On March 14, 2022 Netris Pharma reported The selected companies will receive grants and/or equity investments, depending on their needs, up to a maximum of EUR 17.5 million (Press release, Netris Pharma, MAR 14, 2022, View Source [SID1234611186]). They were selected following a rigorous process, introduced under Horizon Europe, involving an ideas screening stage, and full applications assessed by external experts and an interview with a jury of experienced investors and entrepreneurs.

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The selected companies have a wide geographical spread spanning 21 countries, including seven from Horizon Europe "widening countries" that have lower levels of research and innovation performance. The selection also confirms the high demand for EIC equity investments with 65 of the 99 companies requesting equity investments of up to EUR 414 million (out of the total EUR 627 million). In most cases, the companies will receive the grant financing within the coming months, while the equity investment is likely to take longer as the arrangements for implementing EIC equity need to be re-established under Horizon Europe. This delay will also affect companies selected in the previous funding round.

On March 14, 2022 Heat Biologics, Inc. ("Heat") (NYSE American: HTBX), a clinical-stage biopharmaceutical company focused on developing first-in-class therapies to modulate the immune system, reported that provided strategic, financial, and operational updates for the year ended December 31, 2021 (Press release, Heat Biologics, MAR 14, 2022, View Source [SID1234610043]).

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Jeff Wolf, Chief Executive Officer of Heat, commented, "2021 was a transformative year for Heat as we progress our strategic evolution to become a fully-integrated biopharmaceutical company. On the clinical front, we presented favorable survival data at The American Association of Cancer Research (AACR) (Free AACR Whitepaper) of HS-110 in combination with checkpoint inhibitors for the treatment of non-small cell lung cancer (NSCLC). We recently submitted meeting requests to the FDA for separate Type B ("End of Phase 2 Meeting") and Type C (Phase 3 "CMC Readiness Meeting") meetings to receive feedback on our proposed trial design and regulatory path forward. We anticipate these meetings will be scheduled for the second quarter of 2022. Following these meetings, we should be in a better position to advance potential strategic discussions regarding this program."

"In September, based on our positive review of the Phase 1 data generated to date, we announced additional dose levels for our Phase 1 clinical trial evaluating the safety of PTX-35 monotherapy in patients with solid tumors and look forward to reporting the results of this trial later this year. We also recently announced new data demonstrating a single dose of the preclinical version of PTX-35 (mPTX-35), was able to expand regulatory T cells (Tregs) and significantly improve disease and graft survival outcome in a model of pancreatic islet allotransplantation for the treatment of diabetes."

"We continued to make great strides towards becoming a fully integrated biotech capable of driving innovations from discovery through delivery. With the formation of Skunkworx Bio, we have ramped up our in-house discovery of biologics for our preclinical and clinical development efforts."

"Our biomanufacturing and analytical team at Scorpion Biological Services broke ground on a new facility in San Antonio, Texas, designed to decrease our dependence on third-party CDMOs, reducing costs and accelerating timelines. In addition to supporting our own internal operations, we intend to offer manufacturing services to other biopharma companies on a fee-for-service basis. These efforts reinforce our goal of maximizing efficiency of drug development to expedite the delivery of novel immune activating therapies."

"At the World Vaccine Conference, we announced our new biosecurity/biodefense initiative led by RapidVax, a novel vaccine platform designed to enable an accelerated response and long-lasting immunological memory against a wide variety of infectious agents. RapidVax is designed to utilize a common unprogrammed vaccine base that can be manufactured in bulk, stockpiled, and rapidly customized upon identification of a biological threat to enable an accelerated time to clinic that can harness shared development, clinical safety, and manufacturing synergies. Our gp96-based cellular vaccines have previously demonstrated prophylactic protection in animal models against a range of diseases, including malaria, HIV/SIV, Zika and SARS-CoV-2 in multiple DOD and NIH-funded studies. Since launching this program, we have filed patents to protect our proprietary technology and have established our new Biothreat Advisory Board, which includes highly renowned individuals in the fields of biosecurity and biodefense, to guide the development of our programs."

"In addition to our internal biodefense initiatives, we announced a definitive merger agreement to acquire Elusys Therapeutics, a commercial-stage biodefense company and the manufacturer of ANTHIM (obiltoxaximab), which is approved for the treatment of inhalation anthrax in the U.S., Canada, Europe. Since announcing this acquisition, Elusys completed the first phase of a contract for $50 million with an option to procure up to an additional $31 million of ANTHIM by the first half of 2023. Given the growing global uncertainty, we believe the risks of anthrax and other biological threats are more present than ever. This acquisition is aligned with our strategy to take a leading role in the biodefense market."

2021 Financial Results

Recognized $2.1 million of grant and contract revenue, primarily for qualified expenditures under the CPRIT grant for the year ended December 31, 2021 compared to $2.9 million for the year ended December 31, 2020. The decrease in grant revenue in the current-year period primarily reflects the expected timing of completion of deliveries under the current phase of the contracts. As of December 31, 2021, we had a grant receivable balance of $1.3 million for CPRIT proceeds not yet received but for which the costs had been incurred or the conditions of the award had been met. We continue our efforts to secure future non-dilutive grant funding to subsidize ongoing research and development costs.
Research and development expense was $18.8 million and $12.9 million for the years ended December 31, 2021 and 2020, respectively. The increase was primarily due to manufacturing and patient costs for PTX-35, consulting expenses in our clinical and CMC groups, and a $2.4 million impairment of IPR&D.
General and administrative expense was $16.8 million and $14.9 million for the years ended December 31, 2021 and 2020. The increase was primarily due to the increase in salaries, D&O insurance expense, and legal fees.
Net loss attributable to Heat Biologics was approximately $35.1 million, or ($1.41) per basic and diluted share for the year ended December 31, 2021 compared to a net loss of approximately of $26.0 million, or ($1.63) per basic and diluted share for the year ended December 31, 2021.
As of December 31, 2021, the Company had approximately $96.4 million in cash, cash equivalents and short term investments.

On March 14, 2022 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a synthetic lethality focused precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported that it has entered into additional clinical trial collaboration and supply agreements with Pfizer Inc. (NYSE: PFE) to support further evaluation of darovasertib and crizotinib combination therapy in a Phase 2 potential registration-enabling clinical trial in patients with metastatic uveal melanoma (MUM) and in a Phase 1 clinical trial in patients with cMET-driven tumors, such as hepatocellular carcinoma (HCC) and/or non-small cell lung cancer (NSCLC) (Press release, Ideaya Biosciences, MAR 14, 2022, View Source [SID1234610059]).

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IDEAYA is currently evaluating the combination of darovasertib, a PKC inhibitor, and crizotinib, a cMET inhibitor, in patients with metastatic uveal melanoma (MUM) and in patients with GNAQ or GNA11 mutant skin melanoma in an ongoing Phase 1/2 clinical trial, pursuant to a clinical trial collaboration and supply agreement with Pfizer.

"We are pleased to have Pfizer’s support in connection with a potential registrational clinical trial as our clinical data on the darovasertib / crizotinib combination in MUM continues to mature. Our preliminary clinical data on the darovasertib and crizotinib combination in MUM, reported in December 2021, showed robust clinical activity with a manageable side effect profile. We have an opportunity to positively impact the treatment of patients in this high unmet medical need population," said Dr. Matthew Maurer, M.D., Vice President and Head of Clinical Oncology and Medical Affairs at IDEAYA Biosciences.

"The clinical efficacy of the combination therapy in MUM patients provides proof of concept for potential expansion opportunities in other cMET-driven tumors. We believe that the darovasertib and crizotinib combination therapy can potentially improve on current standard of care treatment paradigms, for example in HCC, where response rates are modest," added Michael White, Ph.D., Senior Vice President and Chief Scientific Officer at IDEAYA Biosciences.

IDEAYA is targeting a clinical data update for its Phase 1/2 clinical trial evaluating the darovasertib and crizotinib combination in MUM in mid-year 2022, including tolerability and clinical efficacy. IDEAYA is also planning to seek FDA regulatory guidance for potential registration-enabling trial design to evaluate darovasertib and crizotinib combination in MUM in mid-year 2022. The timing of the clinical data and FDA regulatory guidance may be influenced by data maturity, including for example, appropriate interim assessments of supportive median duration of response (DoR) and/or median progression free survival (mPFS).

On March 13, 2022 InnoCare Pharma (HKEX: 09969), a leading biopharmaceutical company, reported that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) accepted a supplemental New Drug Application (sNDA) for Bruton’s tyrosine kinase (BTK) inhibitor orelabrutinib for the treatment of patients with relapsed or refractory Waldenström’s Macroglobulinemia (R/R WM) b(Press release, InnoCare Pharma, MAR 13, 2022, View Source [SID1234610009]).

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"According to the evaluation of the Independent Review Committee (IRC) and the principal investigators, the treatment of R/R WM patients with orelabrutinib at a dose of 150mg once a day reached the primary end point, showing encouraging efficacy, which is expected to improve with the extension of treatment time," said Professor Daobin Zhou, Director of Hematology Department of Peking Union Medical College Hospital. "Meanwhile, orelabrutinib is well tolerated with safety profile in the treatment of WM patients. The incidence of adverse events, especially off-target related serious adverse events, is significantly lower in the clinical trial."

Dr. Jasmine Cui, Co-founder, Chairwoman and CEO of InnoCare said, "WM poses a threat to patients’ lives. We are encouraged that NMPA has accepted the supplemental New Drug Application for orelabrutinib in the treatment of R/R WM. This sNDA includes 47 patients from 16 clinical centers in China. We hope that orelabrutinib will bring a novel treatment option to R/R WM patients."

On December 25, 2020, orelabrutinib received approval from NMPA in two indications: the treatment of patients with relapsed/refractory chronic lymphocytic leukemia (CLL) /small lymphocytic lymphoma (SLL), and the treatment of patients with relapsed/refractory mantle cell lymphoma (MCL).

About Waldenström’s Macroglobulinemia

Waldenström’s macroglobulinemia is a relatively slow-growing lymphoma characterized by the invasion of bone marrow by lymphoplasmacytic cells secreting monoclonal immunoglobulin M (IgM). The disease usually affects older adults and is primarily found in the bone marrow, although it may also impact lymph nodes and the spleen.

About Orelabrutinib

Orelabrutinib is a highly selective BTK inhibitor developed by InnoCare for the treatment of cancers and autoimmune diseases.

On Dec. 25 2020, orelabrutinib received approval from the China National Medical Products Administration (NMPA) in two indications: the treatment of patients with relapsed/refractory chronic lymphocytic leukemia (CLL) /small lymphocytic lymphoma (SLL), and the treatment of patients with relapsed/refractory mantle cell lymphoma (MCL). At the end of 2021, orelabrutinib was included into National Reimbursement Drug list to benefit more lymphoma patients.

In addition to the approved indications, multi-center, multi-indication clinical trials are underway in the US and China with orelabrutinib as monotherapy or in combination therapies.

Orelabrutinib was granted as Breakthrough Therapy Designation for the treatment of r/r MCL by U.S. Food and Drug Administration (FDA).

Attributed to its excellent selectivity and clinical safety profiles, orelabrutinib is also evaluated in the global phase II studies for the treatment of Multiple Sclerosis (MS), phase II clinical trials for the treatment of Systemic Lupus Erythematosus (SLE) and Primary Immune Thrombocytopenia (ITP) in China.

On March 13, 2022 Everest Medicines (HKEX 1952.HK), a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products to address critical unmet needs in Asia Pacific markets, reported that it has entered into a license agreement with Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) to develop and commercialize NEFECON for the treatment of primary IgA nephropathy (IgAN) in South Korea, expanding its license in addition to rights held in Greater China and Singapore (Press release, Everest Medicines, MAR 13, 2022, View Source [SID1234610010]). The deal signals the Company’s latest efforts to further enhance its international commercial footprint.

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"We’re thrilled to strengthen our partnership with Calliditas by extending our license agreement to include South Korea and working towards our shared vision of developing and commercializing NEFECON as a potential novel therapy for the treatment of IgAN globally," said Kerry Blanchard, MD, PhD, Chief Executive Officer of Everest Medicines. "While primary IgAN is more common in Asia than elsewhere in the world, there are no established treatments for patients living with the chronic condition, underscoring the significant and urgent unmet need for innovative medicines."

"South Korea is the third largest pharmaceutical market in the Asia Pacific region —after China and Japan—and represents tremendous opportunity," said Kevin Yong Guo, Chief Commercial Officer of Everest Medicines. "South Korea’s sophisticated healthcare model enables innovative medicines such as NEFECON to have a higher potential likelihood of healthcare coverage once approved. We look forward to working closely with all contributing parties to bring NEFECON to patients living with IgAN in South Korea as quickly as possible."

"We continue to have a fruitful and positive collaboration with Everest and are delighted that we have come to an agreement also around South Korea," said CEO Renée Aguiar-Lucander of Calliditas.

In the coming months, Everest Medicines will collaborate with the Ministry of Food and Drug Safety of South Korea to prepare regulatory filings and a New Drug Application (NDA) for NEFECON for the treatment of primary IgAN.

Under the terms of the agreement, Calliditas receives an initial upfront payment of USD$3 million at signing of the agreement.

About NEFECON

NEFECON (approved in the United States under accelerated approval under another trade name) is an oral, delayed release formulation of budesonide, a corticosteroid with potent glucocorticoid activity and weak mineralocorticoid activity that undergoes substantial first pass metabolism. NEFECON was designed as a 4 mg delayed release capsule and is enteric coated so that it would remain intact until it reaches the ileum. Each capsule contains coated beads of budesonide that target mucosal B-cells present in the ileum, including the Peyer’s patches, which are responsible for the production of galactose-deficient IgA1 antibodies (Gd-Ag1) causing IgA nephropathy. It is unclear to what extent NEFECON’s efficacy is mediated via local effects in the ileum vs systemic effects. In June 2019, Everest Medicines entered into an exclusive, royalty-bearing license agreement with Calliditas, which gives Everest Medicines exclusive rights to develop and commercialize NEFECON in Mainland China, Hong Kong, Macau, Taiwan and Singapore. The agreement was extended in March 2022 to include South Korea as part of Everest Medicine’s territories.

About Primary Immunoglobulin A Nephropathy

Primary immunoglobulin A nephropathy (IgA nephropathy or IgAN or Berger’s Disease) is a progressive, chronic autoimmune disease that attacks the kidneys and occurs when galactose-deficient IgA1 are recognized by autoantibodies, creating IgA1 immune complexes that become deposited in the glomerular mesangium of the kidney.1,2 This deposition in the kidney can lead to progressive kidney damage and potentially a clinical course resulting in end- stage renal disease. IgAN most often develops between late teens and late 30s.2,3