On March 27, 2023 ncyte (Nasdaq:INCY) reported that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved Pemazyre (pemigatinib), a selective fibroblast growth factor receptor (FGFR) inhibitor, for the treatment of myeloid/lymphoid neoplasms (MLNs) with FGFR1 fusion (also known as 8p11 myeloproliferative syndrome) (Press release, Incyte, MAR 27, 2023, View Source [SID1234629402]). MLNs are a rare, aggressive group of cancers characterized by an over-production of myeloid cells, or bone tissue, with the tendency to rapidly progress to an acute myeloid leukemia (AML).

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"The MHLW approval of Pemazyre in MLNs is an important step toward potentially providing a therapeutic option for Japanese patients with this rare condition," said Lothar Finke, M.D., Ph.D., Group Vice President and General Manager, Incyte Asia. "Bringing this first and only approved therapy to MLN patients in Japan demonstrates Incyte’s commitment to finding solutions for critical unmet medical needs regardless of the size of the patient population – as is the case with MLNs, an extremely rare disease that is estimated to affect fewer than 100 patients worldwide."

The approval was based on data from the Phase 2 FIGHT-203 study, a multicenter open-label, single-arm trial that evaluated the safety and efficacy of Pemazyre in 41 patients in myeloid or lymphoid neoplasms with FGFR1 fusion gene positive who received Pemazyre 13.5 mg orally once daily continuously or intermittently. The primary endpoint, investigator-assessed complete response rate, was 62.5% (95% CI: 45.8 – 77.3). The complete response rate in the continuous dosing population was 66.7% (95% CI: 46.0 – 83.5). The most common adverse reactions observed in patients receiving Pemazyre were hyperphosphatemia (70.7%), alopecia (56.1%), diarrhea (43.9%) and stomatitis (43.9%).

Previously, the MHLW granted Orphan Drug Designation (ODD) for Pemazyre – a designation granted to investigational compounds intended to treat rare diseases that affect fewer than 50,000 people in Japan, and for which there is a high medical need1. Designated orphan drugs are also eligible for priority review for marketing authorizations to ensure supply to clinical settings at the earliest opportunity1. The MLN ODD is the second such designation granted to Pemazyre by the MHLW, having also received ODD for cholangiocarcinoma, a type of biliary tract cancer.

MLNs with FGFR1 rearrangement are a form of very rare hematological cancers caused by a chromosomal abnormality (translocation) in which the chromosome breaks where the FGFR1 gene is located (position 11 in the short arm of chromosome 8: location 8p11) and fuses with fragments (genes) of other chromosomes. Various partner genes cause a constitutive activation of the FGFR1 tyrosine kinase, impacting cell proliferation and survival. These cancers are largely divided into two phases according to clinical presentation: the chronic phase, if diagnosed as myeloproliferative disorder or myelodysplastic syndrome, or acute phase, if diagnosed as acute leukemia. The prognosis is unfavorable, and while allogenic hematopoietic stem cell transplant is considered to be the only currently available treatment option that may achieve cure or long-term remission, no standard of care has been established.

About FIGHT-203

FIGHT-203 is a Phase 2, multicenter trial that enrolled patients 18 years and older with myeloid/lymphoid neoplasms (MLNs) with a fibroblast growth factor receptor 1 (FGFR1) rearrangement. Sponsored by Incyte, the study evaluated the safety and efficacy of pemigatinib for the treatment of adults with MLNs with FGFR1 rearrangement. Patients received pemigatinib 13.5 mg once daily in 21-day cycles, either on a continuous schedule (the approved recommended starting dosage for use in patients with MLNs with FGFR1 rearrangement) or as an intermittent schedule (14 days on, 7 days off, an unapproved dosage regimen in MLN with FGFR1 rearrangement). Pemigatinib was administered until disease progression or unacceptable toxicity or until patients were able to receive allo-HSCT. For more information about the study, please visit View Source

About 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 test2. 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 and the treatment of myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement.

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.

Incyte has granted Innovent Biologics, Inc. rights to develop and commercialize pemigatinib in hematology and oncology in Mainland China, Hong Kong, Macau and Taiwan. Incyte has retained all other rights to develop and commercialize pemigatinib outside of the United States.

Pemazyre is a trademark of Incyte Corporation.

On March 27, 2023 -Gamida Cell Ltd. (Nasdaq: GMDA), a cell therapy pioneer working to turn cells into powerful therapeutics, reported a business update and financial results for the year ended December 31, 2022 (Press release, Gamida Cell, MAR 27, 2023, View Source [SID1234629401]). Net loss for 2022 was $79.4 million, compared to a net loss of $89.8 million in 2021. As of December 31, 2022, Gamida Cell had total cash and cash equivalents of $64.7 million.

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The company highlighted positive data, productive regulatory interactions and progress on commercial readiness activities supporting its lead product candidate, omidubicel, an advanced cell therapy candidate for allogeneic stem cell transplant, as it advances toward its May 1, 2023 target Prescription Drug User Fee Act (PDUFA) action date. The company also announced a strategic restructuring of its operations to prioritize launch of omidubicel to ensure that, if approved, patients who may potentially benefit will have access to therapy. To reduce expenses, the company will discontinue development of its preclinical NK cell therapy candidates while continuing to enroll patients in the GDA-201 Phase 1 clinical trial.

"Our mission is to bring potentially curative therapies to patients," said Abbey Jenkins, President and Chief Executive Officer of Gamida Cell. "We believe we have a clear path to approval and are preparing for the commercial launch of omidubicel, if approved. Given the challenging economic environment, to date, we have not been able to raise adequate funding to support our full pipeline and enable a more robust launch of omidubicel, if approved. As a result, we are taking decisive actions to do three things 1) prioritize resources toward the launch 2) reduce expenses across the board 3) seek potential commercial or strategic partnerships to maximize patient access to omidubicel, a potentially life-saving therapy. Today’s actions are difficult. Especially since our engineered NK cell therapy candidates, which are derived from healthy donors, have demonstrated encouraging pre-clinical data that differentiate them from other NK cell therapy approaches. The science is promising, but these changes are economically necessary to ensure omidubicel reaches as many patients as possible."

Today Gamida Cell announced it would:

Implement a strategic restructuring to focus on omidubicel launch, if approved: The company intends to allocate the vast majority of its resources to executing a launch of omidubicel, if approved, although with a more limited investment and slower ramp than previously planned in order to manage its financial resources. The company reported productive interactions with the FDA, including a recently completed Late Cycle Meeting and a previously reported Pre-Licensing Inspection of the company’s Kiryat Gat, Israel, manufacturing facility, with no 483 observations received to date. Recently presented data continue to support the clinical benefits and safety of omidubicel, which, if approved, may be a valuable new donor source for patients in need of allogeneic stem cell transplant. Commercial readiness activities have made progress as the company prepares to onboard approximately 10-15 of the top 70 transplant centers in the United States in 2023. Omidubicel has received positive feedback from leading transplant centers, including ones that did not participate in the company’s clinical trials. The company has met with U.S. payers, including payers that cover more than 90% of commercially covered lives, and reported that payers indicate they anticipate covering a one-time therapy with curative intent.
Continue its GDA-201 Phase 1 study: The company will continue to enroll patients in its GDA-201 Phase 1 dose escalation study.
Reduce operating expenses in order to extend its cash runway: The company will discontinue the development of its engineered NK cell therapy preclinical pipeline, including GDA-301, GDA-501 and GDA-601, while maintaining the IP to these candidates. It will implement a headcount reduction of 17%, with the majority of impacted headcount tied to the discontinuation of the pre-clinical NK cell therapy candidates. The company will also close its operations in Jerusalem and consolidate Israel operations at its state-of-the-art manufacturing facility in Kiryat Gat. These changes are expected to extend the company’s cash runway through Q3 2023.
Explore strategic options: The company intends to seek potential commercial or strategic partnerships to maximize patient access to omidubicel, if approved.
Fourth Quarter and Recent Developments

Omidubicel: Advanced Cell Therapy

New data presented at ASH (Free ASH Whitepaper) and TCT: The company presented new data characterizing peripheral blood lymphocytes measured in correlation with time to neutrophil and platelet engraftment in omidubicel-transplanted and standard cord blood-transplanted patients at the 2023 Tandem Meetings, Transplantation & Cellular Therapy (TCT) Meetings of the American Society for Transplantation & Cellular Therapy and the Center for International Blood and Marrow Transplant Research in February. Seven days post-transplant, omidubicel-transplanted patients showed a statistically significant correlation between CD3+/CD4+ T cell counts and time to neutrophil engraftment. Similar correlations were noted between CD3+/CD8+/CD19+ cell counts and time to platelet engraftment. Patients transplanted with standard cord blood showed no such correlations at Day 7 post-transplant, and only began to show correlations starting at 14 days post-transplant. Data support past findings that omidubicel stimulates a faster immune response than standard cord blood, which may be a contributing mechanism resulting in the lower incidence of serious bacterial, fungal and viral infections for omidubicel-transplanted patients.
New publication in press: The company reported a publication in press in Transplantation and Cellular Therapy, now available online, reporting on long-term follow-up of patients transplanted with omidubicel across five clinical trials. The analysis showed a three-year overall survival of 62.5% and disease-free survival of 54%. With up to 10 years of follow-up, omidubicel showed durable hematopoiesis.
Manufacturing readiness: The company’s state-of-the-art manufacturing facility in Kiryat Gat, Israel, is ready for commercial launch if omidubicel is approved and is currently producing omidubicel for the company’s Extended Access Program (EAP) and its ongoing omidubicel aplastic anemia study. The facility, which has completed its Israeli Ministry of Health and FDA pre-licensure inspections with no 483 observations to date, has the ability to deliver omidubicel back to the transplant center within approximately 30 days from the start of manufacturing.
Commercial readiness: The company continues to advance efforts throughout the organization to prepare for the launch of omidubicel, if approved.
GDA-201: Intrinsic NK Cell Therapy

New data presented at Tandem Meetings: The company presented a poster at the 2023 Tandem Meetings, Transplantation & Cellular Therapy (TCT) Meetings of the American Society for Transplantation & Cellular Therapy and the Center for International Blood and Marrow Transplant Research reporting new preclinical data on the cryopreserved formulation of GDA-201, which showed increased potency and enhanced cytotoxicity. GDA-201 cells were tested for viability, phenotyping, function and potency. Previous characterization of GDA-201 showed high levels of CD56, CD16, CD49a and CD62L expression, low levels of CD57, and low levels of immune checkpoints such as LAG3 and CD200R. The new analyses showed that cryopreserved GDA-201 exhibited high viability (>90%) and high purity up to 12 months post-manufacturing and preserved the ability to proliferate post-thaw. GDA-201 maintained high levels of expression of CD16, which mediates antibody-dependent cellular toxicity, and CD62L, which is a homing and retention marker. GDA-201 also demonstrated high potency, based on the intracellular secretion of TNF-alpha & IFN-gamma and extracellular degranulation marker CD107a.

In addition, external investigator Veronika Bachanova, M.D., Ph.D., Professor at the University of Minnesota Medical School, gave an oral presentation highlighting novel observations of "on treatment" tumor biopsies from eight patients treated with GDA-201 in a Phase 1 study. The microscopic spatial analysis demonstrated that while GDA-201 cells were virtually undetectable in tumors after 14 days, T cells were observed in 50-95% of tumor site cellularity. Most biopsies obtained as early as three to seven days post-infusion showed strong indications of widespread tumor death. These observations suggest that GDA-201 infusions trigger profound immune microenvironment changes, supporting the influx of host T cells early post-GDA-201 infusion. These findings further suggest the engagement of the adaptive immune system and effective tumor elimination.
Corporate Developments

On March 20, the company announced that Shawn Cline Tomasello was elected Chairwoman of the Board of Directors, succeeding Chairman Robert I. Blum, who resigned. Ms. Tomasello joined the Gamida Cell Board of Directors in June 2019. She has extensive experience in leading successful commercial activities at several pharmaceutical companies and providing key strategic guidance on company boards. Dr. Anat Cohen-Dayag and Dr. Naama Halevi Davidov also resigned from the company’s Board of Directors.
In December, the company and its wholly owned subsidiary, as borrower, closed on a senior secured convertible term loan of $25 million with certain funds managed by Highbridge Capital Management, LLC. The loan has a maturity date of December 12, 2024.
Full Year 2022 Financial Results

Research and development expenses, net were $42.7 million in 2022, compared to $50.2 million in 2021. The decrease was primarily due to a $9.6 million decrease in clinical and operational activities relating to the conclusion of our Phase 3 study of omidubicel, offset by an increase of $1.1 million in T&E and other expenses as well as a $1.0 million decrease in Israeli Innovation Authority grants.
Commercial expenses in 2022 were $12.9 million, compared to $20.0 million in 2021. The decrease was primarily due to a $8.2 million decrease in commercial launch readiness expenses, offset by an increase of $1.1 million in headcount related expenses.
General and administrative expenses were $19.4 million in 2022, compared to $17.0 million in 2021. The increase was mainly driven by an increase of $1.4 million attributed to our corporate headquarters and headcount-related expenses as well as a $1.0 million increase in professional services expenses.
Financial expenses, net, were $4.4 million for 2022, compared to $2.6 million for 2021. The increase was primarily due to expenses relating to the closing on a senior secured convertible term loan of $25 million with certain funds managed by Highbridge Capital Management, LLC.
Net loss for 2022 was $79.4 million, compared to $89.8 million in 2021.
2023 Financial Guidance

Gamida Cell expects its current cash and cash equivalents will support the company’s ongoing operating activities through the third quarter of 2023. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding and any business development activities that may be undertaken.

Conference Call Information

Gamida Cell will host a conference call today, March 27, 2023, at 8:00 a.m. ET to discuss these financial results and company updates. To access the conference call, please register here and be advised to do so at least 10 minutes prior to joining the call. A live conference call webcast can be accessed in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. A webcast replay will be available approximately two hours after the event for approximately 30 days.

About Omidubicel

Omidubicel is an advanced cell therapy candidate for allogeneic hematopoietic stem cell (bone marrow) transplant that, if approved, has the potential to expand access and improve outcomes for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment compared to standard umbilical cord blood in an international, multicenter, randomized Phase 3 study (NCT02730299) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by a significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the U.S. and E.U. Omidubicel has a PDUFA target action date of May 1, 2023.

Omidubicel is an investigational stem cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit View Source

About GDA-201

GDA-201 is an intrinsic NK cell therapy candidate being investigated for the treatment of hematologic malignancies. Preclinical studies have shown that GDA-201 may address key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, these data suggest GDA-201 may improve antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells. A multicenter Phase 1/2 study of GDA-201 for the treatment of non-Hodgkin lymphoma is ongoing.

GDA-201 is an investigational cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority.

On March 27, 2023 Avacta Group plc, a life sciences company developing innovative, targeted oncology drugs and powerful diagnostics, reported that it will present a poster entitled ‘AVA3996, a novel pre|CISION medicine, targeted to the tumor microenvironment via Fibroblast Activation Protein-alpha (FAP-a) mediated cleavage’, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2023 Annual Meeting, taking place at Orange County Convention Center, Orlando, Florida on 16 April (Press release, Avacta, MAR 27, 2023, View Source [SID1234629400]).

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The poster describes the data and pre-clinical rationale for the further development and disease positioning of the Company’s second pre|CISION candidate, AVA3996, a tumour microenvironment activated proteasome inhibitor. AVA3996 utilises Avacta’s pre|CISION platform to improve the therapeutic index and therefore utility of proteasome inhibitors in tumours with high FAP levels, including both solid and haematological tumours. AVA3996 has the potential to deliver efficacious levels of the proteasome inhibitor warhead directly to the tumour microenvironment while reducing systemic exposure and hence associated toxicities, such as peripheral neuropathy.

Attending the conference from Avacta will be Fiona McLaughlin – Chief Scientific Officer, Neil Bell – Chief Development Officer, David Jones – VP Biology and Francis Wilson – VP Chemistry.

Poster Presentation Title

AVA3996, a novel pre|CISION medicine, targeted to the tumor microenvironment via fibroblast activation protein-alpha (FAP-a) mediated cleavage

Session Category

Experimental and Molecular Therapeutics

Session Title

Targeting the Tumour Microenvironment

Session Date and Time

April 16, 2023 1:30 PM – 5:30 PM ET

Location

Orange County Convention Center, Section 20

Poster Board Number

17

Abstract Presentation Number

583

The abstract is available via the AACR (Free AACR Whitepaper) annual meeting website, here: View Source!/10828/presentation/2606

Copies of the poster will be available on Avacta’s website following the conference at: View Source

Alastair Smith, Avacta Chief Executive Officer, will provide a video presentation overview for investors examining the data presented in the poster. This will be available on 17th April at View Source

On March 27, 2023 OncoVerity, a pioneer in applying advanced multiomic, data driven patient stratification to the development of human therapeutics in oncology, reported that it has secured worldwide licensing rights to cusatuzumab from argenx and received $30M in funding from argenx and a joint venture of UCHealth and University License Equity Holdings, Inc. on the University of Colorado Anschutz Medical Campus for its continued development (Press release, OncoVerity, MAR 27, 2023, View Source [SID1234629399]). Cusatuzumab is a first-in-class anti-CD70 antibody that has generated promising results in early acute myeloid leukemia (AML) human studies. OncoVerity will optimize cusatuzumab’s continued development in AML and explore its application in other cancers.

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OncoVerity is also announcing the appointment of Max Colao, MBA as Chief Executive Officer, and Dr. Clay Smith as Chief Medical Officer.

"We are thrilled to welcome Max and Clay to the OncoVerity leadership team. Together with Max’s extensive commercial leadership experience and Clay’s proven track record in research and development, we are confident that will drive growth and expansion as we enter the next phase of development," said Arjen Lemmen, Vice President, Corporate Development and Strategy at argenx, and OncoVerity Board Member.

Mr. Colao has over 30 years of biotechnology leadership experience. In his most recent position, he served as the Chief Commercial Officer of Aurinia, where he built out the commercial team through launch. Prior to that, Mr. Colao held senior roles of increasing responsibility at Alexion, where he led the US commercial operations and built out the new business units in launching several rare disease therapies. He also spent over 20 years at Amgen, where he launched multiple therapies in the areas of hematology, oncology, and autoimmune disorders supporting Amgen’s growth from small to large biotech.

Dr. Smith joins OncoVerity with more than 30 years of experience in oncology research, clinical care and clinical trials. He was previously Professor and Associate Chief of the Hematology Division at the University of Colorado. He was also the Director at UCHealth Blood Disorders and Cell Therapies Center. With the support of the University of Colorado Anschutz Medical Campus and UCHealth, Dr. Smith and his team pioneered the clinical and single cell multiomics infrastructure necessary to support, de-risk, and accelerate the development of this innovative therapy. Throughout his career, Dr. Smith has published over 150 scientific articles.

"I am excited to lead OncoVerity to its next phase of development and believe that the licensing of cusatuzumab and new funding demonstrate our commitment to advance novel cancer therapies in areas of high unmet need," said Max Colao. "OncoVerity’s differentiated approach and talented team put us in a strong position to answer unresolved disease and therapeutic questions in oncology. I’m grateful for the contributions of the University of Colorado and argenx in establishing OncoVerity and look forward to making significant progress on behalf of cancer patients around the world."

"OncoVerity is an example of a unique partnership that bridges the best in research, healthcare delivery, and the use of advanced multiomic data science-driven drug development to bring cutting edge treatments to patients in the areas of greatest unmet medical need," said Kimberly Muller, Executive Director, CU Innovations, Anschutz Medical Campus, General Partner, CU Healthcare Innovation Fund, and Board Member of OncoVerity.

"This is a very exciting time to be joining OncoVerity as we start this journey to advance cancer research and develop targeted therapies that can make a real difference in the lives of patients," said Dr. Clay Smith. "I am particularly enthusiastic about the potential for our work on cusatuzumab for the treatment of AML, a disease where despite many advances in the treatment paradigm there remains a significant opportunity to develop effective therapies that meaningfully prolong survival."

About Cusatuzumab

Cusatuzumab is a monoclonal antibody targeting CD70, an immune checkpoint target involved in hematological malignancies, several solid tumors and severe autoimmune diseases. Cusatuzumab is designed to: block CD70, kill cancer cells expressing CD70 through complement dependent cytotoxicity, enhanced antibody-dependent cell-mediated phagocytosis and enhanced antibody-dependent cell-mediated cytotoxicity, and restore immune surveillance against solid tumors (Silence K. et al. mAbs 2014; 6 (2):523-532). Cusatuzumab is currently being evaluated in patients with hematologic malignancies, including a Phase 2 trial in combination with azacitidine and a Phase 1b trial in combination with azacitidine and venetoclax in patients with newly diagnosed AML.

About Acute Myeloid Leukemia

Despite the advancements in medical research, there is still a significant lack of effective therapies to treat Acute Myeloid Leukemia (AML). The five-year survival rate for AML patients remains the lowest of all blood malignancies (5-20%), and relapse is common. AML is a heterogeneous disease, which means that it can differ in its genetic and molecular characteristics from one patient to another. This makes it difficult to develop effective treatments that can target all types of AML. Innovative therapies and targeted treatment approaches that can improve the survival rates, reduce toxicity, and target specific subtypes of AML are urgently needed to address this unmet need.

On March 27, 2023 CANbridge Pharmaceuticals, Inc. (1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, reported that the CAN008 (asunercept) Phase 2 trial for the treatment of glioblastoma multiforme (GBM) in China is fully enrolled, with a total of 117 patients (Press release, CANbridge Life Sciences, MAR 27, 2023, View Source [SID1234629398]). The multi-center, randomized, double-blind, placebo-controlled study is evaluating the efficacy and safety of CAN008 plus temozolomide (TMZ) during and after radiation therapy in newly diagnosed patients and is a potentially registrational trial. Five-year follow-up data from the CAN008 Phase 1 trial showed 67% overall survival in the high-dose group and a median 17.95 months of progression-free survival in newly diagnosed GBM patients. The data were presented at the ESMO (Free ESMO Whitepaper) Sarcoma and Rare Cancers Annual Meeting in March. Interim data from the CAN008 Phase 2 GBM China trial is expected in mid-2023.

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"Treatment with CAN008 significantly improved progression-free survival in the Phase 2 clinical trial of recurrent glioblastoma conducted by Apogenix in Germany," said Wenbin Li, Director of Comprehensive Treatment Ward of Neuro-Oncology, Beijing Tiantan Hospital, Capital Medical University, principal investigator of the study. "The recently published long-term follow up data from newly diagnosed GBM patients in the CAN008 Phase 1 clinical trial is also encouraging, with 4 of the 7 patients in the 400mg/week dose group still alive at 5 years. We are pleased to have completed enrollment in the Phase 2 trial of CAN008, which we believe is a promising treatment that could provide new hope to patients. Thank you to all of the researchers and staff who helped ensure that patients got their medications during the difficult COVID-19 prevention and control period and to the patients, who put their trust in us."

"We are delighted to have reached full enrollment in our Phase 2 glioblastoma trial for our first-in-class candidate, CAN008, in China, " said James Xue, Ph.D., founder, chairman and CEO of CANbridge Pharmaceuticals Inc. "In addition, we are greatly encouraged by the five-year overall survival data of 67% in the high-dose group from our Phase 1 trial and look forward to the furthering the clinical development of CAN008 for patients in China."

About CAN008

CAN008 (asunercept) is a CD95-Fc fusion protein that binds to the CD95 ligand to block the interaction between the ligand and the CD95 receptor. CAN008 has a unique dual mechanism of action. It inhibits both the invasive growth and migration of tumor cells, which could reduce the T-cell apoptosis, thereby enhancing immune recognition of the cancer. Earlier asunercept glioblastoma multiforme (GBM) clinical trial data showed favorable safety and tolerability, prolonged survival and improved quality-of-life.

Asunercept has been granted US FDA Orphan Drug Designation and Orphan Medicinal Product Designation by the European Medicines Agency (EMA) for GBM. It has also been accepted into the EMA’s PRIME (Priority Medicines) program, which provides support to medicines that could address unmet medical needs. In China, CAN008 has received a Class 1 New Drug Designation by the National Medical Products Administration. CANbridge holds the rights to develop CAN008 for any indication in Greater China and is currently conducting a CAN008 Phase 2 trial in GBM in China.