million for the three months and year ended December 31, 2022, respectively, were entirely attributable to VONJO product sales in the United States. There were no product sales for the comparable periods in 2021.
  • Operating Loss: Operating loss was $13.6 million and $35.4 million for the three months ended December 31, 2022 and 2021, respectively, and $79.8 million and $95.3 million for the years ended December 31, 2022 and 2021, respectively. The decrease in operating loss between the three-month periods ended December 31, 2022 and 2021 was primarily attributable to VONJO product sales. The decrease in operating loss between the years ended December 31, 2022 and 2021 resulted primarily from VONJO product sales, partially offset by an increase in selling, general and administrative activities related to the commercial launch of VONJO, as well as a $10.3 million milestone expense related to FDA approval of VONJO, which was included in other operating expenses.
  • Net Loss: Net loss for the three months ended December 31, 2022 was $17.5 million, or $0.14 for basic and diluted loss per share, compared to net loss of $36.8 million, or $0.38 for basic and diluted loss per share, for the same period in 2021. Net loss for the year ended December 31, 2022 was $93.0 million, or $0.81 for basic and diluted loss per share, compared to net loss of $97.9 million, or $1.09 for basic and diluted loss per share, for the same period in 2021.
  • Cash Position: As of December 31, 2022, cash, cash equivalents and short-term investments totaled $79.9 million, compared to $65.4 million as of December 31, 2021. Subsequent to the end of the quarter, the Company received $6.5 million in additional contractual funding from DRI Healthcare Trust in January 2023.

Conference Call and Webcast

CTI will host a webcast and conference call at 8:30 a.m. ET today to review its fourth quarter and full year 2022 financial results and provide a corporate update. The live and archived webcast may be accessed on the CTI BioPharma website under the Investors & Media section: Events and Presentations. To participate via telephone, please register in advance using the link provided in the event listing. The Company suggests participants log in 15 minutes in advance of the event.

About VONJO® (pacritinib) capsules

VONJO® (pacritinib) is an oral kinase inhibitor with activity against wild type Janus Associated Kinase 2 (JAK2), mutant JAK2V617F form, IRAK1, ACVR1 (ALK2) and FLT3, which contribute to signaling of a number of cytokines and growth factors that are important for hematopoiesis and immune function. Myelofibrosis is often associated with dysregulated JAK2 signaling. At clinically relevant concentrations, pacritinib does not inhibit JAK1.

VONJO is indicated for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). CTI is conducting the Phase 3 PACIFICA study of VONJO in patients with myelofibrosis and severe thrombocytopenia as a post-marketing requirement.

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Dr. Jain on Unmet Needs in Patients with Myelofibrosis Undergoing Allo-SCT

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Tania Jain, MBBS, director, Adult Chimeric Antigen Receptor T-cell Therapy Program for Hematological Malignancies, Sidney Kimmel Comprehensive Cancer Center, Assistant Professor of Oncology, Johns Hopkins Medicine, discusses the unmet needs for patients with myelofibrosis who receive an allogeneic stem cell transplant (allo-SCT).

At the 2023 Transplantation and Cellular Therapy Meetings, Jain presented data from a retrospective study evaluating outcomes for patients with myelofibrosis who had haploidentical donors (HD) and posttransplant cyclophosphamide vs other common donor types.

Findings from the study reflected that investigators still have considerable work to do in order to optimize allo-SCTs for patients with myelofibrosis, Jain begins. One area of need is relapse prevention, Jain says, adding that investigators are working to set up studies evaluating maintenance strategies post–allo-SCT to improve relapse rates.

Moreover, in the retrospective study, investigators showed that relapse rates were higher for patients with a larger spleen size going into transplant, Jain says. Novel agents currently under investigation in myelofibrosis could improve spleen response in patients, Jain continues. These could be beneficial for patients who will undergo allo-SCT, and it will be helpful to observe what a improved spleen responses with advanced therapies could mean for patients achieve in terms of transplant outcomes, Jain explains.

If patients with myelofibrosis are going to be referred for transplant, it should be done early before they have the chance to get worse or progress, Jain continues. The longer the patient is not referred, the quicker the window for transplant closes, and sicker patients do not do as well on clinical trials, Jain says. Moreover, it is important not to delay transplant looking for a perfectly matched donor when there are options with different donor types that can also help patients, which is important to consider, Jain concludes.

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Incyte Provides Update On Interim Analysis Of Phase 3 LIMBER-304 Study Of Parsaclisib And Ruxolitinib In Patients With Myelofibrosis

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March 3, 2023 at 4:35 PM EST
  • Independent data monitoring committee advises study unlikely to meet primary endpoint, leading to decision to discontinue the study

WILMINGTON, Del.–(BUSINESS WIRE)–Mar. 3, 2023– Incyte (Nasdaq:INCY) today announced that it will discontinue the Phase 3 LIMBER-304 trial following results of a pre-planned interim analysis conducted by an independent data monitoring committee (IDMC) indicating that the study is unlikely to meet the primary endpoint in the intent-to-treat patient population. The recommendation to stop the study was not due to safety. LIMBER-304 is a randomized, double-blind study evaluating the efficacy and safety of parsaclisib plus ruxolitinib (Jakafi®) versus placebo plus ruxolitinib in adult (age ≥18 years) patients living with myelofibrosis (MF) who have an inadequate response to ruxolitinib monotherapy.

While further review of the data is conducted, Incyte will inform investigators of the results and work with them to appropriately conclude the study in a manner consistent with the best interest of each patient. Data from this study will be submitted for presentation at an upcoming scientific meeting.

The primary endpoint of LIMBER-304 (NCT04551053) was the proportion of patients achieving targeted reduction in spleen volume as measured by magnetic resonance imaging or computed tomography. Secondary endpoints included the proportion of patients who have a targeted reduction in Total Symptom Score (TSS), change in TSS, time to the first ≥50% reduction in TSS, overall survival, number of treatment emergent adverse events, time of onset of targetedreduction in spleen volume and duration of maintenance of targeted reduction in spleen volume.

About LIMBER

Incyte is a leader in the discovery and development of therapies for patients with myeloproliferative neoplasms (MPNs) and graft-versus-host disease (GVHD). The LIMBER clinical trial program is designed to evaluate multiple monotherapy and combination strategies to improve and expand treatments for patients with MPNs and GVHD. These include ruxolitinib-based combinations with BET and ALK2, new therapeutic options including axatilimab and novel targets such as mutant CALR.

About Incyte

Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.

Forward-Looking Statements

Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the presentation of data from the Company’s ongoing clinical development program for parsaclisib and ruxolitinib, development plans for ruxolitinib and further development in myelofibrosis, contain predictions, estimates and other forward-looking statements.

These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA; the Company’s dependence on its relationships with its collaboration partners; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission, including its annual report for the year ended December 31, 2022. The Company disclaims any intent or obligation to update these forward-looking statements.

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