On January 31, 2022 Inventiva (Euronext Paris and Nasdaq: IVA), a clinical-stage biopharmaceutical company focused on the development of oral small molecule therapies for the treatment of non-alcoholic steatohepatitis (NASH), mucopolysaccharidoses (MPS) and other diseases with significant unmet medical needs, reported the receipt of a €4 million milestone payment from AbbVie (Press release, Inventiva Pharma, JAN 31, 2022, View Source [SID1234607520]). It follows the inclusion of the first patient with psoriasis in the ongoing Phase IIb clinical trial with cedirogant (ABBV-157)1, an oral RORg inverse agonist jointly discovered by Inventiva and AbbVie for the treatment of autoimmune diseases.

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The Phase IIb clinical trial initiated by AbbVie with cedirogant is a multicenter, randomized, double-blind, placebo-controlled, dose-ranging study to evaluate the safety and efficacy of the drug candidate in adult patients with moderate to severe plaque psoriasis. The details of the clinical trial are available on clinicaltrials.gov2.

Frédéric Cren, Chairman, Chief Executive Officer and cofounder of Inventiva, stated: "This new milestone payment and the initiation of a Phase IIb trial in adult patients with psoriasis are excellent news for Inventiva. It is an important step in the development of cedirogant, after the compound showed promising activity as an oral psoriasis agent in a Phase Ib clinical trial led by AbbVie. We are extremely proud to collaborate with AbbVie, a worldwide leader in autoimmune diseases, and we believe cedirogant has the potential to become a new reference treatment in psoriasis and other autoimmune diseases."

In 2012, Inventiva and AbbVie signed a multi-year drug discovery collaboration agreement to identify potent RORg inverse agonists for the treatment of several auto-immune diseases. Through this collaboration, Inventiva leveraged its discovery expertise and technology platforms to develop drug candidates targeting the nuclear receptor RORg, a validated drug target for the treatment of cutaneous inflammatory disorders such as psoriasis.

This collaboration with AbbVie enables Inventiva to receive payments upon the achievement of clinical, regulatory and commercial milestones, as well as tiered royalties on product sales, from mid single-digit to low double-digit.

About psoriasis

Psoriasis is a common skin disease affecting more than 3% of the US adult population3. In moderate and severe cases, psoriatic lesions can be uncomfortable, itchy and disfiguring. Although the precise pathophysiology of psoriasis is unknown, an abnormal cutaneous immunologic/inflammatory response, associated with epidermal hyper proliferation and abnormal differentiation, seems to be involved.

Current treatment of psoriasis is directed toward the alteration of epidermal differentiation, reducing the inflammatory response and slowing the growth of involved skin cells. The extent and severity of the disease typically determine the therapeutic approach. In mild psoriasis, the most commonly used therapy is topical with the addition of phototherapy in refractory cases. In moderate to severe psoriasis, phototherapy or anoral systemic therapy are used.

On January 31, 2022 Immix Biopharma, Inc. (Nasdaq: IMMX) ("ImmixBio", "Company", "We" or "Us"), a biopharmaceutical company pioneering Tissue-Specific Therapeutics (TSTx)TM targeting oncology and immuno-dysregulated diseases, reported that it has initiated IMX-110 Good Manufacturing Practice ("GMP") scale-up manufacturing, potentially accelerating the timeline to clinical data from two ImmixBio clinical trials planned for 2022: first, planned monotherapy IMX-110 clinical trial in soft tissue sarcoma ("STS"); second, planned combination IMX-110 + BeiGene anti-PD-1 tislelizumab clinical trial in advanced solid tumors (Press release, Immix Biopharma, JAN 31, 2022, View Source [SID1234607519]).

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"Initiating GMP manufacturing 36 days after the closing of our IPO puts us on a path of potentially accelerating our clinical trials," said Ilya Rachman, MD PhD, CEO of ImmixBio. "We believe this step paves the way to execute our planned milestones at an accelerated pace."

About IMX-110

The U.S. Food and Drug Administration ("FDA") has approved orphan drug designation ("ODD") for IMX-110 for the treatment of soft tissue sarcoma. Additionally, the FDA has approved rare pediatric disease ("RPD") designation to IMX-110 for the treatment of a life-threatening pediatric cancer in children, rhabdomyosarcoma. RPD qualifies ImmixBio to receive fast track review and a priority review voucher (PRV) at the time of marketing approval of IMX-110. IMX-110 is currently being evaluated in a phase 1b/2a clinical trial in patients with advanced solid tumors. Learn more at www.immixbio.com/iMX-110

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.