On February 27, 2024 Adaptimmune Therapeutics plc (NASDAQ: ADAP), a company redefining the treatment of solid tumor cancers with cell therapy, reported the reappointment of Cintia Piccina as Chief Commercial Officer effective March 18, 2024 (Press release, Adaptimmune, FEB 27, 2024, View Source [SID1234640504]). Cintia served in this role from January 2022 to March 2023. Cintia will lead a rapidly expanding commercial team as it prepares for the launch of afami-cel for the treatment of advanced synovial sarcoma which, on approval, will be the first engineered TCR T-cell therapy on the market targeting solid tumors.

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Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer: "It is my pleasure and the Company’s great fortune to welcome back Cintia and her wealth of cell therapy commercial expertise. With a priority review and an August 4, 2024 PDUFA date set for afami-cel, we have the opportunity to leverage Cintia’s established track record and her familiarity with Adaptimmune, our products, and the company culture. Cintia was instrumental in designing and steering our commercial model design and her leadership will be critical as we continue launch planning and execution for the late-stage products in our sarcoma franchise."

Cintia Piccina, Adaptimmune’s incoming Chief Commercial Officer: "I am thrilled to have the opportunity to return to Adaptimmune as we prepare for the launch and commercialization of afami-cel. I look forward to joining this incredible team again as we aim to deliver important and much-needed new treatment options for people diagnosed with advanced cancers."

Most recently, Cintia served as Chief Commercial Officer at AlloVir (NASDAQ: ALVR) helping to build the company’s commercialization capabilities and team to support the launch of the first allogeneic multi-Viral Specific T-cell (VST) therapy, AlloVir’s anticipated first commercial product. Prior to her time at Adaptimmune, Cintia served as SVP Commercial Oncology and US General Manager at Bluebird Bio (NASDAQ: BLBD)/2seventy Bio (NASDAQ: TSVT), leading the launch of the first cell therapy product in multiple myeloma, Abecma (idecabtagene vicleucel). Before that, she spent more than 20 years at Novartis (SIX:NOVN; NYSE:NVS) from 1997 to April 2020, first in Brazil then in the United States, where she held a series of commercial, marketing, and sales roles across multiple therapeutic areas including oncology. In her final role at Novartis, Cintia was VP, Global Oncology Cell and Gene Strategy & Program Management Office, for Kymriah and the CAR-T pipeline, leading the cross-functional leadership teams for business (marketing, medical affairs, market access), manufacturing, and pipeline. Cintia holds a Doctorate in Pharmacy and Biochemistry from the University of Sao Paulo, Brazil, and an MBA from the Escola Superior de Propaganda e Marketing, Sao Paulo.

On January 31, 2024, Adaptimmune announced that the U.S. Food and Drug Administration (FDA) had accepted for priority review its Biologics License Application (BLA) for afami-cel, an investigational engineered T-cell therapy for advanced synovial sarcoma. The application has a PDUFA target action date of August 4, 2024.

About Afami-cel

Afami-cel is an engineered T-cell receptor (TCR) T-cell therapy, targeted to the MAGE A4 cancer target, and designed as a single-dose treatment for advanced synovial sarcoma. The last FDA approved therapy for treatment in this setting was for Votrient in 2012. The BLA submission for afami-cel was supported by clinical data from the SPEARHEAD-1 pivotal trial, which has met its primary endpoint for efficacy. ~39% of patients who received afami-cel had clinical responses with a median duration of response of ~12 months (CTOS 2022). Median overall survival (mOS) was ~17 months in SPEARHEAD-1 compared to historical mOS of <12 months for people with synovial sarcoma who received two or more prior lines of therapy. Seventy percent of people with advanced synovial sarcoma who respond to afami-cel are alive two years post-treatment.

On February 27, 2024 Adagene Inc. ("Adagene") (Nasdaq: ADAG), a company transforming the discovery and development of novel antibody-based therapies, reported its participation in the Leerink Partners Global Biopharma Conference 2024, taking place March 11-13 in Miami, Florida (Press release, Adagene, FEB 27, 2024, View Source [SID1234640503]).

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Adagene’s Chairman, Chief Executive Officer and President of R&D, Peter Luo, Ph.D., will provide an update on its masked, anti-CTLA-4 SAFEbody ADG126, including key milestones for 2024. Company management will also host investor meetings.

Leerink Partners Global Biopharma Conference 2024

Date:
Tuesday, March 12
Presentation Time:
1:40 PM (Eastern Time)
Location:
The Fontainebleau Miami
A live webcast of the presentation will also be accessible in the Investors section of the company’s website at View Source A webcast replay will be available for at least 30 days.

On February 27, 2024 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the fourth quarter and full year of 2023 and provided a corporate update (Press release, Aclaris Therapeutics, FEB 27, 2024, View Source [SID1234640502]).

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"As we enter 2024, we are financially strong, focused and motivated," stated Dr. Neal Walker, co-founder and Interim Chief Executive Officer & President of Aclaris. "Returning to the CEO role, I look forward to building on our strong foundation and deep expertise in kinase discovery and development as we look to shape the future of Aclaris."

Research and Development Highlights:

ATI-1777, an investigational topical "soft" JAK 1/3 inhibitor
In January 2024, Aclaris reported positive top-line results from its Phase 2b trial in atopic dermatitis, and is currently seeking a development and commercialization partner for this program.
ATI-2138, an investigational oral covalent ITK/JAK3 inhibitor
Aclaris is assessing the most effective development pathway, including the lead indication, for ATI-2138. Aclaris reported positive results from its Phase 1 MAD trial of ATI-2138 in September 2023.
Zunsemetinib (ATI-450), an investigational oral small molecule MK2 inhibitor
Aclaris plans to support Washington University in St. Louis in its investigator-initiated Phase 1b/2 trials of zunsemetinib as a potential treatment for pancreatic cancer and metastatic breast cancer. Aclaris expects these trials to be primarily funded by grants awarded to Washington University.
ATI-2231, Aclaris’ second MK2 inhibitor, was previously being developed for oncology and Aclaris was supporting Washington University in an investigator-initiated Phase 1a trial of ATI-2231 in patients with advanced solid tumor malignancies. However, Aclaris and Washington University agreed to instead study zunsemetinib in oncology in order to expedite the development timeline by eliminating the need to conduct the Phase 1a trial due to zunsemetinib’s more advanced clinical package.

Discovery

Aclaris plans to continue to advance discovery programs through KINect, its proprietary drug discovery platform.
Financial Highlights:

Liquidity and Capital Resources

As of December 31, 2023, Aclaris had aggregate cash, cash equivalents and marketable securities of $181.9 million compared to $229.8 million as of December 31, 2022.

Financial Results

Fourth Quarter 2023

Net loss was $1.5 million for the fourth quarter of 2023 compared to $27.6 million for the fourth quarter of 2022.

Total revenue was $17.6 million for the fourth quarter of 2023 compared to $7.8 million for the fourth quarter of 2022. The increase was primarily driven by a one-time upfront payment under the license agreement with Sun Pharmaceutical Industries, Inc. (Sun Pharma) received in the fourth quarter of 2023.

Research and development (R&D) expenses were $26.6 million for the quarter ended December 31, 2023 compared to $21.1 million for the prior year period. The $5.5 million increase was primarily the result of an increase in expenses associated with drug candidate manufacturing for zunsemetinib.

General and administrative (G&A) expenses were $8.2 million for the quarter ended December 31, 2023 compared to $7.1 million for the corresponding prior year period. The increase was primarily due to an increase in personnel and stock-based compensation expenses.

Licensing expenses were $5.7 million for the quarter ended December 31, 2023 compared to $0.6 million for the prior year period. The increase was primarily attributable to amounts payable to third parties in connection with amounts earned under the Sun Pharma license agreement.

Revaluation of contingent consideration resulted in a $26.3 million gain for the quarter ended December 31, 2023 compared to a charge of $7.1 million for the prior year period.

Intangible asset impairment charges were $6.6 million for the quarter ended December 31, 2023, representing the full balance of the in-process research and development (IPR&D) intangible asset. The impairment charge resulted from Aclaris’ decision to discontinue further development of the drug candidate for immuno-inflammatory diseases.
Full Year 2023

Net loss was $88.5 million for the year ended December 31, 2023 compared to $86.9 million for the year ended December 31, 2022.

Total revenue was $31.2 million for the year ended December 31, 2023 compared to $29.8 million for the year ended December 31, 2022. The increase was primarily driven by a one-time upfront payment under the license agreement with Sun Pharma received in the year ended December 31, 2023. The increase was partially offset by both a one-time upfront payment received under a license agreement with Eli Lilly and Company and a one-time upfront payment received under a license agreement with Pediatrix Therapeutics, Inc. in the year ended December 31, 2022.

R&D expenses were $98.4 million for the year ended December 31, 2023 compared to $77.8 million for the prior year period.

The $20.6 million increase was primarily the result of higher:
Zunsemetinib development expenses, including costs associated with clinical activities for the Phase 2b trial for rheumatoid arthritis and drug candidate manufacturing costs;
ATI-2138 development expenses, including costs associated with the Phase 1 MAD trial and other preclinical activities; and
Compensation-related expenses due to an increase in headcount.

G&A expenses were $32.4 million for the year ended December 31, 2023 compared to $25.1 million for the prior year period.

The $7.3 million increase was primarily the result of higher compensation-related costs due to increased headcount and the impact of equity awards granted during the year ended December 31, 2023.
Bad debt expense recorded from Aclaris’ determination that collection of amounts due from EPI Health are uncertain as a result of their filing for Chapter 11 bankruptcy protection also contributed to the increase.

Revaluation of contingent consideration resulted in a $26.9 million gain for the year ended December 31, 2023 compared to a charge of $4.7 million for the corresponding prior year period.

Intangible asset impairment charges were $6.6 million for the year ended December 31, 2023, representing the full balance of the IPR&D intangible asset. The impairment charge resulted from Aclaris’ decision to discontinue further development of the drug candidate for immuno-inflammatory diseases.

On February 27, 2024 Genmab A/S (Nasdaq: GMAB) and AbbVie (NYSE: ABBV) reported the U.S. Food and Drug Administration (FDA) granted Priority Review for the supplemental Biologics License Application (sBLA) for epcoritamab-bysp, a T-cell engaging bispecific antibody administered subcutaneously, for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy (Press release, Genmab, FEB 27, 2024, View Source [SID1234640450]).

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The FDA grants Priority Review to investigational therapies that, if approved, may offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. This designation shortens the review period to six months compared to 10 months for Standard Review.i The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 28, 2024.

"While treatment for patients with relapsed and refractory follicular lymphoma has progressed, there remains an urgent need for new treatment options, particularly for patients who are considered difficult to treat due to relapse following standard therapies and other poor prognostic factors," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab. "The acceptance of the epcoritamab application for Priority Review marks an important milestone toward potentially providing a new treatment option to patients affected by R/R follicular lymphoma. Together with AbbVie, we look forward to working with the FDA during the review and remain committed to developing epcoritamab as a potential future core therapy for B-cell malignancies."

The sBLA is based on results from the Phase 1/2 EPCORE NHL-1 clinical trial, which demonstrated high overall and complete responses in patients with R/R FL treated with epcoritamab. Data from the FL cohort of the trial were presented at the Annual Meeting and Exposition of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2023. The FDA previously granted Breakthrough Therapy Designation (BTD) to epcoritamab for the treatment of adult patients with R/R FL after two or more lines of systemic therapy. The application for BTD included additional data from the dose optimization part of EPCORE NHL-1.

Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ oncology collaboration. The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization.

About the Phase 1/2 EPCORE NHL-1 Trial
EPCORE NHL-1 is an open-label, multi-center safety and preliminary efficacy trial of epcoritamab that consists of three parts: a dose escalation part; an expansion part; and an optimization part. The trial was designed to evaluate subcutaneous epcoritamab in patients with relapsed, progressive or refractory CD20+ mature B-cell non-Hodgkin’s lymphoma (B-NHL), including FL. In the expansion part, additional patients were enrolled to further explore the safety and efficacy of epcoritamab in three cohorts of patients with different types of relapsed/refractory B-NHLs who have limited therapeutic options. The optimization part evaluates the potential for alternative step-up dosing regimens to help further minimize Grade 2 cytokine release syndrome (CRS) and mitigate Grade ≥3 CRS. The primary endpoint of the expansion part was ORR as assessed by an IRC. Secondary efficacy endpoints included duration of response, complete response rate, duration of complete response, progression-free survival, and time to response as determined by the Lugano criteria. Overall survival, time to next therapy, and rate of minimal residual disease negativity were also evaluated as secondary efficacy endpoints. The primary endpoint of the optimization part was the rate of ≥ Grade 2 CRS events and all grade CRS events from first dose of epcoritamab through 7 days following administration of the second full dose of epcoritamab.

About Follicular Lymphoma (FL)
FL is typically an indolent (or slow-growing) form of non-Hodgkin’s lymphoma (NHL) that arises from B-lymphocytes.ii FL is the second most common form of NHL overall, accounting for 20-30 percent of all NHL cases, and represents 10-20 percent of all lymphomas in the western world.iii,iv Although FL is an indolent lymphoma, it is considered incurable with conventional therapy and patients who achieve remission also often experience relapse.v,vi,vii Additionally, with each relapse the remission and time to next treatment is shorterviii, adding increased cost to the health system and negatively impacting the patient’s quality of life.ix

About Epcoritamab
Epcoritamab is an IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology and administered subcutaneously. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells.x

Epcoritamab (approved under the brand name EPKINLY in the U.S. and Japan, and TEPKINLY in the EU) has received regulatory approval in certain lymphoma indications in several territories. Use of epcoritamab in FL is not approved in the U.S. or in the EU or in any other territory. Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ oncology collaboration. The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization.

Genmab and AbbVie continue to evaluate the use of epcoritamab as a monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies. This includes three ongoing phase 3, open-label, randomized trials including a trial evaluating epcoritamab as a monotherapy in patients with R/R DLBCL (NCT: 04628494) compared to investigators choice chemotherapy, a phase 3 trial evaluating epcoritamab in combination with R-CHOP in adult participants with newly diagnosed DLBCL (NCT: 05578976), and a phase 3, open-label clinical trial evaluating epcoritamab in combination with rituximab and lenalidomide in patients with R/R FL (NCT: 05409066). Epcoritamab is not approved to treat newly diagnosed patients with DLBCL or FL. The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit clinicaltrials.gov for more information.

On February 26, 2024 Lirum Therapeutics, Inc. ("Lirum"), an innovative clinical-stage biopharmaceutical company focused on the treatment of debilitating diseases, reported that it will present positive data on LX-101, a novel clinical-stage targeted therapy directed to the insulin growth factor-1 receptor (IGF-1R), in patients with high IGF-1R tumor expression at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Targeted Anticancer Therapies Congress in Paris, France on Monday, February 26th at 5:15 PM CET (11:15 ET) (Press release, Lirum Therapeutics, FEB 26, 2024, View Source [SID1234651604]).

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Lirum’s presentation "LX-101, a novel, clinical-stage, payload-bearing IGF-1R targeted therapy demonstrates activity in patients with high IGF-1R tumor expression" will be presented in the Hall Bordeaux at the Palais des Congrès. The poster presentation covers patient outcomes in the previous Phase 1a studies with LX-101, with a focus on those patients that we determined to have high IGF-1R tumor expression. Of the 19 patients treated with LX-101, 4 were considered high IGF-1R expressers. Of these 4 patients, 3 were evaluable for response, and 2/3 (67%) of these patients experienced disease control/response, including the one patient who had an objective response at the highest dose. The poster presentation is available on the Lirum website (www.lirumtx.com) under the Investors and Media tab.

Given the promising results, including that patients with high IGF-1R expressing tumors can be clinically sensitive to LX-101, Lirum is planning new clinical trials with LX-101 focused on oncologic indications, both pediatric and adult, that carry well-established ties to the IGF-1/IGF-1R pathway. Lirum is also developing LX-101 in certain autoimmune diseases, including thyroid dye disease, where IGF-1R has been clinically and commercially validated.