CyGenica Limited Secures USFDA Approval for Orphan Drug Designation for Novel Drug Conjugate in Glioblastoma Multiforme Treatment

On September 12, 2023 CyGenica Limited, a dynamic biotech startup reported a significant achievement in the battle against Glioblastoma Multiforme (GBM), a highly aggressive form of brain cancer (Press release, CyGenica, SEP 12, 2023, View Source [SID1234635121]). Dr. Nusrat J M Sanghamitra, Co-founder and CEO of CyGenica, revealed that the U.S. Food and Drug Administration (USFDA) has granted Orphan Drug Designation for their revolutionary drug conjugate for the treatment of GBM. This momentous approval represents a significant advancement for CyGenica’s innovative intracellular delivery platform, GEENIE, and offers new avenues of hope for patients fighting cancer and rare diseases.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

GBM continues to pose a formidable challenge in the field of oncology, calling for novel and effective treatment approaches. CyGenica’s Orphan Drug Designation approval for their novel drug conjugate demonstrates a resolute commitment to address the unmet medical needs of GBM patients.

This momentous achievement marks a pivotal milestone in CyGenica’s journey as the first application of their targeted intracellular delivery platform, GEENIE, for any disease indication. Beyond GBM, the company envisions a world where GEENIE empowers the development of innovative therapies for various cancers and rare diseases, including nucleotide-based therapeutics like antisense oligonucleotides, SiRNAs, and genome editing therapeutics.

Dr. Nusrat J M Sanghamitra stated, "We are incredibly thrilled to receive the USFDA’s approval for our Orphan Drug Designation for our novel drug conjugate in GBM treatment. This validation paves the way for CyGenica to push the boundaries of innovation and expand our intracellular delivery platform, GEENIE to bring hope to countless patients."

As CyGenica continues to advance their novel drug conjugate and GEENIE platform, the company is eager to collaborate with biotech investors and pharmaceutical companies.

"This is a remarkable opportunity for potential partners to join us in the global fight against GBM, other types of cancers, and rare diseases," Dr. Sanghamitra emphasized. "Together, we can unlock the full potential of GEENIE and create a lasting impact on the lives of patients worldwide."

Henlius Expands Collaboration with KGbio to Develop, Commercialize and Supply Henlius’ novel anti-PD-1 mAb HANSIZHUANG in MENA

On September 12, 2023 Shanghai Henlius Biotech, Inc. (2696.HK) reported that it has entered into an exclusive license agreement with PT Kalbe Genexine Biologics ("KGbio"), an Indonesian pharmaceutical company and a holding subsidiary to PT Kalbe Farma, Tbk ("Kalbe"), for the development and commercialisation of HANSIZHUANG (serplulimab injection) as a treatment for two indications including ES-SCLC, Henlius’ novel anti-PD-1 mAb, in 12 Middle East and North African (MENA) countries including Saudi Arabia, the United Arab Emirates, Egypt, Qatar, Jordan, Morocco, etc (Press release, Shanghai Henlius Biotech, SEP 12, 2023, View Source [SID1234635120]). In 2019, Henlius reached a collaboration agreement with KGbio, upon which KGbio is granted exclusive rights to develop and commercialize HANSIZHUANG in certain therapies in 10 ASEAN member countries.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Under the terms of the agreement, Henlius will be responsible for manufacturing and supply and will receive a US$7 million upfront payment, up to US$8 million in regulatory milestones, and royalties from KGbio ranging from 15% to 20% of net sales of HANSIZHUANG in the licensed MENA territory. Henlius will also be eligible to receive up to US$650 million upon the achievement of sales milestones in the licensed 22 countries in Southeast Asia and MENA.

"HANSIZHUANG is the first and only anti-PD-1 therapy approved for first-line treatment of small cell lung cancer. More than 30,000 Chinese patients have benefited from its excellent efficacy. We are excited to join forces with KGbio in MENA after the license granted in Southeast Asia," said Jason Zhu, Chief Executive Officer, President, and Chief Financial Officer of Henlius. "Our aim is to continue supporting and improving the treatment outcomes and quality of life for local patients. Through KGbio’s commercial network and operational capabilities in MENA, we firmly believe that HANSIZHUANG will bring new hope and health to patients in Southeast Asia and MENA."

Ms. Ping Cao, Chief Business Officer, and VP of Business Development of Henlius, said, "The collaboration between Henlius and KGbio since 2019 was driven by a shared vision and mission and has opened a vast potential market for HANSIZHUANG in Southeast Asia. In the past 4 years, both parties have demonstrated a high level of synergistic effect driven by shared objectives. It has laid a strong foundation for our continued collaboration and I’m confident that together, we will continue to achieve more success in MENA."

"We welcome the collaboration between KGbio and Henlius. With KGbio’s network and operational capabilities in the Middle East and North Africa, this collaboration is an effort for the two companies to develop the products, especially for innovative biological products," said Sie Djohan, President Director of KGbio, who is also the Director of Kalbe, KGbio’s holding company.

The company’s first innovative product, HANSIZHUANG (serplulimab), was approved in China in March 2022. At present, HANSIZHUANG has been approved for 3 indications in China including MSI-H solid tumour, squamous non-small cell lung cancer (sqNSCLC) and extensive stage small cell lung cancer (ES-SCLC). With its breakthrough efficacy and differentiation advantages in the relevant treatment fields, HANSIZHUANG has earned wide recognitions and its pivotal clinical research results have been published in leading medical journals such as the Journal of the American Medical Association (JAMA). Its synergy with in-house products of the company and innovative therapies are being actively promoted and over 10 clinical trials on immuno-oncology combination therapies are in progress in a wide variety of indications, such as lung cancer, esophageal carcinoma, gastric cancer, etc., covering full range of first-line treatments of lung cancers. As of now, the company has enrolled more than 3,600 subjects globally, and the proportion of White is over 30% in two multi-regional clinical trials (MRCTs). Its global clinical trial data will further support marketing applications in global markets and lay a foundation for clinical application all over the world in the future.

In the future, the two companies will fully leverage their respective resources and advantages to promote the commercialisation of HANSIZHUANG in Southeast Asia and MENA. It is anticipated that this will enhance HANSIZHUANG’s accessibility in emerging markets and bring high-quality, affordable, and innovative medicines to more patients.

About HANSIZHUANG

HANSIZHUANG (recombinant humanized anti-PD-1 monoclonal antibody injection, generic name: serplulimab injection) is the first anti-PD-1 mAb for the first-line treatment of SCLC. Up to date, 3 indications are approved for marketing in China, 2 marketing applications are under review in China and the EU, and more than 10 clinical trials are ongoing across the world.

HANSIZHUANG was launched in March 2022 and has been approved by the National Medicinal Products Administration (NMPA) for the treatment of MSI-H solid tumours, squamous non-small cell lung cancer (sqNSCLC) and extensive-stage small cell lung cancer (ES-SCLC). The marketing applications of the first-line treatment for esophageal squamous cell carcinoma (ESCC) and ES-SCLC are under review by the NMPA and the European Medicines Agency (EMA), respectively. Focus on lung and gastrointestinal cancer, the synergy of HANSIZHUANG with in-house products of the company and innovative therapies are being actively promoted. It has successively obtained clinical trial licenses in China, the United States, the European Union and other countries and regions to initiate more than 10 clinical trials on immuno-oncology combination therapies in a wide variety of indications. As of now, the company has enrolled more than 3,600 subjects in China, the U.S., Turkey, Poland, Georgia and other countries and regions, and the proportion of White is over 30% in two MRCTs, making HANSIZHUANG an anti-PD-1 mAb with one of the largest global clinical data pools. The results of 3 pivotal trials of HANSIZHUANG were published in the Journal of the American Medical Association (JAMA), Nature Medicine, and the British Journal of Cancer, respectively. Furthermore, HANSIZHUANG was recommended by the CSCO Guidelines for Small Cell Lung Cancer, the CSCO Guidelines for Non-Small Cell Lung Cancer, the CSCO Guidelines Esophageal Cancer, the CSCO Guidelines for Colorectal Cancer, the CSCO Clinical Practice Guidelines on Immune Checkpoint Inhibitor, the China Guidelines for Radiotherapy of Esophageal Cancer and other definitive guides, providing valuable references for clinical diagnosis and treatment of tumours. On the other hand, Serplulimab was granted orphan drug designations by the FDA and the EC for the treatment of SCLC, and its bridging head-to-head trial in the United States to comparing HANSIZHUANG to standard of care Atezolizumab (anti-PD-L1 mAb) for the first-line treatment of ES-SCLC is well under way.

Dewpoint Therapeutics Partners with Chemify to Apply Groundbreaking Chemistry AI to Radically Accelerate the Discovery of Molecules Targeting Cancer and Neurodegeneration

On September 12, 2023 Dewpoint Therapeutics and Chemify reported a partnership to develop a suite of digitally discovered and automatically synthesized novel molecules on Chemify’s programmable chemistry platform against condensate targets of interest in Dewpoint’s oncology and neurodegeneration pipeline (Press release, Dewpoint Therapeutics, SEP 12, 2023, View Source [SID1234635119]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The partnership brings together Chemify’s pioneering chemistry AI and automation technology platform, capable of designing, discovering and making complex molecules on demand using a chemical programming language, with Dewpoint’s groundbreaking condensate biology and AI technology platform. Chemify, who recently announced raising $43M of funding (read more), has developed an automated design, discovery and synthesis platform, which will be employed to amplify and accelerate Dewpoint’s ongoing chemistry efforts.

Under the partnership, Chemify will design and deliver novel compounds to augment Dewpoint’s advanced oncology and neurodegeneration pipeline. Dewpoint may choose to exercise an option to acquire the compounds designed by Chemify. In exchange, Chemify will receive pre-negotiated, success-driven clinical and regulatory milestones and tiered royalties.

"Dewpoint’s partnership with Chemify offers an innovative pathway to take our novel chemical matter already in optimization and bootstrap Chemify’s novel chemical space exploration tool suite using state-of-the-art digital chemistry AI-powered concepts. This is a new frontier for drug discovery and we’re delighted to partner with Chemify who is at the cutting-edge of this approach," commented Ameet Nathwani, M.D., CEO at Dewpoint.

"We are extremely excited by this partnership which combines Chemify’s technology for molecular design, discovery and synthesis with Dewpoint’s condensate biology approach to produce a seamless drug discovery and design platform. It is truly inspiring to be using the Chemify technology I have been building for 15 years to change and improve the lives of patients," commented Professor Lee Cronin, Chemify’s CEO and Regius Professor of Chemistry at the University of Glasgow.

About Condensates
Condensates are membraneless organelles that form dynamically throughout the cell via a process called phase separation. These subcellular compartments organize and concentrate molecules within cells to enable certain key biochemical processes. The dysregulation of biomolecular condensates has been observed in many diseases, including cancer, diabetes and neurological disorders. Dewpoint’s platform has discovered disease-driving condensates and developed condensate-modifying drugs that potentially provide novel therapeutic options for complex diseases and historically undruggable targets.

Traverse Biotech announces a world-wide license agreement with Genmab to develop and commercialize a novel cancer bispecific antibody

On September 12, 2023 Traverse Biotech, a US-based biotechnology company focused on the development of immunotherapies from international biopharma companies, reported that it has signed a license agreement with Genmab under which Traverse will develop and commercialize a bispecific antibody for cancer immunotherapy (Press release, Traverse Biotech, SEP 12, 2023, View Source [SID1234635118]). This next-generation antibody was created using Genmab’s proprietary DuoBody bispecific antibody technology platform as a targeted treatment candidate for cancers expressing an undisclosed tumor-associated antigen. Targeting this tumor-associated antigen with the bispecific antibody will direct T cell-mediated cytotoxicity against both solid and liquid tumors.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Under the terms of the license agreement, Traverse will use its operational and development expertise to advance and commercialize the bispecific antibody program. This license agreement is expected to bring a much-needed novel oncology product to patients. The aim of the license agreement is to leverage DuoBody, a proven technology and the basis of four FDA-approved bispecific medicines and develop and commercialize a novel immuno-oncology product.

"We are highly motivated to work with a product candidate derived from Genmab’s state-of-the-art bispecific technology," said Brandy Houser, Ph.D., CEO of Traverse Biotech. "By focusing our energy on developing assets from clinically validated technologies, we can accelerate development and ultimately improve the treatment landscape for patients battling cancer," said Bill Polvino, M.D., Executive Chairman.

Pierre Fabre Laboratories Acquires Vertical Bio and its Innovative Targeted Therapy Candidate for Patients Suffering From Non-Small Cell Lung Cancer With MET Alteration

On September 12, 2023 Pierre Fabre Laboratories, the French pharmaceutical and dermo-cosmetic company, reported the acquisition of Vertical Bio AG, a developer of novel cancer therapies (Press release, Pierre Fabre, SEP 12, 2023, View Source [SID1234635117]). This first acquisition of a biotechnology company allows Pierre Fabre Laboratories to add VERT-002 to its oncology discovery pipeline. VERT-002 is a monoclonal antibody with a novel and differentiated mechanism of action, acting as a degrader of c-MET. This target is a known disease driver in patients suffering from Non-Small Cell Lung Cancer (NSCLC) with mutations or amplification of MET. Deal terms were not disclosed.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Vertical Bio was founded by Versant Ventures and was launched out of the firm’s Ridgeline Discovery Engine based in Basel, Switzerland. Leveraging Ridgeline’s biology capabilities, Vertical Bio advanced VERT-002 through preclinical studies and towards an IND submission. The first-in-human studies are expected to begin in 2024.

This acquisition enables Pierre Fabre Laboratories to further strengthen their R&D portfolio in precision oncology with a product about to enter clinical development. In line with its innovation strategy, the Group has made oncology its top priority in medical care and dedicates every year circa 80% of its R&D spendings to this therapeutic area. In 2022, the Oncology Franchise achieved revenues of 467 M€, a sharp 221% increase vs 2019 revenues.

Pierre Fabre Laboratories have a long history in the treatment of lung cancers, dating back to the 90’s with the launch of their first chemotherapy. Earlier this year, a collaboration agreement was signed with Scorpion Therapeutics to co-develop and commercialize two candidates for patients with EGFR-mutant NSCLC. And through its longstanding partnership with Pfizer, the company expects to harness soon the full potential of a promising clinical development program in NSCLC.

"We are excited about the acquisition of this biotechnology company and the addition of VERT-002 to reinforce our research and development portfolio in lung cancer. This acquisition is another testimony of our commitment to invest in the discovery and development of innovative treatments in precision oncology" said Eric Ducournau, Chief Executive Officer of Pierre Fabre Laboratories.

"We are proud of the rapid progress that Vertical Bio made in developing its lead antibody, which represents a potential new option for patients with difficult-to-treat forms of lung cancer," said Alex Mayweg, Ph.D., chairman of Vertical Bio and managing director at Versant. "Pierre Fabre Laboratories are an ideal partner to take VERT-002 into the clinic and we look forward to the to the continued progress of this differentiated molecule."

About VERT-002

VERT-002, an antibody targeting c-MET that leads to its degradation, with potential as best-in-class therapeutic option for patients with MET alterations, including resistance settings.

VERT-002 is currently completing IND-enabling studies and Vertical Bio expects to begin first in human studies in 2024.