On August 8, 2024 Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, reported financial results and business highlights for the second quarter of 2024 (Press release, Geron, AUG 8, 2024, View Source [SID1234645600]).

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!

"We are thrilled to have begun the launch of RYTELO, our first commercial product, in June, and are encouraged by the early success we are seeing and the reception from the medical community over these first six weeks," said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. "Our field teams have mobilized efficiently and have already interacted with approximately 60% of our top decile 1-4 accounts across the community and academic settings. This has contributed to gratifying uptake – as of July 31, 2024, we estimate that approximately 160 patients have received RYTELO, which is encouraging given the very early stage of this launch. Further, the MDS NCCN Guidelines, which guide clinical decision-making, prescriber behavior and reimbursement decisions, were updated at the end of July to include RYTELO as a Category 1 and 2A treatment of symptomatic anemia in patients with lower-risk MDS. With the introduction of RYTELO as a new therapeutic option, we are seeing increasing dialogue among hematologists rethinking treatment approaches for eligible patients with lower-risk MDS with transfusion-dependent anemia, regardless of ring sideroblast status, and we believe that RYTELO can become part of the standard-of-care for these patients."

Business Highlights

Received approval on June 6, 2024 from the U.S. Food & Drug Administration (FDA) of RYTELO for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent (TD) anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs).
Launched RYTELO commercially in the U.S., with both dosage strengths available for prescribers to order from specialty distributors as of June 27, 2024.
The National Comprehensive Cancer Network (NCCN) updated the MDS NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) to include RYTELO for the treatment of symptomatic anemia in patients with LR-MDS. For both RS+ and RS- patients, RYTELO has a Category 1 designation for second-line treatment regardless of first-line treatment and a Category 2A designation for first-line treatment of patients who are ESA ineligible (serum EPO >500 mU/mL).
Achieved approximately 70% enrollment in the Phase 3 IMpactMF trial of imetelstat as of August 2024, which has a primary endpoint of overall survival, in patients with myelofibrosis (MF) who are relapsed/refractory to JAK-inhibitors. As previously disclosed, based on the most recent planning assumptions for enrollment and death rates in the trial, an interim analysis is expected in early 2026 (when approximately 35% of planned enrolled patients have died) and final analysis is expected in early 2027 (when approximately 50% of planned enrolled patients have died).
Expanded the dose level 4 cohort (imetelstat 9.4 mg/kg) in the Part 1 dose-finding stage of the Phase 1 IMproveMF study evaluating imetelstat as a combination therapy with ruxolitinib in patients with frontline MF. This followed a unanimous decision by the study’s Safety Evaluation Team (SET), who in July 2024 reviewed data from the first three patients in the dose level 4 cohort and identified no dose-limiting toxicities.
Second Quarter 2024 Financial Results

As of June 30, 2024 the Company had approximately $430.4 million in cash, cash and equivalents and marketable securities.

Net Loss

For the three and six months ended June 30, 2024, the Company reported a net loss of $67.4 million, or $0.10 per share, and $122.8 million, or $0.19 per share, respectively, compared to $49.2 million, or $0.09 per share and $87.3 million, or $0.16 per share, respectively, for the three and six months ended June 30, 2023.

Revenues

Total product revenue, net for the three and six months ended June 30, 2024, was approximately $780,000.

Total net revenues for the three and six months ended June 30, 2024, were $882,000 and $1.2 million, compared to $29,000 and $50,000 for the same periods in 2023. The increase in revenue is due to product revenue from U.S. sales of RYTELO, which was available for prescribers to order from specialty distributors as of June 27, 2024.

Operating Expenses

Total operating expenses for the three and six months ended June 30, 2024 were $70.2 million and $126.7 million, compared to $52.0 million and $92.1 million for the same periods in 2023.

Cost of goods sold was approximately $17,000 for the three and six months ended June 30, 2024, which consisted of costs to manufacture and distribute RYTELO.

Research and development expenses for the three months and six months ended June 30, 2024 were $30.8 million and $60.2 million, respectively, and $35.5 million and $62.7 million, for the same periods in 2023. The decrease is primarily due to IMerge Phase 3 operations moving into the long-term follow-up stage.

Selling, general and administrative expenses for the three and six months ended June 30, 2024 were $39.4 million, and $66.5 million, respectively, and $16.5 million and $29.4 million for the same periods in 2023. The increase in selling, general and administrative expenses primarily reflects higher commercial launch expenses, increases in headcount and stock-based compensation expense recognized due to the vesting of performance-based stock options upon FDA approval of RYTELO.

Interest income was $5.3 million and $9.6 million for the three and six months ended June 30, 2024, compared to $4.7 million and $8.6 million for the same periods in 2023. The increase in interest income primarily reflects a larger marketable securities portfolio with the receipt of net cash proceeds from the underwritten offering completed in March 2024, as well as higher yields from recent marketable securities purchases.

Interest expense was $3.3 million and $6.8 million for the three and six months ended June 30, 2024, compared to $2.0 million and $3.9 million for the same periods in 2023. The increase in interest expense primarily reflects rising interest rates. Currently, we have $80.0 million in principal debt outstanding. Interest expense reflects interest owed under the loan agreement, as well as amortization of associated debt issuance costs and debt discounts using the effective interest method and accrual for an end of term charge.

2024 Financial Guidance

For fiscal year 2024, we expect total operating expenses to be in the range of approximately $270 million to $280 million, which includes non-cash items such as stock-based compensation expense, amortization of debt discounts and issuance costs, and depreciation and amortization.

Based on our current operating plans and assumptions, we believe that our existing cash, cash equivalents, and marketable securities, together with projected revenues from U.S. sales of RYTELO, will be sufficient to fund our projected operating requirements into the second quarter of 2026.

As of June 30, 2024, we had 220 full-time employees. We plan to grow to a total of approximately 230-260 employees by year-end 2024.

Conference Call

Geron will host a conference call at 8:00 a.m. ET on Thursday, August 8, 2024, to discuss business updates and second quarter financial results.

A live webcast of the conference call and related presentation will be available on the Company’s website at www.geron.com/investors/events . An archive of the webcast will be available on the Company’s website for 30 days.

Participants may access the webcast by registering online using the following link, View Source

About RYTELO (imetelstat)

RYTELO (imetelstat) is an FDA-approved oligonucleotide telomerase inhibitor for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). It is indicated to be administered as an intravenous infusion over two hours every four weeks.

RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration.

Geron aims to ensure broad access to RYTELO for eligible patients. Accordingly, our REACH4RYTELO Patient Support Program provides a range of resources that support access and affordability to eligible patients prescribed RYTELO.

On August 8, 2024, Genmab reported interim Report for the First Six Months Ended June 30, 2024 (Press release, Genmab, AUG 8, 2024, View Source [SID1234645599]).

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!

Highlights

Completed acquisition of ProfoundBio Inc (ProfoundBio), granting Genmab worldwide rights to three candidates in clinical development, including rinatabart sesutecan (Rina-S), plus ProfoundBio’s novel antibody-drug conjugate technology platforms
The U.S. Food and Drug Administration (U.S. FDA) approved EPKINLY (epcoritamab-bysp) for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the granting of conditional marketing authorization of TEPKINLY (epcoritamab) for the treatment of adult patients with relapsed or refractory FL after two or more lines of systemic therapy
Tivdak (tisotumab vedotin-tftv) received full U.S. FDA approval to treat recurrent or metastatic cervical cancer
Genmab submitted a Japan New Drug Application (J-NDA) to the Ministry of Health, Labor and Welfare (MHLW) in Japan for Tivdak (tisotumab vedotin) for the treatment of adult patients with advanced or recurrent cervical cancer that has progressed on or after chemotherapy
Genmab revenue increased 36% compared to the first six months of 2023, to DKK 9,545 million
Genmab 2024 financial guidance updated
"In the second quarter of 2024, we reached a number of significant milestones for the company. The acquisition of ProfoundBio, along with the regulatory approvals for EPKINLY and Tivdak, further solidify our commitment to the development of differentiated antibody therapies and will advance Genmab towards our ambitious 2030 vision of transforming the lives of patients with our innovative antibody medicines," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Financial Performance First Half of 2024

Revenue was DKK 9,545 million for the first six months of 2024 compared to DKK 7,003 million for the first six months of 2023. The increase of DKK 2,542 million, or 36%, was primarily driven by higher DARZALEX (daratumumab) and Kesimpta (ofatumumab) royalties achieved under our collaborations with Janssen Biotech, Inc. (Janssen) and Novartis Pharma AG (Novartis), respectively, and increased EPKINLY net product sales.
Royalty revenue was DKK 7,673 million in the first six months of 2024 compared to DKK 5,886 million in the first six months of 2023, an increase of DKK 1,787 million, or 30%. The increase in royalties was driven by higher net sales of DARZALEX and Kesimpta.
Net sales of DARZALEX, including sales of the subcutaneous (SC) product (daratumumab and hyaluronidase-fihj, sold under the tradename DARZALEX FASPRO in the U.S.), by Janssen were USD 5,570 million in the first six months of 2024 compared to USD 4,695 million in the first six months of 2023, an increase of USD 875 million or 19%.
Total costs and operating expenses were DKK 7,104 million in the first six months of 2024 compared to DKK 5,118 million in the first six months of 2023. The increase of DKK 1,986 million, or 39%, was driven by the expansion of our product pipeline, EPKINLY post-launch activities in the U.S. and Japan, the continued development of Genmab’s broader organizational capabilities and related increase in team members to support these activities, as well as profit-sharing amounts payable to AbbVie Inc. (AbbVie) related to EPKINLY sales.
Operating profit was DKK 2,441 million in the first six months of 2024 compared to DKK 1,885 million in the first six months of 2023.
Net financial items resulted in income of DKK 1,402 million for the first six months of 2024 compared to DKK 75 million in the first six months of 2023. The increase of DKK 1,327 million was primarily driven by movements in USD to DKK foreign exchange rates impacting Genmab’s USD denominated cash and cash equivalents and marketable securities, with strengthening of the USD/DKK rate in the first six months of 2024 compared to the weakening of the USD/DKK rate in the first six months of 2023.
Significant Event Post-quarter End

August: Genmab announced that it will assume sole responsibility for the continued development and potential commercialization of acasunlimab. BioNTech SE (BioNTech) has opted not to participate in the further development of the acasunlimab program under the parties’ existing collaboration agreement. The program will be subject to payment of certain milestones and a tiered single-digit royalty on net sales by Genmab to BioNTech. While the emerging clinical profile of acasunlimab is encouraging, BioNTech informed the company that it has taken this decision for reasons relating to its portfolio strategy. The companies’ long-standing collaboration in antibody science remains in place, and both parties will continue with the existing programs under development under their existing agreements, which were expanded in 2022.
Outlook
As announced in Company Announcement No. 52, Genmab is updating its 2024 financial guidance.

Revised Revised
Guidance Guidance
(DKK million) ex. Acquisition and Integration related charges incl. Acquisition and Integration related charges Previous Guidance
Revenue 20,500 – 21,700 20,500 – 21,700 18,700 – 20,500
Royalties 16,600 – 17,400 16,600 – 17,400 15,600 – 16,700
Net product sales/Collaboration revenue* 2,000 – 2,200 2,000 – 2,200 1,700 – 2,200
Milestones/Reimbursement revenue 1,900 – 2,100 1,900 – 2,100 1,400 – 1,600
Gross profit** 19,600 – 20,800 19,600 – 20,800 18,000 – 19,500
Operating expenses** (13,700) – (14,300) (14,100) – (14,700) (12,400) – (13,400)
Operating profit 5,300 – 7,100 4,900 – 6,700 4,600 – 7,100

*Net Product Sales and Collaboration Revenue consists of EPKINLY Net Product Sales in the U.S. and Japan and Tivdak (Genmab’s share of net profits) in the U.S.
**Operating Expenses Range excludes Cost of Product Sales Range, which is included in Gross Profit Range

Conference Call
Genmab will hold a conference call to discuss the results for the first half of 2024 today, August 8, 2024, at 6:00 pm CEST, 5:00 pm BST or 12:00 pm EDT. To join the call please use the below registration link. Registered participants will receive an email with a link to access dial-in information as well as a unique personal PIN: https://register.vevent.com/register/BI61134ed097674233a89964e3bc06a69e. A live and archived webcast of the call and relevant slides will be available at www.genmab.com/investor-relations.

On August 8, 2024 Foghorn Therapeutics Inc. (Nasdaq: FHTX), a clinical-stage biotechnology company pioneering a new class of medicines that treat serious diseases by correcting abnormal gene expression, reported a financial and corporate update in conjunction with the Company’s 10-Q filing for the quarter ended June 30, 2024 (Press release, Foghorn Therapeutics, AUG 8, 2024, View Source [SID1234645598]). With an initial focus in oncology, Foghorn’s Gene Traffic Control Platform and resulting broad pipeline have the potential to transform the lives of people suffering from a wide spectrum of 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!

"We anticipate topline data from our Phase 1 combination trial with FHD-286 in patients with relapsed and/or refractory AML in the fourth quarter of 2024. We believe FHD-286 has the potential to be a first-in-class, mutation-agnostic differentiation therapeutic," said Adrian Gottschalk, President and Chief Executive Officer of Foghorn. "Additionally, the IND for FHD-909 cleared in May and we anticipate FHD-909 to be the first SMARCA2 selective inhibitor to enter the clinic. Dosing of the first patient in our Phase 1 trial, with primary target population in SMARCA4 mutated NSCLC, is planned for the second half of the year. We are also on track to initiate IND-enabling studies in the fourth quarter of 2024 for our Selective CBP degrader program targeting tumors harboring EP300 mutations including bladder, gastric and endometrial cancers."

Mr. Gottschalk continued, "The biological foundation for the development of therapeutics targeting the chromatin regulatory system in oncology and other disease areas continues to get stronger. In April, we were pleased to present preclinical data at AACR (Free AACR Whitepaper) reinforcing the potential of our platform to deliver innovative medicines across cancers by selectively drugging historically very challenging targets. The conviction in our pipeline was further strengthened by our recent successful financing with new and long-term investors, which extended our expected cash runway into 2027 and through key inflection points, strongly positioning us for continued advancement."

Corporate Updates

Strengthened Balance Sheet to Advance Pipeline. In May, Foghorn successfully closed an approximately $110 million registered direct offering to advance the Company’s pipeline. The offering included new investors BVF Partners, Deerfield Management and other leading healthcare specialist investors as well as current investors, including founding investor Flagship Pioneering.

Presented at AACR (Free AACR Whitepaper) Annual Meeting. In April, Foghorn presented preclinical data highlighting pipeline progress on the advancement of multiple potential first-in-class medicines, including the first presentation of preclinical data for FHD-909, at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

Hosted Chromatin Regulation Summit. In April, Foghorn hosted the first Future of Disease and Chromatin Regulation Summit at the Foghorn corporate headquarters in Cambridge, Massachusetts. The live event featured presentations and panel discussions with world-renowned industry and academic key opinion leaders on therapeutic opportunities in chromatin regulatory biology.

Strengthened Executive Leadership. In April, Foghorn appointed Kristian Humer as Chief Financial Officer. Mr. Humer joined Foghorn with over 14 years of diversified financial strategy and business development experience in the life science industry and more than 20 years of experience in the financial industry.

Key Recent Program Updates and Upcoming Milestones

FHD-286. FHD-286 is a potent, first-in-class, selective inhibitor of the SMARCA2 (BRM) and SMARCA4 (BRG1) subunits of the BAF chromatin remodeling complex where dependency on SMARCA2/SMARCA4 is well-established preclinically with multiple tumor types, including acute myeloid leukemia (AML)/myelodysplastic syndrome (MDS), non-small cell lung cancer (NSCLC) and prostate cancer.
•AML Phase 1 trial. The ongoing Phase 1 dose escalation trial is evaluating the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of FHD-286 in combination with decitabine or low-dose cytarabine in patients with relapsed and/or refractory AML who have failed multiple previous courses of therapy. FHD-286 previously demonstrated a promising mutation-agnostic differentiation effect in a single-agent dose escalation trial.
◦Topline clinical data are anticipated in the fourth quarter. We anticipate this will include topline safety, tolerability, initial efficacy and PK/PD data.

•Overcoming Tyrosine Kinase Inhibitor Resistance. Data published in Cancer Cell in August 2023, together with additional preclinical studies conducted by Foghorn, suggest that FHD-286 may play an important role in overcoming resistance in EGFR/KRAS tumors. Preclinical data profiling the ability of FHD-286 to amplify and/or restore tumor sensitivity will be presented with FHD-286 Phase 1 dose escalation data.

FHD-909. FHD-909 is a first-in-class oral SMARCA2 (BRM) selective inhibitor that has demonstrated in preclinical studies to have high selectivity over its closely related paralog SMARCA4 (BRG1), two proteins that are the catalytic engines across all forms of the BAF complex. SMARCA4 mutations are common across tumor types, including approximately 10% of NSCLC, and result in tumors being dependent on SMARCA2 activity for their survival. Selectively blocking SMARCA2 activity is a promising synthetic lethal strategy intended to induce tumor death while sparing healthy cells.
•In December 2021, Foghorn announced a strategic collaboration with Lilly to create novel oncology medicines. The collaboration includes a U.S. 50/50 co-development and co-commercialization agreement for Foghorn’s Selective SMARCA2 oncology program, which includes a selective inhibitor and a selective degrader, and an additional undisclosed oncology target. In addition, the collaboration includes three discovery programs using Foghorn’s proprietary Gene Traffic Control platform.
•In April 2024, Foghorn and Lilly presented new preclinical data as a poster presentation at the AACR (Free AACR Whitepaper) Annual Meeting and during a pipeline update call demonstrating at tolerable doses high SMARCA2 selectivity and dose-dependent single agent activity in SMARCA4 mutated cancers.
•In May 2024, the investigational new drug (IND) application was approved by the Food and Drug Administration. The primary target patient population is SMARCA4 mutated NSCLC.
•Dosing of the first patient in the Phase 1 trial for FHD-909 is planned to begin in the second half of 2024.

Selective CBP degrader program and Selective EP300 degrader program.
Foghorn is advancing two separate programs targeting either CBP or EP300, paralog histone acetyltransferases with a synthetic lethal relationship in tumor cells. Attempts to selectively drug CBP or EP300 have been challenging due to the high level of similarity between the two proteins. Additionally, dual inhibition of CBP/EP300 has been historically limited by hematopoietic toxicity.

Selective CBP degrader program. In April, Foghorn presented new pharmacodynamic and pharmacokinetic preclinical data at the 2024 AACR (Free AACR Whitepaper) Annual Meeting and during a pipeline update call highlighting:
•Deep and sustained CBP degradation significantly inhibited tumor growth in mouse xenograft solid tumor models.
•Robust monotherapy preclinical anti-tumor activity that was not associated with significant body weight loss, thrombocytopenia or anemia.
•Identification of potent and selective CBP protein degraders with first-in-class potential to address tumors harboring EP300 mutations in many types of cancer, including bladder, gastric and endometrial cancers.
•IND-enabling studies on track to initiate by the fourth quarter of 2024.

Selective EP300 degrader program. In April, Foghorn presented new pharmacodynamic and pharmacokinetic preclinical data at the 2024 AACR (Free AACR Whitepaper) Annual Meeting and during a pipeline update call highlighting:
•Well-tolerated in vivo with no observed decrease in platelet levels, with no effects on megakaryocyte viability at pharmacologically relevant concentrations in ex vivo studies.
•Identification of potent and selective EP300 degraders with anti-tumor activity in prostate and hematological malignancies, including prostate cancer, multiple myeloma and diffuse large B cell lymphoma (DLBCL).

Selective ARID1B degrader program.
ARID1A is the most mutated subunit in the BAF Complex and amongst the most mutated proteins in oncology. These mutations lead to a dependency on ARID1B, in several types of cancer, including ovarian, endometrial, colorectal and bladder. Attempts to selectively drug ARID1B have been challenging because of the high degree of similarity between ARID1A and ARID1B and the fact that ARID1B has no enzymatic activity to target.
•In April, Foghorn presented data at the AACR (Free AACR Whitepaper) Annual Meeting demonstrating potent and selective small molecule binders to ARID1B. The Company is in the process of converting these selective binders into heterobifunctional degraders.

Second Quarter 2024 Financial Highlights

•Collaboration Revenues. Collaboration revenue was $6.9 million for the three months ended June 30, 2024, compared to $5.6 million for the three months ended June 30, 2023. The increase year-over-year was primarily driven by the continued advancement of programs under the Lilly Collaboration Agreement.

•Research and Development Expenses. Research and development expenses were $23.8 million for the three months ended June 30, 2024, compared to $29.2 million for the three months ended June 30, 2023. This decrease was primarily due to lower personnel-related costs and lower development program spend following the shutdown of two clinical studies (FHD-286 in metastatic uveal melanoma and FHD-609 (BRD9 degrader) in synovial sarcoma).

•General and Administrative Expenses. General and administrative expenses were $7.3 million for the three months ended June 30, 2024, compared to $8.4 million for the three months ended June 30, 2023. This decrease was primarily due to lower personnel-related costs.

•Net Loss. Net loss was $23.0 million for the three months ended June 30, 2024, compared to a net loss of $29.5 million for the three months ended June 30, 2023.

•Cash, Cash Equivalents and Marketable Securities. As of June 30, 2024, the Company had $285.2 million in cash, cash equivalents and marketable securities, providing expected cash runway into 2027.

About FHD-286
FHD-286 is a highly potent, first-in-class, selective, allosteric, and orally available small-molecule, enzymatic inhibitor of SMARCA2 (BRM) and SMARCA4 (BRG1), two highly similar proteins that are the ATPases, or the catalytic engines, of the BAF complex, one of the key regulators within the chromatin regulatory system. In preclinical studies, FHD-286 has shown anti-tumor activity across a broad range of malignancies including both hematologic and solid tumors.

About AML
Adult acute myeloid leukemia (AML) is a cancer of the blood and bone marrow and the most common type of acute leukemia in adults. AML is a diverse disease associated with multiple genetic mutations. It is diagnosed in about 20,000 people every year in the United States.

About FHD-909
FHD-909 (LY4050784) is a potent, first-in-class, allosteric and orally available small molecule that selectively inhibits the ATPase activity of SMARCA2 (BRM) over its closely related paralog SMARCA4 (BRG1), two proteins that are the catalytic engines across all forms of the BAF complex, one of the key regulators of the chromatin regulatory system. In preclinical studies, tumors with mutations in SMARCA4 rely on SMARCA2 for BAF function. FHD-909 has shown significant anti-tumor activity across multiple SMARCA4-mutant lung tumor models.

On August 8, 2024 Flamingo Therapeutics ("Flamingo") reported that the PEMDA-HN trial has expanded beyond the United States into the UK and Korea (Press release, Flamingo Therapeutics, AUG 8, 2024, View Source;utm_medium=rss&utm_campaign=flamingo-therapeutics-announces-first-patients-dosed-in-the-united-kingdom-and-korea-in-ongoing-phase-ii-pemda-hn-study-for-head-and-neck-squamous-cell-carcinoma-hnscc [SID1234645597]).

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!

Patient enrollment in the USA started in February 2024 and now the first patients have been dosed in these new geographies. The PEMDA-HN trial is evaluating danvatirsen in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, in patients with recurrent/metastatic head and neck squamous cell carcinoma (HNSCC).

Flamingo’s lead oncology program, danvatirsen, is an antisense oligonucleotide discovered by Ionis that selectively targets STAT3 and has shown clinical activity in HNSCC.

"HNSCC is a difficult-to-treat cancer, with approximately 890,000 new cases each year globally, and accounts for almost 5% of global cancer deaths. Patients are in need of new therapies and we are pleased to have expanded our study beyond the United States," said Andrew Denker, MD PhD, CMO of Flamingo. "This is a significant milestone in the advancement of the PEMDA-HN study, and we are thankful for the support of our study participants, their families and our global collaborators."

Dr. Kevin Harrington, Consultant Oncologist at The Royal Marsden NHS Foundation Trust and Professor of Biological Cancer Therapies at The Institute of Cancer Research, London, commented, "STAT3 blocking using Flamingo’s investigational therapy, danvatirsen, is a potential novel approach to help patients with HNSCC in my clinic. I am pleased that my patients have an opportunity to participate in the PEMDA-HN trial, which I believe may greatly benefit the global oncology field."

PEMDA-HN (NCT05814666) is a multicenter, open-label, randomized study evaluating the efficacy and safety of danvatirsen in combination with pembrolizumab compared with pembrolizumab alone as first-line treatment of patients with recurrent/metastatic HNSCC whose tumor expresses PD-L1. Two-thirds of patients will be randomized to receive danvatirsen and pembrolizumab and one-third will be randomized to receive pembrolizumab alone. The primary endpoint of the study is to determine the overall response rate by RECIST 1.1 as assessed by the investigator. The secondary endpoints will include safety, duration of response, disease control rate, progression free survival and overall survival. More information on PEMDA-HN can be found here.

On August 8, 2024 Evaxion Biotech A/S (NASDAQ: EVAX) ("Evaxion"), a clinical-stage TechBio company specializing in developing AI-Immunology powered vaccines, reported the presentation of one-year clinical efficacy Phase 2 data for its lead compound EVX-01 at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024, taking place in Barcelona, Spain, from September 13-17, 2024 (Press release, Evaxion Biotech, AUG 8, 2024, View Source [SID1234645596]). The EVX-01 vaccine is a personalized therapy currently being assessed in patient with advanced melanoma (skin cancer).

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!

Christian Kanstrup, CEO of Evaxion, comments: "Having our abstract selected for presentation by the ESMO (Free ESMO Whitepaper) Congress 2024 Scientific Committee is a testament to the significant progress and impact of our work in the field of medical oncology. This is one of the most prestigious medical oncology conferences in the world and, as such, a great opportunity for us to make the data available to a large global audience of experts, researchers and collaborators, as well as potential partners. Presenting one-year Phase 2 clinical efficacy data for our lead pipeline candidate is a major milestone for Evaxion and advancing our own high-value programs to key value inflection points is an important part of our strategy."

Evaxion’s innovative approach to develop personalized cancer vaccines builds on its AI-Immunology platform. The vaccines are designed to target the unique genetic makeup of an individual’s tumor and are tailored to the patients’ immune system, potentially enhancing the efficacy of treatment and improving patient outcomes.

Presentation Details:

Abstract Title: Phase 2 study of AI-designed personalized neoantigen cancer vaccine, EVX-01, in combination with pembrolizumab in advanced melanoma
Abstract#: 1084P
Poster#: 2904
Track: Melonama and other skin tumours
Location: Hall 6
Date/Time: September 14 at 12.00 – 13.00 CEST
Presenter: Dr. Paola Queirolo, Director, Medical Oncology of Melanoma, Sarcoma and Rare Tumors, European Institue of Oncology, Milan, Italy
About EVX-01 Phase 2 Clinical Trial

EVX-01 is Evaxion’s lead clinical asset and constitutes a peptide-based personalized cancer vaccine. The Phase 2 clinical study is a self-sponsored open-label, single-arm, multi-center trial carried out in collaboration with Merck Sharp & Dohme LLC that, together with leading principal investigators and research centers from Italy and Australia, aims to evaluate the efficacy and safety of EVX-01 vaccination in combination with anti-PD1 therapy KEYTRUDA (pembrolizumab) in treatment-naive patients with metastatic or unresectable malignant stage III or IV melanoma. More information can be accessed under clinical trial ID NCT05309421.