On November 7, 2022 Galapagos NV (Euronext & NASDAQ: GLPG) reported its strategy for accelerated growth and value creation, its financial results for the first nine months of 2022, and the outlook for the remainder of 2022 (Press release, Galapagos, NOV 7, 2022, View Source [SID1234623242]). The results are further detailed in the Q3 2022 financial report available on the financial reports section of the website.

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Forward, Faster strategy to accelerate innovation

"Guided by our purpose to bring transformational medicines to patients around the world to help them live longer and healthier lives, we today announce our Forward, Faster strategy to accelerate growth and value creation by reshaping the way we innovate and operate. This strategy provides a clear path forward based on three key pillars. First, we will shift from novel target-based discovery to patient-centric medical need research and development with a focus on our key therapeutic areas of immunology and oncology. Second, we will build on our current capabilities and derisk R&D through multiple drug modalities, including CAR-T, and by focusing on best-in-disease validated targets in our strategic therapeutic areas with shorter time-to-patient potential. Third, we will increase our business development efforts to complement our pipeline and continue to work with our collaboration partner Gilead to bring more medicines to patients worldwide," said Dr. Paul Stoffels1, CEO and Chairman of the Board of Directors of Galapagos.

"Our new fit-for-purpose organizational structure and operating model will focus on accelerating our pipeline in immunology and oncology, supported by externally sourced opportunities, and we will discontinue our activities in fibrosis and kidney disease2.

As a result of our new strategic direction, we intend to reduce our workforce by approximately 200 positions across our sites in Europe to create room to reinvest in new capabilities and programs in our oncology franchise. This is a difficult but necessary decision, and we will follow all applicable processes with respect for our people."

Third quarter financial performance
"Jyseleca continues to perform very well with a growing European base and a solid €60.5 million in net sales as of 30 September. As a result, we further increase our 2022 net sales guidance to €80-€90 million from our initial guidance of €65-€75 million. We ended the third quarter of the year with a strong balance sheet of €4.4 billion in cash and current financial investments, which provides us with the necessary means to invest in immunology and oncology and execute on smart business development. We reiterate our cash burni guidance of €480-€520 million for the full year 2022," added Bart Filius, President, COO and CFO of Galapagos.

Q3 2022 operational review and recent events
Jyseleca commercial & regulatory progress

Strong adoption across Europe with reimbursement for rheumatoid arthritis (RA) in 15 countries and for ulcerative colitis (UC) in 10 countries
Marketing Authorization Application (MAA) submitted for the treatment of UC to Swissmedic, the regulatory authority in Switzerland
Article 20 pharmacovigilance procedure ongoing by the European Medicines Agency (EMA)

Pipeline update

Preparations advanced to start Phase 2 of GLPG3667 (TYK2 inhibitor) in dermatomyositis, with the aim to recruit the first patients around year-end

Corporate update

Received various transparency notifications from FMR LLC, indicating that its shareholding in Galapagos changed, without crossing below the 5% threshold, to 5.65% of the current outstanding Galapagos shares
Raised €6.7 million year-to-date through the exercise of subscription rights
Post-period events

On 27 October 2022, the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA) concluded the safety review (Article 20 procedure) of all JAK inhibitors approved in the EU for the treatment of inflammatory diseases. On 28 October 2022, the PRAC recommended the harmonization of all labels and concluded that JAK inhibitors should remain indicated for the treatment of patients with RA who have responded inadequately to or who cannot tolerate disease modifying anti-rheumatic drugs (DMARDs) therapy, and for patients with UC who have responded inadequately to or who cannot tolerate conventional therapy or biologics. The PRAC also recommended to update all product labels to include a precautionary approach for use of JAK inhibitors in patients with identified risk factors only if no suitable treatment alternative is available (Section 4.4 – Warning and Precautions). The PRAC recommendations will now be considered by the Committee for Medicinal Products for Human Use (CHMP) for an opinion
Abstract accepted for poster presentation at the Annual Society of Hematology (ASH) (Free ASH Whitepaper) conference taking place 10-13 December 2022 on the initial data from the ATALANTA-1 Phase 1/2 study in recurring/refractory Non-Hodgkin Lymphoma (rrNHL) evaluating the feasibility, safety and efficacy of the CD19 CAR-T candidate manufactured at point-of-care
New post hoc analyses from SELECTION Phase 3 data set with filgotinib in UC patients presented at the United European Gastroenterology (UEG) Week
Received positive CHMP opinion for Jyseleca European label update based on testicular function safety data from MANTA/RAy studies
We reported product net sales of Jyseleca in Europe for the first nine months of 2022 amounting to €60.5 million (€6.1 million in the first nine months of 2021). Our counterparties for the sales of Jyseleca were mainly hospitals and wholesalers located across Europe.

Cost of sales related to Jyseleca net sales in the first nine months of 2022 amounted to €7.9 million.

Collaboration revenues amounted to €349.7 million for the first nine months of 2022, compared to €311.7 million for the first nine months of 2021.

Revenues recognized related to the collaboration agreement with Gilead for the filgotinib development were €166.8 million in the first nine months of 2022 compared to €136.4 million for the same period last year. This increase was due to a higher increase in the percentage of completion, as well as a higher revenue recognition of milestone payments, strongly influenced by the milestone achieved related to the regulatory approval in Japan for UC in the first nine months of 2022. The revenue recognition related to the exclusive access rights for Gilead to our drug discovery platform amounted to €172.6 million for the first nine months of 2022 (€173.3 million for the same period last year).

We have recognized royalty income from Gilead for Jyseleca for €8.2 million in the first nine months of 2022 (compared to €1.9 million in the same period last year) of which €3.6 million royalties on milestone income for UC approval in Japan.

Additionally, we recorded milestones of €2.0 million triggered by the first sales of Jyseleca in the Czech Republic and Portugal by our distribution and commercialization partner Sobi, in the first nine months of 2022.

Our deferred income balance on 30 September 2022 includes €1.6 billion allocated to our drug discovery platform that is recognized linearly over the remaining period of our 10-year collaboration, and €0.5 billion allocated to the filgotinib development that is recognized over time until the end of the development period.

Our R&D expenditure in the first nine months of 2022 amounted to €364.1 million, compared to €378.0 million for the first nine months of 2021. This decrease was primarily explained by a decrease in subcontracting costs from €189.1 million in the first nine months of 2021 to €158.5 million in the first nine months of 2022, primarily due to the winding down of the ziritaxestat (IPF) program and reduced spend on our SIKi and TYK2 programs. This was partly offset by cost increases for our filgotinib program, on a nine month basis compared to the same period in 2021. Personnel costs decreased from €134.3 million in the first nine months of 2021 to €130.0 million for the same period this year. Depreciation and impairment amounted to €35.6 million for the first nine months of 2022 (€14.1 million for the same period last year). This increase was primarily due to an impairment of €26.7 million of previously capitalized upfront fees related to our collaboration with Molecure on the dual chitinase inhibitor OATD-01 (GLPG4716) recorded in Q2 2022.

Our G&A and S&M expenses amounted to €202.7 million in the first nine months of 2022, compared to €151.3 million in the first nine months of 2021. This increase was primarily due to the termination of our 50/50 filgotinib co-commercialization cost sharing agreement with Gilead for filgotinib in 2022. The cost increase was also explained by an increase in personnel costs for the first nine months of 2022 compared to the same period last year explained by an increase in the commercial work force driven by the commercial launch of filgotinib in Europe.

Other operating income (€29.5 million vs €36.3 million for the same period last year) decreased, mainly driven by lower grant and R&D incentives income.

Net financial income in the first nine months of 2022 amounted to €127.5 million, compared to net financial income of €33.6 million for the first nine months of 2021. Net financial income in the first nine months of 2022 was primarily attributable to €102.1 million of unrealized currency exchange gains on our cash and cash equivalents and current financial investments at amortized cost in U.S. dollars, and to €26.0 million of positive changes in (fair) value of current financial investments. The financial expenses also contained the effect of discounting our long term deferred income of €5.7 million.

We realized a net loss from continuing operations of €10.8 million for the first nine months of 2022, compared to a net loss of €141.8 million for the first nine months of 2021.

The net profit from discontinued operations for the nine months ended 30 September 2021 consisted of the gain on the sale of Fidelta, our fee-for-services business, for €22.2 million.

We reported a group net loss for the first nine months of 2022 of €10.8 million, compared to a group net loss of €119.6 million for the first nine months of 2021.

Cash position
Current financial investments and cash and cash equivalents totaled €4,362.1 million on 30 September 2022, as compared to €4,703.2 million on 31 December 2021.

Total net decrease in cash and cash equivalents and current financial investments amounted to €341.1 million during the first nine months of 2022, compared to a net decrease of €295.2 million during the first nine months of 2021. This net decrease was composed of (i) €343.1 million of operational cash burn, (ii) offset by €6.7 million of cash proceeds from capital and share premium increase from exercise of subscription rights in the first nine months of 2022, (iii) €26.0 million positive changes in (fair) value of current financial investments and €105.6 million of mainly positive exchange rate differences, and (iv) the cash out from the acquisitions of CellPoint and AboundBio, net of cash acquired, of €136.4 million.

Acquisitions of CellPoint and AboundBio
The preliminary accounting of the acquisitions of CellPoint and AboundBio are included in our Q3 2022 condensed consolidated financial statements. To date, we have performed a preliminary fair value analysis of the business combinations. We expect the provisional amount of goodwill to change significantly upon the completion of the purchase price allocation, resulting from the valuation of the different assets and liabilities acquired.

Near term outlook

Immunology – an area in which we have built deep scientific know-how and expertise since our founding

We expect reimbursement decisions in most key European markets for Jyseleca in UC this year and anticipate that Sobi will further progress with reimbursement discussions in RA and UC in Eastern and Central Europe, Greece, and the Baltic countries. We also expect to report initial results from the FILOSOPHY Real-World Evidence Phase 4 trial in RA later this year, and topline results from the DIVERSITY Phase 3 study in Crohn’s disease (CD) in the first quarter of 2023. Before the end of this year, we anticipate a CHMP opinion following the PRAC Article 20 recommendation issued on 28 October 2022.

We aim to recruit the first patients in a Phase 2 study of our TYK2 inhibitor product candidate, GLPG3667, in dermatomyositis around year-end, and we intend to start a Phase 2 study in patients with Systemic Lupus Erythematosus (SLE) in 2023. Finally, we continue to advance select compounds with optimized pharmacology and selectivity from our SIKi portfolio.

Oncology portfolio – an area where we will continue to grow and invest

We will present the initial data from the ATALANTA-1 Phase 1/2 study of the CD19 CAR-T product candidate in patients with rrNHL at the annual ASH (Free ASH Whitepaper) conference in December. The objectives of the ATALANTA-1 study are to evaluate the feasibility, safety and efficacy of the CD19 CAR-T candidate manufactured at point-of-care and will provide initial clinical validation of the CAR-T decentralized supply model.

The recruitment of the ongoing Phase 1/2 studies of the CD19 CAR-T candidate in patients with rrNHL (ATALANTA study) and relapsed/refractory Chronic Lymphocytic Leukemia (rrCLL) (EUPLAGIA study) is progressing well, and we are on track to report topline results of the dose escalation cohorts in the first half of 2023, which will be followed by one or more dose expansion cohorts.

Financial guidance and Forward, Faster strategy presentation

For the full year 2022, we reiterate our net cash burn of €480-€520 million, including the acceleration in oncology, and we further increase our net sales guidance for Jyseleca to €80-€90 million.

A detailed update of the strategy, portfolio and pipeline goals and commercial progress will be presented by Galapagos management and key opinion leaders at the company’s R&D Day 2022 which will be held tomorrow, Friday, 4 November 2022, from 8:00 am to 10:30 am EDT (13:00 to 15:30 CET) in New York.

The event will include a live webcast on the Investors section of the company’s website and a replay will be available on the Galapagos website within 48 hours after the event. Presentations showcased during the event will be featured on the Presentations section of the company’s website.

To participate in the conference call, please register in advance using this link. Upon registration, the dial-in numbers will be provided. The conference call can be accessed 10 minutes prior to the start time by using the conference access information provided in the e-mail received at the point of registering, or by selecting the call me feature.

Third quarter 2022 financial report

Galapagos’ financial report for the first nine months ended 30 September 2022, including details of the unaudited consolidated results, is accessible on the financial reports section of our website.

On November 7, 2022 Repertoire Immune Medicines reported that Torben Straight Nissen has been appointed as interim Chief Executive Officer, succeeding John Cox, who will continue with the company in an advisory role (Press release, Repertoire, NOV 7, 2022, View Source;utm_source=dlvr.it&utm_medium=twitter [SID1234623241]). With more than two decades of leadership experience, Straight Nissen joins the company as it shifts its resources and operations to focus primarily on unlocking the potential of Repertoire’s DECODE Platform to develop transformative immune medicines against novel immune targets in cancer, autoimmune disorders, and infectious disease.

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"I want to thank John for his leadership and vision for harnessing the potential of the immune repertoire to develop new medicines," said Doug Cole, M.D., Chairman, Repertoire and Managing Partner, Flagship Pioneering. "As the first CEO of Repertoire, John established the company to deliver novel immune medicines to patients, and we will continue to build upon this bold ambition."

Cole continued, "We welcome Torben as the next CEO of Repertoire. His significant experience leading and shaping platform-based biotech companies makes him well-positioned to drive Repertoire forward toward unlocking the potential of its DECODE platform."

"Repertoire has applied its proprietary DECODE platform to develop an unprecedented and unique understanding of the immune synapse. I am energized by the opportunity to leverage Repertoire’s powerful platform to develop novel and highly selective immune-based medicines for the treatment of cancer, autoimmune disorders, and infectious disease," said Straight Nissen. "I look forward to working with Repertoire’s impressive team to provide new and important therapies for patients with these diseases."

"The mission of Repertoire is to discover the immune codes that drive cellular immunity and to use those codes to create new immune medicines," said Cox. "Our recent discoveries of novel epitopes and T cell receptors in cancer and autoimmune diseases demonstrate the promise of the platform that we built. I am grateful to each member of the Repertoire team for making these advancements possible, and to the leadership team that built this company. I look forward to seeing Repertoire continue its mission to create a new class of immune medicines under Torben’s leadership."

About Torben Straight Nissen

Torben Straight Nissen is a senior partner at Flagship Pioneering, contributing to Flagship’s strategic and operational objectives, including the origination of new Flagship Labs companies. He is an experienced biotech entrepreneur and has been a member of Flagship’s ecosystem since first joining as President of Rubius Therapeutics in 2016. Prior to joining Flagship, Straight Nissen was at Pfizer where he led Strategic Portfolio Management for the company’s worldwide R&D organization and oversaw a portfolio spanning from discovery to Phase 2 across all major therapeutic areas. He has held leadership positions in multiple biotech companies, including Ascendis Pharma and Maxygen. Straight Nissen currently serves on the board of Axcella Health and is the founding CEO of FL84. He is also a co-author or co-inventor of over 100 publications and published patents.

Company Update

In October, the company stopped Phase 1 clinical studies of its autologous multi-targeted T cell therapeutic candidates, RPTR-147 and RPTR-168 in human papillomavirus (HPV) positive tumors. This followed an assessment of recent data which showed that while stable disease was observed in a small group of patients, this effect was limited and does not support advancing these programs.

The company plans to apply the translational insights from these studies to inform its ongoing development of novel treatments for solid tumors. These changes reposition the company as a pre-clinical organization with reduced operational needs, and as a result, the company reduced its staff earlier this month.

Going forward, Repertoire will focus on unlocking the potential of its proprietary DECODE platform to develop transformative medicines against novel immune targets in cancer, autoimmune disorders and infectious disease.

About the DECODE Platform

The DECODE platform is a powerful discovery engine that characterizes essential elements of the immune synapse. In particular, the platform identifies T cell receptor-antigen pairs in the context of other important features of the immune synapse, such as T cell function and how antigens are presented by molecules on antigen-presenting cells, known as major human leukocyte antigen, or HLA, molecules. Repertoire intends to utilize these insights into key drivers that govern immune function to design and develop novel immune product candidates.

On November 7, 2022 FibroGen, Inc. (NASDAQ: FGEN) reported financial results for the third quarter 2022 and provided an update on the company’s recent developments (Press release, FibroGen, NOV 7, 2022, View Source [SID1234623240]).

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"I am very pleased with our progress across our clinical development programs with 3 pivotal trials expected to read out for pamrevlumab in 2023 and two more in 2024. Notably, our Phase 3 trials of roxadustat in the U.S. and Europe for anemia of myelodysplastic syndromes, and chemotherapy-induced anemia in China add two more pivotal readouts next year," said Enrique Conterno, Chief Executive Officer, FibroGen. "The strategic financing transaction with NovaQuest provides additional non-dilutive capital which strengthens our balance sheet to support the development and commercialization of pamrevlumab while continuing to advance and expand our pipeline."

Recent Developments:

Announced non-dilutive royalty monetization transaction with NovaQuest Capital Management secured by 22.5% of roxadustat royalty revenue in the territories licensed to Astellas Pharma Inc., providing $50 million to support our strategic priorities.
Completed enrollment of the MATTERHORN Phase 3 study of roxadustat in patients with anemia of myelodysplastic syndromes (MDS).
Roxadustat continues to gain approvals in additional countries around the world. It is now approved in China, Europe, Japan, and numerous other territories for the treatment of anemia in chronic kidney disease (CKD) patients on dialysis and not on dialysis.
China Performance:

FibroGen’s net product revenue under U.S. GAAP from the sale of roxadustat in China was $17.4 million compared to $13.4 million in the third quarter of 2021, an increase of 29%.
Third quarter total roxadustat net sales in China1 by FibroGen and the distribution entity (JDE) jointly owned by FibroGen, and AstraZeneca was $59.0 million, compared to $57.8 million in the third quarter of 2021.
Roxadustat continues to be the number one brand based on value share in the anemia of CKD market in China.
Upcoming Milestones:

Pamrevlumab

Topline data from the LELANTOS-1 Phase 3 study of pamrevlumab in non-ambulatory DMD patients expected 1H 2023.
Topline data from the ZEPHYRUS-1 Phase 3 study of pamrevlumab in IPF expected mid-2023.
Topline data from the LELANTOS-2 Phase 3 study of pamrevlumab in ambulatory DMD patients expected 2H 2023.
Topline data from the LAPIS Phase 3 study of pamrevlumab in LAPC expected 1H 2024.
Topline data from the ZEPHYRUS-2 Phase 3 study of pamrevlumab in IPF expected mid-2024.
Roxadustat

Topline data from the MATTERHORN Phase 3 study of roxadustat in anemia of MDS expected 1H 2023.
Topline data from the China Phase 3 study of roxadustat for the treatment of chemotherapy-induced anemia (CIA) expected mid-2023.
Financial:

Total revenue for the third quarter of 2022 was $15.7 million, as compared to $156.0 million for the third quarter of 2021, which included $120 million of milestone payments from Astellas related to the EU approval of roxadustat.
Net loss for the third quarter of 2022 was $91.7 million, or $0.98 net loss per basic and diluted share, compared to a net income of $49.8 million, or $0.54 net income per basic and diluted share one year ago.
At September 30, 2022, FibroGen had $441.6 million in cash – defined as cash, cash equivalents, investments, and accounts receivable.
Today we announced a non-dilutive royalty monetization transaction with NovaQuest Capital Management, providing $50 million to support our strategic priorities.
After this transaction and based on our latest forecast, we anticipate our 2022 ending cash balance to be $380-$410 million.
_________________________

1 Total roxadustat net sales in China includes sales made by the distribution entity as well as FibroGen China’s direct sales, each to its own distributors. The distribution entity jointly owned by AstraZeneca and FibroGen is not consolidated into FibroGen’s financial statements.

Conference Call and Webcast Details
FibroGen will host a conference call and webcast today, Monday, November 7, 2022, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time) to discuss financial results and provide a business update. A live audio webcast of the call may be accessed in the investor section of the Company’s website, www.fibrogen.com. To access the call by phone, please go to this link (registration link), and you will be provided with dial-in details. To avoid delays, we encourage participants to dial into the conference call fifteen minutes ahead of the scheduled start time. A replay of the webcast will also be available for a limited time at the following link (webcast replay).

About Pamrevlumab
Pamrevlumab is a potential first-in-class antibody being developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Pamrevlumab is in Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF), locally advanced unresectable pancreatic cancer (LAPC), and Duchenne muscular dystrophy (DMD), and in Phase 2/3 for the treatment of metastatic pancreatic cancer. The U.S. Food and Drug Administration has granted Orphan Drug Designation (ODD), and Fast Track designation to pamrevlumab for the treatment of patients with IPF, DMD, and LAPC. The U.S. Food and Drug Administration has also granted Rare Pediatric Disease Designation to pamrevlumab for the treatment of patients with DMD. Pamrevlumab has demonstrated a safety and tolerability profile that has supported ongoing clinical investigation in IPF, DMD, and LAPC. Pamrevlumab is an investigational drug and not approved for marketing by any regulatory authority. For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.

About Roxadustat
Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is in clinical development for anemia of chronic kidney disease (CKD) and anemia associated with myelodysplastic syndromes (MDS), and for chemotherapy-induced anemia (CIA).

Roxadustat is approved in China, Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). Several other licensing applications for roxadustat have been submitted by partners, Astellas and AstraZeneca to regulatory authorities across the globe, and are currently under review.

Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in territories including Japan, Europe, Turkey, Russia and the Commonwealth of Independent States, the Middle East, and South Africa. FibroGen and AstraZeneca are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in the U.S., China, and other markets not licensed to Astellas.

On November 7, 2022 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for patients with cancer, reported that ONO Pharmaceutical Co., Ltd. (ONO) has exercised its option to FT825/ONO-8250, a multiplexed-engineered, iPSC-derived, chimeric antigen receptor (CAR) T-cell product candidate targeting human epidermal growth factor receptor 2 (HER2)-expressing solid tumors (Press release, Fate Therapeutics, NOV 7, 2022, View Source [SID1234623239]). The preclinical product candidate incorporates multiple functional elements to enhance the activity and overcome unique challenges in treating solid tumors with cell-based cancer immunotherapies.

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"We are encouraged by the compelling preclinical data package generated for FT825/ONO-8250 under our collaboration, which combines the antigen binder that ONO provides and Fate’s industry-leading iPSC product platform to overcome the challenges in solid cancer treatment," said Toichi Takino, Senior Executive Officer / Executive Director, Discovery & Research of ONO. "We look forward to initiating clinical development of the off-the-shelf, iPSC-derived CAR T-cell product candidate with the aim of delivering benefit to patients with some of the most difficult to treat cancers."

Under the terms of the Collaboration and Option Agreement, Fate will receive a milestone payment in connection with ONO’s exercise of its option to FT825/ONO-8250. The parties will jointly develop and commercialize FT825/ONO-8250 in the U.S. and Europe, and ONO maintains exclusive development and commercialization rights for FT825/ONO-8250 in the rest of the world. Fate is eligible to receive clinical, regulatory and commercial milestone payments as well as tiered royalties on net sales outside of the United States and Europe by ONO. The parties recently expanded their collaboration to initiate preclinical development of an additional program targeting a second solid tumor antigen.

"Over the past four years, we have worked closely with ONO to discover and integrate novel functional elements into our iPSC-derived CAR T-cell product platform that are specifically designed to address challenges in treating solid tumors, including cell trafficking and immune cell suppression in the tumor microenvironment," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "The preclinical data indicate FT825/ONO-8250 has a highly-differentiated therapeutic profile, including exhibiting anti-tumor activity against HER2-low tumor cells. We are excited to initiate IND-enabling activities under our collaboration with ONO with the goal of submitting an IND application to FDA in 2023."

Although CAR T-cell therapy has shown significant efficacy in treating hematologic malignancies, its wider application to solid tumors has been hampered by tumor-associated antigen heterogeneity, inefficient CAR T-cell trafficking to the tumor, and immunosuppression inherent to the tumor microenvironment. The Company’s multiplexed-engineered, iPSC-derived CAR T-cell product platform is designed to specifically address these challenges and enable the safe and effective treatment of solid tumors as monotherapy and in combination with monoclonal antibody therapy. At the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 37th Annual Meeting to be held from November 8-12, 2022 in Boston, the Company is presenting a poster presentation of FT825/ONO-8250 entitled "Off-the-shelf iPSC-derived CAR-T cells containing seven functional edits overcome antigen heterogeneity, improve trafficking and withstand immunosuppression associated with failed tumor treatment" (Abstract ID: 304; November 11, 2022, 9:00 AM – 9:00 PM), which highlights the incorporation of a synthetic CXCR2 receptor to promote cell trafficking, a synthetic TGFβ receptor to redirect immunosuppressive signals in the tumor microenvironment, and a synthetic interleukin-7 receptor fusion protein to induce T-cell activation into its iPSC-derived CAR T-cell product platform.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that are designed to be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 350 issued patents and 150 pending patent applications.

On November 7, 2022 EpiAxis Therapeutics reported that it has signed a material transfer agreement (MTA) with Seattle Children’s Research Institute that will help researchers study a new approach to treating paediatric brain cancer (Press release, EpiAxis Therapeutics, NOV 7, 2022, View Source;utm_medium=rss&utm_campaign=epiaxis-enters-agreement-with-seattle-childrens-research-institute [SID1234623238]).

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As part of the agreement, EpiAxis Therapeutics will supply its lead peptide candidates for assessment in the laboratory of Dr Myron Evans II, principal investigator at the Ben Towne Center for Childhood
Cancer Research at Seattle Children’s Research Institute and assistant professor in the Department of Pediatrics at the University of Washington School of Medicine, with the goal of informing a new
wave of brain tumour treatments that are more targeted than current therapies, with fewer side effects.

"Using novel inhibitors against LSD1 as a treatment for paediatric brain tumours has the potential to enhance efficacy, which could bypass a number of problems associated with existing chemotherapy
and radiotherapy," said EpiAxis Therapeutics CEO Dr. Jeremy Chrisp.

"Our first-in-class assets at EpiAxis Therapeutics have dual action and target dormant chemotherapy resistant cells while also improving the immune response against the persisting cells."EpiAxis is very pleased to be supplying our lead peptides candidates to Seattle Children’s in hope of accelerating innovation in the treatment of paediatric brain cancer."

Dr Evans II will be leading the project utilising EpiAxis’ lead peptide candidates. His research program focuses on epigenetic regulation in central nervous system development and how
deregulation contributes to paediatric brain tumours.

"For paediatric tumours, deregulation of normal epigenetic processes is central to tumour formation and provides novel avenues for targeted cancer therapy" said Dr Evans II.

"We are excited to work with EpiAxis to evaluate their peptides in the lab, and hopefully pursue them as novel therapies for children."