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MHLW GRANTS ORPHAN DRUG DESIGNATION IN JAPAN TO NOVEL FIBROBLAST GROWTH FACTOR (FGF) RECEPTOR SELECTIVE TYROSINE KINASE INHIBITOR E7090 WITH PROSPECTIVE INDICATION FOR UNRESECTABLE BILIARY TRACT CANCER WITH FGFR2 GENE FUSION

On February 22, 2021 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that it has received orphan drug designation for a prospective indication for unresectable biliary tract cancer with FGFR2 gene fusion by the Ministry of Health, Labour and Welfare, Japan (MHLW) for its in-house discovered fibroblast growth factor (FGF) receptor (FGFR1, FGFR2, FGFR3) selective tyrosine kinase inhibitor E7090, which is currently under development as an orally available novel anti-cancer agent (Press release, Eisai, FEB 22, 2021, View Source [SID1234575351]).

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FGFRs with genetic aberrations are known to play an important role in the proliferation, survival and migration of cancer cells as well as tumor angiogenesis and drug resistance. These genetic aberrations in FGFRs have been observed in various types of cancers, therefore, there is growing interest in FGFRs as a promising target for cancer therapy. By selectively inhibiting FGFR1, 2 and 3, and blocking those signals, E7090 has the potential to become a new molecular targeted therapy for cancers with FGFR genetic aberrations.

In Japan, a Phase I clinical trial of E7090 was conducted, and E7090 has been designated as the target drug for the SAKIGAKE Designation System of the MHLW for the treatment of unresectable biliary tract cancer. Currently, a Phase II clinical trial (Study 201) of E7090 is underway in patients with cholangiocarcinoma with FGFR2 gene fusion in Japan and China.

Eisai positions oncology as a key therapeutic area and is aiming to discover innovative new medicines with the potential to cure cancer. Eisai is committed to exploring the potential clinical benefits of E7090 for cancer treatment, as it seeks to contribute further to addressing the diverse needs of, and increasing the benefits provided to, patients with cancer and their families.

Discovered in-house by Eisai’s Tsukuba Research Laboratories, E7090 is an orally available novel tyrosine kinase inhibitor that demonstrates selective inhibitory activity against fibroblast growth factor receptors (FGFR) FGFR1, FGFR2 and FGFR3. Distinct from prior known FGFR inhibitors, E7090 has a basic structure which lacks the dimethoxyphenyl moiety, and in a kinetic interaction analysis study, it was observed that E7090 demonstrates antitumor effects due to inhibition of kinase activity with a binding mode (Type V) that exhibits rapid and potent binding as well as high selectivity to FGFR.1

A Phase II clinical trial (Study 201) of E7090 is underway in Japan and China to evaluate efficacy and safety in patients with cholangiocarcinoma with FGFR2 gene fusion. A Phase I clinical trial of E7090 is also underway in Japan in patients with estrogen receptor-positive and HER2-negative breast cancer.

2. About Biliary Tract Cancer with FGFR2 Gene Fusion
The five-year survival rate for biliary tract cancer is approximately 20%, which makes it an intractable cancer with the second worst prognosis following pancreatic cancer.2 Drug therapy options are limited in comparison with other cancers, and as such it is a disease with significant unmet medical needs. The estimated number of patients with biliary tract cancer is approximately 32,000 in Japan.3,4,5 FGFR2 gene fusion is observed in approximately 14% of intrahepatic cholangiocarcinoma, which account for 15-30% of biliary tract cancers.6

3. Orphan Drug Designation System in Japan
The orphan drug designation system in Japan aims to support the development of drugs for diseases for which the number of patients is small and research and development is not progressing, despite high unmet medical need. As the requirement for designation based on Article 77-2 of the Pharmaceutical and Medical Device Act (PMD Act) of Japan, a drug must meet the following conditions in order to be considered for orphan drug designation in Japan: the number of people expected to use the drug for its intended use is less than 50,000 people in Japan; there is no suitable alternative drug or treatments in Japan, or the proposed drug is expected to be significantly more effective or safer than drugs already available on the Japanese market; and there is a scientific rationale to support the necessity of the drug for the target disease, and the development plan for the drug is appropriate. Specific measures to support the development of orphan drugs include giving prioritized consultation regarding clinical development and conducting priority examinations, reducing application fees, extending registration validity period, granting subsidies for research and development expenditures, and tax incentives.
1 Watanabe Miyano S. et al., "E7090, a Novel Selective Inhibitor of Fibroblast Growth Factor Receptors, Displays Potent Antitumor Activity and Prolongs Survival in Preclinical Models", Molecular Cancer Therapeutics, 2016, 15(11), 2630-2639.

2 Latest statistics, Cancer Information Service, National Cancer Center, Japan.

3 Official Statics of Japan (e-Stat), 2017 Patient Survey View Source(New Window)

4 The 21st Follow-up Survey Reports for Primary Liver Cancer Cases in Japan (2010-2011), 2020.

5 Shin Ishihara. et al., "Biliary tract cancer registry in Japan from 2008 to 2013", J Hepatobiliary Pancreat Sci., 2016, 23, 149-157.

6 Arai Y. et al., "Fibroblast growth factor receptor 2 tyrosine kinase fusions define a unique molecular subtype of cholangiocarcinoma", Hepatology, 2014, 59, 1427-1434.

JHL Biotech Officially Changes Name to EDEN Biologics

On February 19, 2021 JHL Biotech, Inc., a global frontrunner in large-molecule biological drug development and manufacturing platform technologies, reported that it has changed its corporate name to Eden Biologics, Inc (Press release, JHL Biotech, FEB 19, 2021, View Source [SID1234635605]).

The name change reflects the company’s continual commitment to producing affordable medicines for all patients in need and its evolving expertise in driving the innovations needed to shape the future of high-quality biologics development.

Please visit our new website at: www.edenbiologics.com

Eden Biologics will continue to fully operate in its current structure and all business contacts will remain unchanged.

"This is an exciting change for us and our evolving positioning effectively stamps our footprint as a global leader in the healthcare industry with focus on innovative therapeutics, which are needed now more than ever," said James Huang, Executive Chairman and CEO of Eden Biologics.

"We have developed numerous unique and expert competencies in-house, serving both our extensive Biologics Portfolio and our CDMO business. While our commitment to our Mission and Vision remains as strong as ever, we have an incredible ambition to grow and want Eden Biologics to become synonymous with the highest product quality and a pioneering technology platform for global biologics development."

The new name is effective immediately and has already been implemented across the company’s pipeline and services.

Junshi Biosciences Announces Acceptance by the NMPA of Supplemental New Drug Application for Toripalimab Combined with Chemotherapy for The First-Line Treatment of Nasopharyngeal Carcinoma

On February 19, 2021 Junshi Biosciences (HKEX: 1877; SSE: 688180), a leading innovation-driven biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies, reported that the National Medical Products Administration (NMPA) of China has accepted its supplemental New Drug Application (sNDA) for Toripalimab combined with chemotherapy for the first-line treatment of patients with recurrent or metastatic nasopharyngeal carcinoma (Press release, Shanghai Junshi Bioscience, FEB 19, 2021, View Source [SID1234575791]).

The supplemental NDA is based on the JUPITER-02 study (NCT03581786), which is a randomized, double-blind, placebo-controlled Phase III study led by Professor Ruihua Xu from Sun Yat-sen University Cancer Center. The results of the study showed that Toripalimab combined with gemcitabine/cisplatin as a first-line treatment for patients with recurrent or metastatic nasopharyngeal carcinoma significantly prolonged the progression-free survival as compared with the standard first-line treatment of gemcitabine/cisplatin. This study is the world’s largest international Phase III clinical study for any checkpoint inhibitor combined with chemotherapy in the first-line treatment of recurrent or metastatic nasopharyngeal carcinoma.

Junshi Biosciences has developed clinical programs using Toripalimab alone or in combination with other therapies for the treatment of nasopharyngeal carcinoma regardless of extent of prior treatments. The company has submitted two sNDAs of toripalimab for the treatment of NPC in China. Toripalimab also obtained Breakthrough Therapy and Orphan Drug Designations from the US FDA for this indication, and its Biologics License Applications (BLA) in the United States will be submitted in the near future. Toripalimab is likely to become the first Chinese anti-PD-1 monoclonal antibody to achieve commercialization in the overseas markets.

About Nasopharyngeal carcinoma
Nasopharyngeal carcinoma is a malignant tumor that occurs in nasopharyngeal mucosal epithelium, which is one of the most common head and neck cancers. According to the World Health Organization, the number of newly diagnosed nasopharyngeal carcinoma cases in 2020 has reached approximately 133,000 worldwide, and nearly half of the cases occurred in China.

About Toripalimab
Toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing in China. More than thirty company-sponsored clinical studies covering more than fifteen indications have been conducted globally, including in China and the United States. On 17 December 2018, Toripalimab obtained a conditional approval from the NMPA for the second-line treatment of unresectable or metastatic melanoma. Toripalimab was included in the 2019 and 2020 Guidelines of Chinese Society of Clinical Oncology (CSCO) for the Diagnosis and Treatment of Melanoma. Two supplemental New Drug Applications ("NDAs") of Toripalimab for the third-line treatment of recurrent or metastatic nasopharyngeal carcinoma and the second-line treatment of metastatic urothelial carcinoma were accepted by the NMPA in April 2020 and May 2020, respectively. Both supplemental NDAs received priority review designations from the NMPA in July 2020. In addition, Toripalimab has been granted Breakthrough Therapy Designation ("BTD") by the US Food and Drug Administration ("FDA") for the treatment of recurrent or metastatic nasopharyngeal carcinoma in September 2020. In December 2020, Toripalimab Injection was successfully included in the updated National Reimbursement Drug List ("NRDL"). Currently, Toripalimab has been granted 1 Breakthrough, 1 Fast Track , and 3 Orphan Drug Designations by the FDA for the treatment of mucosal melanoma, nasopharyngeal carcinoma, and soft tissue sarcoma.

Year End Report 2020

On February 19, 2021 Affibody Medical AB (publ) ("Affibody" or "the Company"), a Swedish biotech company focused on developing next generation biopharmaceuticals based on its unique proprietary technology platforms: Affibody molecules and Albumod, reported its Year-End Report for 2020 (Press release, Affibody, FEB 19, 2021, View Source [SID1234575695]).

Financial Highlights

Revenue for the quarter amounted to SEK 1.9 (17.2) m, and to 121.3 (311.8) m for the full year
Operating result for the quarter amounted to SEK -87.5 (-66.1) m, and to -214.9 (44.8) m for the full year
Net result for the quarter amounted to SEK -89.4 (-72.9) m, and to -220.3 (44.6) m for the full year
Cash flow for the quarter amounted to SEK -42.3 (-14.4) m, and to -238.9 (283.8) m for the full year
Cash and cash equivalents at the end of the period amounted to SEK 135.9 (374.8)
Significant Events during the rest of the Reporting Period

On November 10, 2020, we announced the initiation of a 52-week trial investigating the novel bispecific IL-17A inhibitor ABY-035 in patients with psoriatic arthritis (PsA).
Significant Events during the rest of the Year

An EGM on February 17, 2020 elected José Suarez as Board member.
On April 30, 2020 we announced that Daewoong, a South Korea-based pharmaceutical company, had exercised an option under the collaboration related to a half-life extended biotherapeutics product
On May 15, 2020, we and Inmagene Biopharmaceuticals announced a strategic partnership to develop and commercialize ABY-035, a bispecific molecule targeting Interleukin-17A (IL-17), for multiple auto-immune diseases. Inmagene will be responsible for commercialization in mainland China, Hong Kong, Taiwan, and Macau (Greater China), and South Korea, as well as development activities in the Asia Pacific region, excluding Japan. Affibody will retain global commercial rights outside of Greater China and South Korea. The partners will work together to enroll patients into global registrational trials to support Biologics License Applications (BLAs) in multiple indications worldwide. Under the terms of the agreement, Affibody will receive a $10 million upfront payment and is eligible to receive up to $215.5 million in additional regulatory and sales milestones, plus royalties on sales in Inmagene’s commercialization territory.
On June 15, 2020 we announced positive top-line data from our Phase 2, 52-week trial investigating the novel bispecific IL-17A inhibitor ABY-035 in patients with moderate-to-severe psoriasis ("AFFIRM-35").
On June 15, 2020 we announced the completion of the ABY-039 Phase 1 trial and the termination of the ABY-039 program, our FcRn inhibitor, due to tolerability observations that would limit the target product profile of subcutaneous high dose once monthly maintentance injections. Based on these observations Alexion has terminated the co-development agreement with Affibody.
Significant Events after the close of the Reporting Period

On February 10, Affibody AB and Inmagene Biopharmaceuticals announced that the U.S. FDA has cleared izokibep (ABY-035) to proceed to Phase 2 clinical development in Ankylosing Spondylitis (AS), a subset of Axial Spondyloarthritis (axSpA). The partners are jointly developing izokibep to treat multiple autoimmune diseases, and Inmagene has taken the responsibility to manage the global clinical trials for axSpA.
Affibody is a clinical stage biopharmaceutical company with a broad product pipeline focused on developing innovative bi- and multi-specific next generation biopharmaceuticals based on its unique proprietary technology platforms: Affibody molecules and Albumod.

The company operates a focused experimental medicine model and currently has two clinical stage programs. The first is a therapeutic program that targets IL-17 mediated autoimmune and inflammatory diseases. The second program is a diagnostic imaging program that is directed primarily towards metastatic breast cancer.

Affibody AB is a holding of Patricia Industries.

Octapharma: New Consensus Recommendations on Treatment of Secondary Antibody Deficiency in Patients With Haematological Malignancies

On February 19, 2021 Octapharma reported that New expert consensus guidelines on the use of immunoglobulin replacement therapy (IgRT) in patients with haematological malignancy and secondary antibody deficiencies (SAD) were recently published in the European Journal of Haematology (Press release, Octapharma, FEB 19, 2021, View Source [SID1234575392]).

The publication, sponsored by Octapharma, marks the first pan-European consensus guidance on the use of IgRT in patients with hypogammaglobulinaemia secondary to haematological malignancies and aims to support harmonisation of clinical practice across Europe.

Octapharma has a longstanding commitment to improving the management of patients with secondary immunodeficiencies (SID) and in 2020 launched PRO-SID, a phase III clinical trial investigating primary infection prophylaxis with intravenous immunoglobulin (IVIg) in patients with chronic lymphocytic leukaemia (CLL). These efforts address the need for both robust clinical data and unified guidance on managing infection risk in patients with SID.

Absence of guidance leaves patients at risk

SAD is a common complication in patients with haematological malignancies such as CLL and multiple myeloma (MM). Up to 85% of CLL patients and up to 83% of patients with smouldering MM have low immunoglobulin levels, which leaves patients more susceptible to infections1,2. Infections are the likely cause of death in 22% of patients with MM and up to 50% of patients with CLL3,4. IgRT is effective at reducing the risk of infections in patients with haematological malignancies5.

In 2019, the EMA approved an expanded use of IVIg in patients with SID6. However, detailed European guidelines on the use of IgRT in patients with haematological malignancies and SAD were lacking. Differences in treatment approaches to reduce the infection burden remain significant across Europe, including different strategies for initiation, dosing and discontinuation of IgRT.

Stephen Jolles, lead author of the publication and Professor at the Immunodeficiency Centre for Wales in Cardiff, UK, commented: "Developing consensus guidelines for the use of IgRT in secondary antibody deficiency (SAD) aims to address a major need for treatment recommendations for patients with haematological malignancies and SAD. IgRT can reduce morbidity and mortality in a selected group of these patients and it is important that physicians have consistent guidance on defining this group and managing infection risk."

A Task Force of eight experts in immunology and haemato-oncology developed statements on key aspects of IgRT, which were reviewed by a panel of 32 European experts. This Delphi consensus exercise developed clear recommendations for SAD due to haematological malignancies such as: measurement of IgG levels at the beginning of anti-cancer treatment; initiation of IgRT in patients who have received appropriate anti-infective therapy during or after a single severe infection or during recurrent or persistent infections when IgG levels are <4 g/l or if test immunization has failed; initiation of IgRT with a minimum IgG dose of 0.4 g/kg bodyweight every 3-4 weeks or stopping IgRT after at least 6 months without infections and concomitant evidence of immunological recovery. The 21 consensus statements emphasise the importance of IgRT for patients with SAD who experience severe, recurrent or persistent infections and provide guidance on initiation, dosing and discontinuation of IgRT, as well as measurement of IgG levels and the use of subcutaneous immunoglobulin (SCIg) therapy. The publication is available through open access at "Treating Secondary Antibody Deficiency in Patients with Haematological Malignancy: European Expert Consensus".

More information on SID in patients with haematological malignancies, including detailed information on the recent consensus guidelines, can be found at View Source

Patient recruitment continues for the PRO-SID study

Recruitment for the phase III PRO-SID study (NCT04502030) of IVIg in patients with CLL and SID is underway, in 22 sites across 7 countries. The PRO-SID study is investigating the efficacy and safety of IVIg (Panzyga) as primary prophylaxis in patients with CLL and SID. Secondary prophylaxis with IVIg is an established approach to reduce the rate of infections in patients with haematological malignancies and SAD, but there is a need for robust data on IVIg as primary infection prophylaxis, i.e. before a major infection occurs7.

Commenting on Octapharma’s involvement in the field of SID, Olaf Walter, Board Member at Octapharma, said that: "Infections remain a serious concern for patients with haematological malignancies and SID, and at Octapharma we continue to strive for a better understanding of how to minimise the risk of such potentially life-threatening complications."

About the PRO-SID study

The PRO-SID study (NCT04502030) is a prospective, double-blind, randomised, multi-centre, placebo-controlled, interventional, phase III study investigating the efficacy and safety of Panzyga in patients with chronic lymphocytic leukaemia (CLL) and hypogammaglobulinaemia (IgG < 5 g/L) who are receiving antineoplastic treatment. The study is conducted at multiple sites across Europe (Italy, Poland, Denmark, Hungary, Germany and Russia) and the USA and plans to recruit at least 240 patients.

About Panzyga

Panzyga is a 10% human normal immunoglobulin solution ready for intravenous administration. Panzyga is approved for use in treatment of primary immunodeficiency and idiopathic thrombocytopenic purpura in the USA, Europe and Canada. It is also approved for secondary immunodeficiencies and Guillain Barré syndrome in Europe and Canada and for Kawasaki disease, chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) and multifocal motor neuropathy (MMN) in Europe.