On May 14, 2024 Calidi Biotherapeutics Inc. (NYSE American: CLDI) ("Calidi"), a clinical-stage biotechnology company developing a new generation of targeted virotherapies, reported its first quarter 2024 operating and financial results and reviewed recent business highlights (Press release, Calidi Biotherapeutics, MAY 14, 2024, View Source [SID1234643275]).

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"Calidi’s current focus is on raising additional capital and implementing continued cost cutting measures in order to advance our promising pipeline of novel immunotherapies designed to successfully target and eliminate solid tumors across the body. We plan to present three posters at ASCO (Free ASCO Whitepaper) including an update from our ongoing trial with City of Hope for recurrent high-grade glioma," said Allan Camaisa, CEO and Chairman of the Board at Calidi Biotherapeutics. "In parallel to our clinical progress, Calidi has strengthened our preclinical programs through our powerful R&D engine, unveiled our novel systemic delivery platform to target diverse tumor types, and executed a capital raise to strengthen our balance sheet. We believe these efforts will help propel the company through important milestones including an interim clinical update from our ongoing Phase 1 trial and the initiation of the Phase 1 trial with Northwestern University anticipated in the third quarter of 2024, subject to additional capital being raised."

First Quarter 2024 and Recent Corporate Developments

Announced upcoming poster presentations at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The posters will include an update from the Phase 1 trial in collaboration with City of Hope for CLD-101 in recurrent high-grade glioma. Additional posters will focus on preclinical data from the company’s RTNova (CLD-400) and CLD-201 platforms.
Presented new data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (AACR) (Free AACR Whitepaper) supporting CLD-101 and CLD-201’s first-in-class use of stem cells to protect and potentiate antitumor virotherapies. The results of experiments, conducted in collaboration with City of Hope, provide further detail around the unique stem cell-based mode of delivery and the therapeutic potential at the core of Calidi’s novel technology.
Highlighted the company’s novel RTNova systemic platform, a targeted enveloped virotherapy, that has demonstrated preclinically the ability to resist inactivation by human serum and an ability to target diverse tumor types. This novel therapeutic approach has the potential to be available to a diverse patient population with different forms of cancer while remaining cost-effective and easy to administer.
Closed public offering of 15,197,500 shares of common stock and accompanying warrants, with gross proceeds of $6.1 million.
First Quarter 2024 Financial Results

The company reported a net loss of $7.2 million, or $0.20 per share, for the three months ended March 31, 2024, compared to a net loss of $6.5 million, or $0.75 per share, for the same period in 2023.

Research and development expenses were $2.7 million for the three months ended March 31, 2024, compared to $2.6 million for the comparable period in 2023, respectively.

General and administrative expenses were $4.0 million for the three months ended March 31, 2024, compared to $2.8 million for the comparable period in 2023, respectively.

The company had approximately $1.2 million in cash and $0.2 million in restricted cash as of March 31, 2024, compared to $1.9 million in cash and $0.2 million in restricted cash as of December 31, 2023.

On May 14, 2024 Moffitt Cancer Center, a world-renowned cancer treatment and research center, and Fulgent Pharma, a subsidiary of Fulgent Genetics, Inc. (NASDAQ: FLGT) and a leading nanobiotechnology company specializing in innovative cancer therapeutics, reported a strategic partnership aimed at advancing cancer treatment through clinical development and pre-clinical co-development initiatives (Press release, Fulgent Pharma, MAY 14, 2024, View Source [SID1234643274]). This collaboration combines Moffitt’s cutting-edge clinical capabilities with Fulgent’s groundbreaking nanotherapeutics and genomics platforms in an effort to accelerate the development of personalized cancer therapies. Under the agreement, Moffitt will provide Fulgent with priority access to its clinical expertise and resources, with the aim of expediting the advancement of Fulgent’s clinical pipeline. This priority access at Moffitt includes prioritized clinical trial activation, enhanced patient screening, and data sharing designed to get investigational therapies to patients in need more quickly and efficiently.

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Fulgent Pharma, a leader in nanoencapsulation technology, has transformed paclitaxel, a chemotherapy drug known for its poor solubility, into a soluble counterpart suitable for intravenous injection called FID-007. In Phase 1 clinical studies conducted with FID-007 to date, substantial tumor reduction across various cancer types, including breast, head and neck, lung, bile duct, and pancreatic cancers, has been observed.

Additionally, the partnership will focus on co-developing next-generation personalized treatment options for cancer. Moffitt will leverage its broad scientific and immunology expertise, access to tissue samples and nonclinical research resources, while Fulgent will contribute its novel and proprietary nano-particle-based drug development platform along with its broad spectrum genetic and genomic testing capabilities.

"Pooling our unique strengths, Moffitt and Fulgent will strive to create new precision oncology therapies customized to meet the specific needs of each patient, thereby improving future outcomes," said Fulgent Pharma President and CSO Ray Yin, Ph.D.

"This partnership aligns with Moffitt’s mission to contribute to the prevention and cure of cancer. We are looking forward to working with Fulgent to accelerate the translation of scientific discoveries into lifesaving treatments for our patients," said Moffitt President and CEO Patrick Hwu, M.D.

"We are excited to collaborate with Moffitt Cancer Center, a global cancer research and treatment leader," said Ming Hsieh, CEO of Fulgent Genetics. "By combining Moffitt’s clinical and research expertise with Fulgent’s innovative nanotherapeutic platform and growing patent portfolio, we believe we can make significant strides in advancing personalized cancer treatment options."

On May 14, 2024 Nuvation Bio Inc. (NYSE: NUVB), a late-stage, global biopharmaceutical company tackling some of the greatest unmet needs in oncology by developing differentiated and novel therapeutic candidates, reported financial results for the first quarter ended March 31, 2024, and provided a business update (Press release, Nuvation Bio, MAY 14, 2024, View Source [SID1234643273]).

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"The first quarter of 2024 included multiple significant events for Nuvation Bio. We announced and subsequently completed the acquisition of AnHeart Therapeutics, which transformed Nuvation Bio into a late-stage, global company developing multiple clinical programs for some of the most difficult-to-treat cancers. We also treated the first patient in a Phase 1/2 study of NUV-1511, our first DDC to enter the clinic," said David Hung, M.D., Founder, President, and Chief Executive Officer of Nuvation Bio. "With our robust cash balance and the recent appointment of Colleen Sjogren as our Chief Commercial Officer, along with some new key hires and the integration of new colleagues from AnHeart, we are thoughtfully scaling our organization to execute on our goal of potentially becoming a commercial organization by the end of 2025, while continuing to advance multiple internal programs in various stages of preclinical and clinical development."

Recent Pipeline Updates:

Taletrectinib, ROS1 inhibitor: Advanced ROS1-positive non-small cell lung cancer (NSCLC)

Taletrectinib is being evaluated for the treatment of patients with advanced ROS1-positive NSCLC in two Phase 2 single-arm pivotal studies: TRUST-I in China, and TRUST-II, a global study.
Updated data from the Phase 2 TRUST-I clinical study (NCT04395677) evaluating taletrectinib in patients in China with advanced ROS1-positive NSCLC will be reported in an oral presentation (abstract #8520) at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting on Saturday, June 1, 2024, at 4:30-6:30 p.m. CT/5:30-7:30 p.m. ET.
Safusidenib, mIDH1 inhibitor: Grades 2 and 3 IDH1-mutant glioma

Safusidenib is being evaluated in a global Phase 2 study for the treatment of patients with grades 2 and 3 IDH1-mutant glioma.
NUV-868, BD2-selective BET inhibitor: Advanced solid tumors

NUV-868 is being evaluated in a Phase 1b dose escalation study in combination with olaparib for the treatment of patients with ovarian cancer, pancreatic cancer, metastatic castration-resistant prostate cancer (mCRPC), triple negative breast cancer, and other solid tumors, and in combination with enzalutamide for the treatment of patients with mCRPC.
NUV-1511, DDC: Advanced solid tumors

NUV-1511, the Company’s first clinical-stage DDC, is being evaluated in a Phase 1/2 study for the treatment of patients with advanced solid tumors who previously received and progressed on or after treatment with Enhertu and/or Trodelvy per approved U.S. Food and Drug Administration (FDA) labeling, human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer, mCRPC, advanced pancreatic cancer, and platinum-resistant ovarian cancer (PROC).
Corporate Updates:

Announced entry into a definitive agreement to acquire AnHeart Therapeutics in March 2024 and completed the acquisition in April 2024.
Appointed Colleen Sjogren as Chief Commercial Officer. Ms. Sjogren will lead Nuvation Bio’s commercial strategy and operations, including marketing, sales, and market access.
First Quarter 2024 Financial Results

As of March 31, 2024, Nuvation Bio had cash, cash equivalents and marketable securities of $597.0 million.

For the three months ended March 31, 2024, research and development expenses were $12.8 million, compared to $18.8 million for the three months ended March 31, 2023. The decrease was primarily due to a $6.7 million decrease in third-party research services and drug manufacturing costs as a result of completing the Phase 1 monotherapy study of NUV-868, offset by a $0.7 million increase in personnel-related costs driven by stock-based compensation and other benefits.

For the three months ended March 31, 2024, general and administrative expenses were $7.3 million, compared to $7.7 million for the three months ended March 31, 2023. The decrease was due to a $0.5 million decrease in professional fees, $0.4 million decrease in insurance, and a $0.2 million decrease in personnel-related costs, offset by a $0.4 million increase in legal fees, a $0.2 million increase in occupancy expense, and a $0.1 million increase in miscellaneous expense.

For the three months ended March 31, 2024, Nuvation Bio reported a net loss of $14.8 million, or $(0.07) per share. This compares to a net loss of $21.7 million, or $(0.10) per share, for the comparable period in 2023.

On May 14, 2024 Nabla Bio (aka "Nabla"), pioneers in generative protein design, reported the close of a $26 million Series A financing, led by Radical Ventures with participation from all existing investors, and strategic collaborations with AstraZeneca, Bristol Myers Squibb Company and Takeda, worth more than $550 million in upfront and milestone payments, plus royalties (Press release, Nabla Bio, MAY 14, 2024, View Source [SID1234643272]).

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Nabla develops integrated AI and wet-lab technologies that enable atomically precise drug design and high-throughput measurement of drug function, with an initial focus on antibodies targeting multipass membrane proteins, including for example, G protein-coupled receptors (GPCRs), ion channels, and transporters.

"We are unlocking new opportunities to build highly selective drugs against validated, but hard-to-drug targets with a degree of structural precision not previously possible," said Nabla Co-Founder and CEO Surge Biswas. "We are excited to welcome Radical Ventures as our newest investor and to announce collaborations with three of the world’s largest pharmaceutical companies to help us bring our work closer to patients."

Today, nearly all approved protein drugs are focused on soluble, extracellular targets, for which traditional brute force drug discovery technologies can yield antibodies with comparative ease. On the other hand, multipass membrane proteins are far more challenging to drug, due to their membrane-integrated nature, limited extracellular availability, conformational dynamics, and structural similarity across targets.

Given multipass membrane targets comprise two-thirds of all cell surface proteins and their central and varied involvement in controlling cell behavior, these targets have long been the "holy grail" of protein drug development. Nabla’s generative protein design platform is unlocking hundreds of these previously inaccessible targets by enabling the precise design of conformation- and target-selective antibody binders.

"With the technologies we’re developing, we could double the number of disease-relevant drug targets the industry goes after," said Nabla Co-Founder Frances Anastassacos. "That’s an untapped therapeutic opportunity and why several leading pharmaceutical companies have collaborated with us."

"We’re excited to be working with Nabla and using their generative antibody design and high throughput screening capabilities with the aim to design new drug candidates against challenging targets," said Puja Sapra, Senior Vice President of Biologics Engineering and Oncology Targeted Delivery, AstraZeneca.

To maximize the patient impact of its platform, Nabla collaborates with leading pharmaceutical companies with a track record of successful drug development to expand their pipelines with high-quality drug candidates against some of the most challenging, high-impact targets. These collaborations also offer valuable insights into disease biology and platform development while offsetting a significant portion of R&D expense, enabling Nabla to grow quickly and sustainably.

Across its collaborations, Nabla has demonstrated the broad applicability of its platform, beyond drug design for multipass membrane proteins. This includes, for example, the design of novel cytokines, complex multi-domain antibodies, and receptor traps with greater in vitro activity and developability than leading drugs on the market. These results are an important step in translating generative protein design to clinical therapies and evidence of Nabla’s expanding capabilities.

"Generative AI and large language models are poised to master the language of proteins in the same way that they have mastered natural language in recent years, with profound implications for medicine," said Radical Ventures Partner Rob Toews. "The world-class team at Nabla Bio helped pioneer this field. Their groundbreaking work on multipass membrane protein targets offers to revolutionize antibody therapeutics and has won them major commercial partnerships with several of the world’s largest pharmaceutical companies. We are thrilled to lead Nabla’s Series A."

Nabla Bio launched in December 2021 with funding from Khosla Ventures and Zetta Venture Partners, to apply cutting-edge generative models to the design of protein-based therapies. The company builds on co-founder Surge Biswas’ work on the first protein language models published in a series of Nature Methods and Nature Biotechnology papers (2019, 2021, 2022) alongside colleagues in George Church’s Harvard Lab, which helped establish the field. Frances Anastassacos co-founded Nabla after working in biotech venture capital and completing her PhD at Harvard.

On May 14, 2024 Incyte (Nasdaq: INCY) reported that several abstracts featuring data from its oncology portfolio will be presented at the upcoming 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting held May 31 – June 4 in Chicago, and at the European Hematology Association (EHA) (Free EHA Whitepaper) 2024 (EHA2024) Congress held on June 13-16 in Madrid, Spain, and virtually (Press release, Incyte, MAY 14, 2024, View Source [SID1234643271]).

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"The data being presented at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) demonstrates Incyte’s dedication to our comprehensive research approach to identifying new first and best in class treatments for cancer patients," stated Steven Stein, M.D., Chief Medical Officer at Incyte. "The research we’re presenting emphasizes promise within our oncology pipeline in areas where there is a continued need for therapeutic development across a breadth of cancers."

Key abstracts accepted for presentation at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) include:

ASCO Abstracts

Abstracts will be available to registered attendees on the ASCO (Free ASCO Whitepaper) Congress platform beginning on May 23, 2024. Posters and slides will be available to registered attendees at the scheduled session start time.

Poster Presentations

INCB099280

A Phase 1 study of the Small-Molecule PD-L1 inhibitor INCB099280 in Select Advanced Solid Tumors: Updated Safety, Efficacy, and Pharmacokinetics (PK) Results (Abstract #2608. Session: Developmental Therapeutics—Immunotherapy. Saturday, June 1, 9:00 a.m. -12:00 p.m. ET)

INCB057643

Bromodomain and Extra-Terminal (BET) Inhibitor INCB057643 in Patients with Relapsed or Refractory Myelofibrosis (MF) and Other Advanced Myeloid Neoplasms: A Phase 1 Study (Abstract #6576. Session: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant. Monday, June 3, 9:00 a.m.-12:00 p.m. ET)

EHA Abstracts

Abstracts are available on the EHA (Free EHA Whitepaper)2024 Congress platform and accessible for on-demand viewing until August 15, 2024.

Oral Presentations

Ruxolitinib

Analysis of Molecular Mechanisms and Predictive Biomarkers of Disease Transformation in Polycythemia Vera (Abstract #S217. Topic: Myeloproliferative Neoplasms – Biology & Translational Research. Friday, June 14, 8:45 a.m. – 10:00 a.m. ET)

Poster Presentations

Ruxolitinib

A Real-World Evaluation of Risk Factors for Disease Progression in Patients With Polycythemia Vera (PV) Enrolled in REVEAL (Abstract #P1047. Topic: Myeloproliferative Neoplasms – Clinical. Friday, June 14)

Ruxolitinib Treatment in Patients With Polycythemia Vera Reduces JAK2 Allele Burden and Improves Hematocrit Control and Symptom Burden (Abstract #P1049. Topic: Myeloproliferative Neoplasms – Clinical. Friday, June 14)

The Impact of New or Worsening Anemia on Clinical Outcomes in 2233 Patients With Myelofibrosis Treated With Ruxolitinib: Results From the Expanded-Access JUMP Study (Abstract #P1044. Topic: Myeloproliferative Neoplasms – Clinical. Friday, June 14)

High Rate of Disease Progression in Patients With Low-Risk Myelofibrosis (MF) Enrolled in the Prospective MOST Study (Abstract #P1053. Topic: Myeloproliferative Neoplasms – Clinical. Friday, June 14)

Progression to Myelofibrosis in Patients With Essential Thrombocythemia: An Analysis From the Prospective MOST Study (Abstract #P1030. Topic: Myeloproliferative Neoplasms – Clinical. Friday, June 14)

Zilurgisertib

ALK2 and JAK2 Inhibition for Improved Treatment of Anemia in Myelofibrosis Patients: Preclinical Profile of an ALK2 Inhibitor Zilurgisertib in Combination With Ruxolitinib (Abstract #P1019. Topic: Myeloproliferative Neoplasms – Biology & Transitional Research. Friday, June 14)

The Activin Receptor-Like Kinase-2 Inhibitor Zilurgisertib (INCB000928) as Monotherapy or With Ruxolitinib in Patients with Anemia Due to Myelofibrosis: Phase 1/2 Study Results (Abstract #P1060. Topic: Myeloproliferative Neoplasms – Clinical. Friday, June 14)

INCB057643

Bromodomain and Extra-terminal (BET) Inhibitor INCB057643 in Patients With Relapsed or Refractory Myelofibrosis (MF) and Other Advanced Myeloid Neoplasms: A Phase 1 Study (Abstract #P1065. Topic: Myeloproliferative Neoplasms – Clinical. Friday, June 14)

INCA033989

Efficacy of INCA033989 in Chronic and Advanced Forms of CALRdel52 and CALRins5 MPN models (Abstract #P1002. Topic: Myeloproliferative Neoplasms – Biology & Translational Research. Friday, June 14)

INCB160058

Preclinical Evaluation of INCB160058 – A Novel and Potentially Disease-Modifying Therapy for JAK2V617F Mutant Myeloproliferative Neoplasms (Abstract #P1028. Topic: Myeloproliferative Neoplasms – Biology & Translational Research. Friday, June 14)

Axatilimab

Axatilimab for Chronic Graft-Versus-Host Disease: Responses in Fibrosis-Dominant Organs in AGAVE-201 (Abstract #P1321. Topic: Stem Cell Transplantation – Clinical. Friday, June 14)

Pemigatinib

A Phase 2 Study of Pemigatinib (FIGHT-203; INCB054828) in Patients With Myeloid/Lymphoid Neoplasms With Fibroblast Growth Factor Receptor 1 Gene Rearrangement (Abstract #P1042. Topic: Myeloproliferative Neoplasms – Clinical. June 14)

Tafasitamab

CD19 Expression Persists in Diffuse Large B-Cell Lymphoma Patient Biopsies After Treatment With Tafasitamab (Abstract #P1234. Topic: Lymphoma Biology & Translational Research. Friday, June 14

EARLYMIND, a Retrospective, Multicentric Study in Real World Settings to Characterize the Efficacy of Tafasitamab-Lenalidomide in Transplant Ineligible Patients With Relapsed/Refractory Large B-cell Lymphoma (Abstract #P1214. Topic: Aggressive Non-Hodgkin Lymphoma – Clinical. Friday, June 14)

For full session details and data presentation listings, please see the ASCO (Free ASCO Whitepaper) (View Source) and EHA (Free EHA Whitepaper)2024 (View Source) online programs.

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 older.

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 Monjuvi (tafasitamab-cxix) [or Minjuvi (tafasitamab)]

Monjuvi (tafasitamab-cxix) [or Minjuvi (tafasitamab)] is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. 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). MorphoSys and Incyte entered into: (a) in January 2020, a collaboration and licensing agreement to develop and commercialize tafasitamab globally; and (b) in February 2024, an agreement whereby Incyte obtained exclusive rights to develop and commercialize tafasitamab globally.

In the United States, Monjuvi (tafasitamab-cxix) received accelerated approval 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). In Europe, Minjuvi (tafasitamab) received conditional Marketing Authorization from the European Medicines Agency 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).

XmAb is a registered trademark of Xencor, Inc.

Monjuvi, Minjuvi, the Minjuvi and Monjuvi logos and the "triangle" design are registered trademarks of Incyte.

About Pemazyre (pemigatinib)

Pemazyre (pemigatinib) 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 test*. 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.