On January 31, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, reported that key program and business updates (Press release, FierceBiotech, JAN 31, 2022, View Source [SID1234607518]).

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Initiating rolling BLA submission for omidubicel. Following the recent receipt of positive Type B meeting correspondence from the U.S. Food and Drug Administration (FDA), Gamida Cell will initiate a rolling Biologics License Application (BLA) submission for omidubicel, a potentially life-saving treatment for patients with blood cancers in need of a stem cell transplant, in the first quarter of 2022 and plans to complete the full BLA submission in the first half of 2022.
Evaluating strategic alternatives for omidubicel. In parallel with the planned BLA submission, the company will be assessing alternatives for the commercialization of omidubicel, including potential U.S. or global partnerships.
Reducing operating expenses. With the objective of extending its cash runway into mid-2023, consistent with the timeline for potential U.S. approval of omidubicel, the company is reducing operating expenses primarily by implementing a workforce reduction of approximately 10% and delaying other hiring and planned spending in 2022.
Readying to advance GDA-201. The company is addressing comments received from FDA in connection with the clinical hold placed on the IND submission for GDA-201, its lead NAM-enabled innate NK cell immunotherapy. Gamida Cell expects to initiate a company-sponsored Phase 1/2 clinical study in patients with follicular and diffuse large B-cell lymphomas in 2022.
Advancing genetically modified NK cell immunotherapy programs. The company continues to advance itsNAM-enabled genetically modified NK pipeline, which utilizes CAR, membrane bound- and CRISPR-mediated strategies to increase targeting, potency and persistence against hematologic malignancies and solid tumors. The company plans to execute preclinical proof of concept studies for these genetically modified NK therapeutic targets and to select pipeline candidates for IND enabling studies by the end of 2022.
"We are pleased that productive interactions with the FDA enable us to initiate a rolling submission of the BLA for omidubicel this quarter and to complete the full BLA submission during the first half of this year," said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. "As we advance omidubicel towards potential approval, we will be assessing strategic alternatives for the best way to bring this important therapy to patients, including potential U.S. or global commercialization partnerships. With the strategic steps we are taking, we believe Gamida Cell will be in a stronger position to support omidubicel through the regulatory approval process while we also continue to advance our NK cell pipeline programs, all as intended to serve our goal of providing access to life-saving cell therapies to patients in need."

2022 Financial Guidance

Gamida Cell ended 2021 with approximately $96.1 million in cash and cash equivalents (unaudited). The company expects cash used for ongoing operating activities in 2022 to range from $60 million to $70 million in cash and cash equivalents based on its current operating plans. The company anticipates that its current cash will support the company’s ongoing operating activities into mid-2023, excluding any additional financing or business development activities that may be undertaken. Gamida Cell plans to report its fourth quarter and full-year 2021 financial results on March 16, 2022, at which time the company will provide an update on its 2022 milestones and more detailed financial guidance.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with blood cancers. Omidubicel is the first bone marrow transplant graft to receive Breakthrough Therapy Designation from the U.S. FDA and has also received Orphan Drug Designation in the U.S. and EU. Gamida Cell has completed an international, multi-center, randomized Phase 3 study (NCT0273029) evaluating the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing allogeneic bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. That study achieved its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in a patient’s recovery from a bone marrow transplant. The Phase 3 study also achieved its secondary endpoints of reduced time to platelet engraftment, reduced infections and shorter days of hospitalization. For more information about omidubicel, please visit View Source

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About GDA-201

Gamida Cell applied the capabilities of its nicotinamide (NAM)-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, GDA-201 improves antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells. For more information about GDA-201, please visit View Source

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

On January 31, 2022 Aravive, Inc. (Nasdaq: ARAV), a late clinical-stage oncology company developing targeted therapeutics to treat metastatic disease, reported that the Company has dosed the first patient in the Phase 2 portion of the Phase 1b/2 study of batiraxcept for the treatment of clear cell renal cell carcinoma (ccRCC) (Press release, Aravive, JAN 31, 2022, View Source [SID1234607516]).

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"Safety and preliminary activity data from the Phase 1b study of batiraxcept in combination with cabozantinib in patients with 2L+ ccRCC gives us confidence to initiate the Phase 2 portion of the trial and expand the study to additional cohorts," said Gail McIntyre, Ph.D., DABT, Chief Executive Officer of Aravive. "I’m grateful to our team for their diligence and dedication to expedite the ccRCC program. The data reported to date shows clinically relevant benefit of adding batiraxcept to the current standard of care in ccRCC without adding to the toxicity profile. We will continue to update the ongoing Phase 1b portion of the study as data mature and anticipate providing clinical activity and safety updates of the P2 portion of the study throughout 2022."

The Phase 2 portion of the Phase 1b/2 clinical trial of batiraxcept in ccRCC is an open-label study in which 55 patients are anticipated to enroll across three parts. Part A is expected to enroll approximately 25 patients and will investigate batiraxcept 15 mg/kg in combination with cabozantinib in 2L+ ccRCC patients. Part B is expected to enroll approximately 20 patients and evaluate batiraxcept 15 mg/kg in combination with standard of care nivolumab and cabozantinib in first-line ccRCC patients. Part C is expected to evaluate batiraxcept 15 mg/kg monotherapy in approximately 10 patients with ccRCC who are not eligible for curative intent therapies. The primary endpoint of each part of the Phase 2 portion of the trial is objective response rate ("ORR") and key secondary endpoints include duration of response ("DOR"), progression free survival ("PFS"), and overall survival ("OS"). Additional information on the trials: NCT04300140.

About Batiraxcept (AVB-500)
Batiraxcept is a therapeutic recombinant fusion protein that has been shown to neutralize GAS6 activity by binding to GAS6 with very high affinity in preclinical models. In doing so, batiraxcept selectively inhibits the GAS6-AXL signaling pathway, which is upregulated in multiple cancer types including ovarian, renal and pancreatic. In preclinical studies, GAS6-AXL inhibition has shown anti-tumor activity in combination with a variety of anticancer therapies, including radiation therapy, immuno-oncology agents, and chemotherapeutic drugs that affect DNA replication and repair. Increased expression of AXL and GAS6 in tumors has been correlated with poor prognosis and decreased survival and has been implicated in therapeutic resistance to conventional chemotherapeutics and targeted therapies. Batiraxcept is currently being evaluated in multiple clinical trials and has been granted Fast Track designation by the U.S. Food and Drug Administration and orphan drug designation by the European Commission in platinum resistant recurrent ovarian cancer.

On January 31, 2022 Semmelweis University and AstraZeneca it’s Hungarian subsidiary reported that they have signed a strategic cooperation agreement to further investigate the links between certain common diseases, such as diabetes, chronic kidney disease (CKD) and heart failure, to strengthen and expand joint research and development activities, and to implement joint programmes to help treat, educate and diagnose patients (Press release, Semmelweis University, JAN 31, 2022, View Source [SID1234607513]). A framework agreement for clinical trials was also signed at the event, which will allow for faster and more efficient trial start-ups.

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„Today, an agreement is being signed between AstraZeneca, one of the world’s leading biotechnology pharmaceutical companies, which also has a dominant market position in Hungary, and Semmelweis University, the leading medical and health sciences higher education institution of the Central European region. What we certainly have in common is a commitment to research and development and a dedication to providing patients with better and more innovative therapies," highlighted Dr. Béla Merkely, rector of Semmelweis University on signing the strategic partnership cooperation agreement. „This cooperation will allow us to increase our joint research and development activities in the fields of oncology, cardiology, diabetology, nephrology and other areas of internal medicine. As a first step, a complex program on chronic kidney disease (CKD) could be implemented in collaboration with our Department of Family Medicine," the rector explained, adding that CKD is an under-diagnosed condition which can increase the risk of cardiovascular damage many times over.

Semmelweis University and AstraZeneca also signed a framework agreement for clinical trials. „This will enable the contracting of individual clinical trials, and thus the launching of trials and the involvement of patients, to be faster and more efficient. Currently, Semmelweis University takes part in two-thirds of AstraZeneca’s Hungarian trials, but our aim is to increase this to over 80 percent," Dr. Béla Merkely emphasized. The rector also pointed out that one of the university’s strategic objectives is to increase clinical research activity by building strategic partnerships with the pharmaceutical industry. This allows patients to have access to the most innovative therapeutic options as soon as possible, and increases scientific performance as well, which contributes to moving up the rankings.

Kuuno Vaher, AstraZeneca’s Cluster Country Director of Central Europe reminded that the company’s main therapeutic areas are oncology, cardiovascular diseases, renal and metabolic diseases, respiratory diseases and immunology. "We know a lot more about these diseases by now, but the implementation of these new information is becoming more challenging – more and more aspects need to be taken into account in treatment," Kuuno Vaher said. „This is why an important part of the company’s partnerships in different countries, including now Hungary, is to look at how to improve the organisation of healthcare and how to use digitalisation, to enable doctors making better healthcare decisions based on all the available data. Basically, there are four pillars of collaboration: diagnostics, screening, education and analyzing the accumulated data, to see if what we are doing is really improving patients’ lives. For example, it is not enough to know how many people are utilizing a certain medicine; we also need to see if an innovation is having a real impact on patients’ outcome," Kuuno Vaher pointed out.

Dr. Mátyás Faluvégi, Managing Director of AstraZeneca’s Research Division, explained that the framework agreement on clinical trials is based on the fact that Semmelweis University has an optimal background for conducting such trials. In the last three years, a total of 855 clinical trials have been conducted in Hungary, out of which AstraZeneca owned 49 projects, and the university was involved in 31. „A large number of cardiovascular and pulmonary trials are already taking place at Semmelweis, so our aim is to launch more oncology trials in the coming years," the managing director said. „By signing the agreement, we would like to accelerate the start-up of trials in the short term, and our long-term goal is to strengthen AstraZeneca’s presence not only at the university, but throughout Hungary," he added.

Dr. Péter Ferdinandy, Vice-Rector for Science and Innovation of Semmelweis University, thanked the Clinical Research Coordination Center led by Dr. János Filakovszky for the work done in the preparation of the agreement. He then expressed hope that the cooperation will further increase the number of clinical trials, which could be a first step towards the joint development of new therapies, as well as the creation of shared intellectual property and a broader boost to scientific cooperation.

The strategic cooperation agreement and the framework agreement on clinical trials were signed by Rector Béla Merkely and Chancellor Lívia Pavlik on behalf of Semmelweis University, while the former document was signed by Cluster Director Kuuno Vaher on behalf of AstraZeneca and the latter by Managing Director Mátyás Faluvégi.

On January 31, 2022 Tollys, a biopharmaceutical company developing the first anti-cancer immunotherapy based on a synthetic Toll-Like Receptor 3 (TLR3) specific agonist, reported that it has renewed and extended its research collaboration started in 2020 with a global pharmaceutical company, a leader in immuno-oncology (Press release, Tollys, JAN 31, 2022, View Source [SID1234607500]). The company also announces the acceleration and expansion of its R&D platform in the fields of TLR3 agonist candidates for intravenous administration and Antibody-Drug-Conjugates (ADC, or AOC for Antibody-Oligonucleotide-Conjugates).

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Based on the strong preclinical data collected to date with locally administered TL-532, Tollys is also accelerating and expanding its internal and collaborative R&D activities on TLR3 agonist candidates designed for intravenous administration and antibody-drug-conjugates. Tollys is thus running several parallel preclinical programs using different vectorization and targeting methods for its TLR3 agonist candidate(s). According to Tollys, TL-532 is the first chemically conjugable specific TLR3 agonist usable as a payload with antibodies and other carriers.

This acceleration in the development of candidates for intravenous administration is in line with the recommendations of the international board of the European oncology innovation acceleration program MATWIN which awarded Tollys TLR3 agonist the status of ‘best-in-class innovation of the year’ in May 2021.

"Our renewed and extended pharma collaboration is further confirmation of the potential of specific TLR3 agonists. We are also very excited to advance our R&D programs for the selection of candidates for intravenous administration; we estimate that a lot more patients could benefit from treatment with TLR3 agonists, if administered intravenously rather than locally," said Vincent Charlon, CEO of Tollys.

About TL-532
TL-532 is the first synthetic specific TLR3 agonist with a proprietary defined double-stranded RNA sequence. As such, TL-532 has the potential to be the best-in-class and first-to-market TLR3 agonist. TL-532 was shown to have a triple mechanism of action inducing 1) death by apoptosis selective to cancer cells-not in normal cells, leading to the in-situ release of tumor specific antigens, 2) activation of the myeloid dendritic cells of the immune system to mount a specific T-cell response against the tumor antigens and 3) switch of the tumor microenvironment, by producing cytokines and chemokines which prevent tumor development. The result is the immunogenic cell death of tumor cells, together with an autovaccination preventing the recurrence of cancer.

On January 31, 2022 NEC reported that Consolidated Financial Results for the Nine-month Period Ended
December 31, 2021 (April 1, 2021 – December 31, 2021) (Press release, NEC, JAN 31, 2022, View Source [SID1234607499])

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1. Consolidated Financial Results for the Nine-month Period Ended December 31, 2021

(1) Consolidated Operating Results
(2) Consolidated Financial Position

2. Dividends

3. Consolidated Financial Results Forecast for the Year Ending March 31, 2022 (April 1, 2021 – March 31, 2022)

This consolidated financial results falls outside the scope of quarterly review procedures to be performed by certified public accountants or an audit firm.

*Explanation concerning the appropriate use of the financial results forecast and other special matters
(Adjusted profit (loss))
"Adjusted operating profit (loss)" is an indicator for measuring underlying profitability in order to clarify the contribution of acquired companies to the NEC Group’s overall earnings. It is measured by deducting amortization of intangible assets recognized as a result of M&A and expenses for acquisition of companies (financial advisory fees and other fees) from operating profit (loss). Also, "Adjusted net profit (loss) attributable to owners of the parent" is an indicator for measuring underlying profitability attributable to owners of the parent. It is measured by deducting adjustment items of operating profit (loss) and corresponding amounts of tax and non-controlling interests from net profit (loss) attributable to owners of the parent.