Aileron Therapeutics and Advancium Health Network Announce an Exclusive Option Agreement for the Acquisition of ALRN-6924 for Retinoblastoma

On October 30, 2024 Aileron Therapeutics, Inc. ("Aileron") (NASDAQ: ALRN), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, and Advancium Health Network ("Advancium"), a public charity founded by Deerfield Management and the Deerfield Foundation to meet the needs of underserved patient populations, reported entry into an exclusive option agreement for the acquisition of ALRN-6924, a clinical-stage oncology agent developed by Aileron prior to its 2023 merger with Lung Therapeutics, Inc (Press release, Aileron Therapeutics, OCT 31, 2024, View Source [SID1234647580]).

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During the option period, Advancium intends to evaluate ALRN-6924 as a potential therapy for retinoblastoma (RB), a rare but devastating cancer of the eye, with around 300 cases diagnosed in the United States and 9,000 cases worldwide each year, nearly all in children [1]. With early intervention the disease is rarely fatal; however, non-selective chemotherapy with its attendant side effects remains the usual treatment strategy, and removal of the affected eye(s) is a frequent outcome. In contrast to chemotherapy, ALRN-6924 selectively targets MDM2 and MDMX, the endogenous inhibitors of the regulatory protein p53, to activate p53-mediated tumor suppression in cancer cells [2]. Previous studies have implicated the MDMX protein as a key driver of RB tumorigenesis [3], and ALRN-6924 is the first and only clinical-stage drug that acts on MDMX. ALRN-6924 has previously been studied in preclinical models of RB, where it showed potent, on-mechanism anti-proliferative activity in RB cell lines and was found to be highly soluble and compatible with intraocular injection [4].

Under the terms of the option agreement, Advancium paid Aileron a non-refundable fee for the exclusive option to acquire ALRN-6924 and related assets. If Advancium exercises its option, Aileron will receive an exercise payment with potential for additional development, regulatory and commercial milestone payments and sales royalties.

"This agreement is a significant landmark for our work at Advancium, as it is the first drug opportunity we are pursuing in our mission as a public charity to develop much-needed therapies for children with cancer," said Mark Veich, Chief Executive Officer of Advancium and Vice President of Philanthropy and Executive Director of the Deerfield Foundation. "We look forward to evaluating ALRN-6924 as a potential treatment for children with retinoblastoma who currently have limited care options."

"Aileron is a strong supporter of Advancium’s cause," said Brian Windsor, Ph.D., President and Chief Executive Officer of Aileron. "Although we remain focused on developing novel therapies for the treatment of orphan pulmonary and fibrosis indications, we are honored that they have chosen to partner with us in this important step towards their goal of delivering therapies to pediatric patients with high unmet need."

Agios Reports Business Highlights and Third Quarter 2024 Financial Results

On October 31, 2024 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, reported business highlights and financial results for the third quarter ended September 30, 2024 (Press release, Agios Pharmaceuticals, OCT 31, 2024, View Source [SID1234647579]).

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"We had a strong quarter, marked by several important advancements across our pipeline. We completed enrollment of our Phase 3 RISE UP study of mitapivat in sickle cell disease, on our way to sharing topline results in late 2025. Our Phase 2b study of tebapivat in lower-risk MDS was initiated, and we received orphan drug designation from the FDA to support the development of tebapivat in this indication," said Brian Goff, chief executive officer of Agios. "Our cash position was further strengthened by the receipt of $1.1 billion in payments. This will allow us to maintain this great momentum and fuel our next phase of growth, building towards a franchise with multi-billion-dollar potential. We remain focused on progressing our promising clinical programs to address the critical needs of rare disease patients and look forward to sharing our progress in the coming months."

Third Quarter 2024 and Recent Highlights

PYRUKYND Revenues: Generated $9.0 million in net revenue for the third quarter of 2024, a 4 percent increase from the second quarter of 2024, primarily driven by increased patient demand. A total of 211 unique patients have completed prescription enrollment forms, representing an increase of 5 percent over the second quarter of 2024. A total of 127 patients are on PYRUKYND therapy, inclusive of new prescriptions and continued therapy.
Sickle Cell Disease: Completed enrollment of the 52-week Phase 3 RISE UP study of mitapivat in sickle cell disease, with more than 200 patients enrolled worldwide, and topline data expected in late 2025.
Lower-risk Myelodysplastic Syndromes:
Initiated patient enrollment in the Phase 2b study of tebapivat (AG-946).
Granted Orphan Drug Designation from the FDA to tebapivat for the treatment of myelodysplastic syndromes (MDS).
Presented topline data from Agios’ Phase 3 ACTIVATE-KidsT trial of mitapivat in pediatric patients with PKD.
Observed transfusion reduction response rates were higher in the mitapivat arm compared to placebo and were clinically meaningful despite not meeting prespecified statistical criterion for the primary endpoint; secondary endpoints of transfusion-free response and normal hemoglobin response were observed only in the mitapivat arm.
Safety was consistent with the profile observed in adults with PK deficiency who are regularly transfused.
Presented data from the Phase 3 ENERGIZE trial as an encore session at the 19th Annual Academy for Sickle Cell and Thalassemia (ASCAT) Conference.
Corporate Development:
The FDA approved Servier’s vorasidenib for the treatment of IDH-mutant diffuse glioma. As a result, Agios received a $200 million milestone payment from Servier and a $905 million payment from Royalty Pharma in connection with the purchase agreement announced in May 2024.
Entered into a distribution agreement with NewBridge Pharmaceuticals to advance commercialization of PYRUKYND in the Gulf Cooperation Council (GCC) region. NewBridge, a leading specialty company headquartered in Dubai, will commercialize PYRUKYND in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia and the United Arab Emirates.
Key Upcoming Milestones & Priorities

Agios expects to achieve the following key milestones by the end of 2024:

Thalassemia: File sNDA for mitapivat in thalassemia based on the positive results from the Phase 3 ENERGIZE and ENERGIZE-T clinical trials.
Present additional clinical data at the 66th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Congress, taking place December 7-10, 2024, in San Diego.
Third Quarter 2024 Financial Results

Revenue: Net product revenue from sales of PYRUKYND for the third quarter of 2024 was $9.0 million, compared to $7.4 million for the third quarter of 2023.

Cost of Sales: Cost of sales for the third quarter of 2024 was $0.8 million.

Research and Development (R&D) Expenses: R&D expenses were $72.5 million for the third quarter of 2024, compared to $81.8 million for the third quarter of 2023. The year-over-year decrease was primarily driven by the $17.5 million upfront payment associated with the license agreement with Alnylam, which was recorded in the prior year.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $38.5 million for the third quarter of 2024 compared to $25.8 million for the third quarter of 2023. The year-over-year increase was primarily attributable to an increase in commercial-related activities as the company prepares for the potential approval of PYRUKYND in thalassemia.

Net Income (Loss): Net income was $947.9 million for the third quarter of 2024 compared to a net loss of $91.3 million for the third quarter of 2023, reflecting the milestone and royalty agreement income recorded in the third quarter of 2024.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of September 30, 2024, were $1.7 billion compared to $806.4 million as of December 31, 2023. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND launches in thalassemia and sickle cell disease, advance existing programs, and to opportunistically expand its pipeline through both internally and externally discovered assets.

Conference Call Information

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss third quarter 2024 financial results and recent business highlights. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

Chugai Files for Additional Indication of Tecentriq for the Treatment of Extranodal Natural Killer/T-cell Lymphoma, Nasal Type, a Rare Disease

On October 31, 2024 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it filed regulatory application with the Ministry of Health, Labour and Welfare for the anti-cancer agent/humanized anti-PD-L1 monoclonal antibody Tecentriq Intravenous Infusion [generic name: atezolizumab (genetical recombination)] for an additional indication of relapsed or refractory extranodal natural killer/T-cell lymphoma, nasal type (R/R ENKL) (Press release, Chugai, OCT 31, 2024, View Source [SID1234647537]).

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"ENKL is a rare type of malignant lymphoma that primarily develops in the nose. It is known to have a poor prognosis, with about 60% of patients in advanced stages relapsing after initial treatment, and there is no standard therapy for relapsed cases. We are working to obtain approval so that Tecentriq, a cancer immunotherapy that demonstrated favorable efficacy, can be delivered to patients as soon as possible as a new therapeutic option for ENKL," said Chugai’s President and CEO, Dr. Osamu Okuda.

This filing is based on the results from a phase II ATTACK study initiated by investigators in Japan including National Cancer Center Hospital, which evaluated the efficacy and safety of Tecentriq in patients with R/R ENKL. Response was shown in 7 out of 13 cases and the study met its primary endpoint with an overall response rate of 53.8% (95% CI: 25.1-80.8%). The safety profile was consistent with that in other tumors.

Chugai Pharmaceutical, a leading company in the oncology field, remains committed to addressing unmet medical need in cancer treatment with innovative medicines for patients and healthcare professionals.

About ATTACK study1
ATTACK study (NCCH1903, jRCT2031190177) is a Japanese Phase II, multicenter, open-label, single-arm study led by physicians including National Cancer Center Hospital to evaluate the efficacy and safety of Tecentriq in patients with relapsed or refractory extranodal natural killer/T-cell lymphoma, nasal type. The study enrolled 14 patients to investigate safety and efficacy. The primary endpoint is independent review committee (IRC)-assessed overall response rate. Key secondary endpoints include progression-free survival, overall survival, and safety.
ATTACK study is being conducted as a substudy of the MASTER KEY project, which promotes the development of treatments for rare cancers through industry-academia collaboration with the National Cancer Center Hospital.

About extranodal natural killer/T-cell lymphoma, nasal type (ENKL)
ENKL is a form of malignant lymphoma that primarily affects the nasal cavity. It can occur in individuals of all ages, from children to adults.2,3,4 ENKL is rare, accounting for approximately 0.68% of all malignant lymphoma cases (annual incidence: about 36,000 cases) in Japan.5 For patients with advanced ENKL, about 60% experience relapse following initial treatment.6,7 Relapsed or refractory ENKL has a poor prognosis, and there is currently no established standard treatment.

About Tecentriq8
Tecentriq is a cancer immune checkpoint inhibitor targeting PD-L1, which is a protein expressed on tumor and tumor-infiltrating immune cells. PD-L1 blocks T cell activity by binding with PD-1 and B7.1 receptors on T cell surface. By inhibiting PD-L1, Tecentriq may enable the activation of T cells and boost immune response against cancer cells. In Japan, Tecentriq was launched in April 2018 and has obtained approval for 4 indications (extensive-stage small cell lung cancer, non-small cell lung cancer, breast cancer, and hepatocellular carcinoma). Tecentriq was filed for additional indication of alveolar soft part sarcoma in March 2024.

Trademarks used or mentioned in this release are protected by law.

Conference on Q2 FY2024 (April 1, 2024 to September 30, 2024)

On October 30, 2024 Sumitomo Dainippon Pharma reported second quarter results (April 1, 2024 to September 30, 2024) (Presentation, Sumitomo Dainippon Pharma, OCT 30, 2024, View Source [SID1234649696]).

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Summary of Consolidated Financial Results for the Second Quarter (First Half) of the Year Ending March 31, 2025

On October 30, 2024 Sumitomo Dainippon Pharma reported summary of Consolidated Financial Results for the Second Quarter (First Half) of the Year Ending March 31, 2025 (Press release, Sumitomo Dainippon Pharma, OCT 30, 2024, View Source [SID1234649695]).

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