On November 15, 2024 Bristol Myers Squibb (NYSE: BMY) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval for repotrectinib, a next-generation tyrosine kinase inhibitor (TKI), as a treatment for adult patients with ROS1 -positive advanced non-small cell lung cancer (NSCLC) and for the treatment of adult and pediatric patients 12 years of age and older with advanced solid tumors expressing a NTRK gene fusion, and who have received a prior NTRK inhibitor, or have not received a prior NTRK inhibitor and treatment options not targeting NTRK provide limited clinical benefit, or have been exhausted (Press release, Bristol-Myers Squibb, NOV 15, 2024, View Source [SID1234648438]). The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP recommendation. The final EC decision is expected in January 2025.

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"Patients in the EU with ROS1 -positive non-small cell lung cancer and NTRK -positive solid tumors face a great unmet need for new therapies that may improve their outcomes and address or delay the difficult issue of treatment resistance," said Joseph Fiore, vice president, global program lead, repotrectinib, Bristol Myers Squibb. "We look forward to the EC’s upcoming decision and to potentially bringing this next-generation treatment to patients with tumors harboring ROS1 or NTRK fusions in the EU."

The positive opinion is based on results from the TRIDENT-1 and CARE trials, in which repotrectinib demonstrated clinically meaningful response rates in patients across ROS1 -positive NSCLC and NTRK -positive solid tumor cohorts, both as an initial targeted treatment or after progression on a prior targeted treatment. Durability of response was robust and intracranial responses were observed in both settings, as well as in patients whose tumors harbor common resistance mutations. The safety profile of repotrectinib was well characterized and generally manageable with routine standard-of-care treatments. The study remains ongoing to assess long-term outcomes and additional endpoints across patient populations with ROS1 -positive advanced NSCLC and NTRK -positive advanced solid tumors.

In November 2023 the U.S. Food and Drug Administration approved Augtyro (repotrectinib) for the treatment of adult patients with locally advanced or metastatic ROS1 -positive NSCLC. In June 2024 the U.S. Food and Drug Administration approved Augtyro for the treatment of patients with NTRK-positive locally advanced or metastatic solid tumors.

Bristol Myers Squibb thanks the patients and investigators involved in the TRIDENT-1 and CARE clinical trials.

About TRIDENT-1

TRIDENT-1 is a global, multicenter, single-arm, open-label, multi-cohort Phase 1/2 clinical trial evaluating the safety, tolerability, pharmacokinetics and anti-tumor activity of repotrectinib in patients with advanced solid tumors, including non-small cell lung cancer (NSCLC). Phase 1/2 includes patients with locally advanced or metastatic solid tumors harboring ROS1 and NTRK fusions. Additional analyses of the trial are still being conducted; asymptomatic central nervous system (CNS) metastases are allowed. The trial excludes patients with symptomatic brain metastases, among other exclusion criteria. Phase 1 of the trial included the dose escalation that determined the recommended Phase 2 dose.

Phase 2 of the trial has a primary endpoint of overall response rate (ORR). Key secondary endpoints include duration of response (DOR) according to Response Evaluation Criteria in Solid Tumors (RECIST v1.1) as assessed by Blinded Independent Central Review (BICR), progression-free survival (PFS), and intracranial response in six distinct expansion cohorts, including tyrosine kinase inhibitor (TKI)-naïve and TKI-pretreated patients with ROS1 -positive locally advanced or metastatic NSCLC and NTRK -positive locally advanced or metastatic solid tumors.

About CARE

CARE is a Phase 1/2 open-label, safety, tolerability, pharmacokinetics and anti-tumor activity clinical trial evaluating repotrectinib in pediatric and young adult patients with locally advanced or metastatic solid tumors harboring ALK , ROS1 or NTRK1-3 gene alterations.

Phase 1 of the study aims to evaluate the safety and tolerability at different dose levels. Phase 1 of the trial has primary endpoints of dose limiting toxicities (DLTs) and pediatric recommended Phase 2 dose (RP2D). Secondary endpoints include overall response rate (ORR), clinical benefit rate (CBR), time to response (TTR), duration of response (DOR) and intracranial ORR (IC-ORR). Patients less than 12 years of age will initially be enrolled in Phase 1 to determine the RP2D for this age group.

Once the pediatric RP2D is determined, patients less than 12 years of age may be enrolled into Phase 2 of the study. Patients ages 12 to 25 years will be directly enrolled into Phase 2 concurrent with Phase 1 enrollment. Phase 2 of the study will seek to demonstrate the efficacy and anti-tumor activity of repotrectinib in pediatric and young adult patients. The primary endpoint of Phase 2 is ORR and secondary endpoints include CBR, TTR, DOR, IC-ORR, progression-free survival (PFS), central nervous system PFS (CNS-PFS) and overall survival (OS).

About ROS1 -Positive Lung Cancer

Lung cancer is the leading cause of cancer deaths in the United States. The two main types of lung cancer are non-small cell and small cell. Non-small cell lung cancer (NSCLC) represents up to 85% of diagnoses. Survival rates vary depending on the stage and type of the cancer when diagnosed. ROS1 fusions are rare and occur in about 1-2% of patients with NSCLC. With a median age of 50, patients with tumors that are ROS1-positive tend to be younger than the average lung cancer patient, more often female than male and may have little to no smoking history. ROS1 -positive lung cancer tends to be aggressive and can often spread to the brain. ROS1 tyrosine kinase inhibitor (TKI) therapy is the current standard of care for patients with a tumor harboring this gene alteration.

About NTRK -Positive Solid Tumors

Neurotrophic tropomyosin receptor kinase (NTRK) are a family of receptors involved in neural development. An NTRK gene fusion is an alteration that occurs when a piece of the chromosome containing the NTRK gene breaks off and joins with a gene on another chromosome. These fusions lead to abnormal proteins, which may cause cancer cells to grow. While NTRK gene fusions are rare in patients with solid tumors, testing for NTRK gene fusions allows for the identification of patients who may benefit from TRK inhibitor therapy.

On November 15, 2024 Bristol Myers Squibb (NYSE: BMY) reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Opdivo (nivolumab) plus Yervoy (ipilimumab) for the first-line treatment of adult patients with microsatellite instability–high (MSI-H) or mismatch repair deficient (dMMR) unresectable or metastatic colorectal cancer (mCRC) (Press release, Bristol-Myers Squibb, NOV 15, 2024, View Source [SID1234648437]). Of significance, the CheckMate -8HW trial results showed reduction in the risk of disease progression or death by 79% (HR: 0.21; 95% CI: 0.14-0.32; p<0.0001) compared to chemotherapy in this patient population. The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the recommendation and make their decision.

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"Approximately 5-7% of metastatic colorectal cancer patients have dMMR or MSI-H tumors, and current treatment options often do not provide sufficient benefit," said Dana Walker, M.D., M.S.C.E., vice president, global program lead, gastrointestinal and genitourinary cancers, Bristol Myers Squibb. "This is the first dual checkpoint inhibitor treatment for first-line metastatic colorectal cancer, delivering a transformative benefit for MSI-H/dMMR patients in this population. We are focused on bringing Opdivo plus Yervoy to these patients in the European Union and look forward to EC’s upcoming decision."

The positive opinion is based on results from the CheckMate -8HW trial, which were presented at medical congresses earlier this year. These data formed the basis for the Company’s Type II variation application, which was validated by the European Medicines Agency (EMA). In the study, Opdivo plus Yervoy demonstrated a statistically significant and clinically meaningful improvement in the dual primary endpoint of progression-free survival (PFS) compared to the investigator’s choice of chemotherapy as assessed by Blinded Independent Central Review. In addition to the risk of disease progression or death the results noted, the safety profile for the dual immunotherapy combination remained consistent with previously reported data and was manageable with established protocols, with no new safety signals identified.

In October 2024, it was announced that Opdivo plus Yervoy also demonstrated a statistically significant and clinically meaningful improvement in the dual endpoint of PFS per BICR compared to Opdivo monotherapy across all lines of therapy. The study is ongoing to assess various secondary endpoints, including overall survival (OS).

Bristol Myers Squibb thanks the patients and investigators involved in the CheckMate -8HW clinical trial.

About CheckMate -8HW

CheckMate -8HW (NCT04008030) is a Phase 3 randomized, open-label trial evaluating Opdivo plus Yervoy compared to Opdivo alone or the investigator’s choice chemotherapy (mFOLFOX-6 or FOLFIRI with or without bevacizumab or cetuximab) in patients with microsatellite instability–high (MSI-H) or mismatch repair deficient (dMMR) unresectable or metastatic colorectal cancer (mCRC).

839 patients were randomized to receive either Opdivo monotherapy ( Opdivo 240 mg Q2W for six doses, followed by Opdivo 480 mg Q4W), Opdivo plus Yervoy (Opdivo 240 mg plus Yervoy 1 mg/kg Q3W for four doses, followed by Opdivo 480 mg Q4W ), or investigator’s choice of chemotherapy. The dual primary endpoints of the trial are progression-free survival (PFS) per blinded independent central review (BICR) for Opdivo plus Yervoy compared to investigator’s choice of chemotherapy in the firstline setting and PFS per BICR for Opdivo plus Yervoy compared to Opdivo alone across all lines of therapy.

CheckMate-8HW has also met its other dual primary point of PFS per BICR Opdivo plus Yervoy compared to Opdivo across all lines therapy in randomized subjects with centrally confirmed MSI-H/dMMR mCRC at the second interim analysis in September 2024, and data disclosure is planned for an upcoming medical conference. The trial also evaluates several secondary safety and efficacy endpoints, including overall survival, which is ongoing.

About dMMR or MSI-H Colorectal Cancer

Colorectal cancer (CRC) is cancer that develops in the colon or the rectum, which are part of the body’s digestive or gastrointestinal system. CRC is the third most commonly diagnosed cancer in the world. In 2020, it is estimated that there were approximately 1,931,000 new cases of the disease; it is the second leading cause of cancer-related deaths among men and women combined.

Mismatch repair deficiency (dMMR) occurs when the proteins that repair mismatch errors in DNA replication are missing or non-functional, leading to microsatellite instability-high (MSI-H) tumors. Approximately 5-7% of metastatic CRC patients have dMMR or MSI-H tumors. These patients are less likely to benefit from conventional chemotherapy and typically have a poor prognosis.

On November 15, 2024 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported its financial results for the third quarter ended September 30, 2024 and provided an update on recent corporate developments (Press release, Bio-Path Holdings, NOV 15, 2024, View Source [SID1234648436]).

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"The third quarter was a particularly productive period for Bio-Path as we initiated our obesity program, which marks the first application of our DNAbilize platform beyond oncology and highlights its broad therapeutic potential," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. "Importantly, we continued to advance our oncology programs and were delighted with the swift enrollment of the third cohort in our Phase 1/1b clinical trial of BP1002 in venetoclax-resistant AML patients, which was ahead of our projected timelines. We also published an article highlighting the broad anti-tumor effect of BP1003 in numerous preclinical solid tumor models, including breast, ovarian, and pancreatic cancer, in the peer-reviewed journal, Biomedicines."

Recent Corporate Highlights

Initiated BP1001-A Therapeutic Program for Treatment of Obesity. In October, the Company announced the initiation of a therapeutic program to develop BP1001-A for the treatment of obesity and related metabolic diseases. This program marks the first application of DNAbilize technology for development of a non-cancer application, which highlights the broad therapeutic potential of this technology. The disease pathology leading to obesity suggests that BP1001-A, which suppresses the adaptor protein Grb2, has the potential to treat insulin resistance, a major contributor to obesity, Type 2 diabetes and other related metabolic diseases. Bio-Path expects downregulating Grb2 expression with BP1001-A will enhance insulin sensitivity.

Completion of Enrollment for Third Dosing Cohort of Phase 1/1b Clinical Trial of BP1002 in Venetoclax-Resistant Acute Myeloid Leukemia (AML) Patients. In October, Bio-Path announced completion of enrollment for the third dosing cohort of the Company’s Phase 1/1b trial of BP1002 in venetoclax-resistant AML patients. The cohort enrolled more quickly than projected, which underscores the ongoing need for new treatment options for these relapsed/refractory patients.

Publication in Peer-Reviewed Journal, Biomedicines. In September, Bio-Path published an article highlighting the therapeutic potential of BP1003 in a variety of cancer types in the peer-reviewed journal, Biomedicines. The article describes the broad anti-tumor effect of BP1003 in numerous preclinical solid tumor models including breast, ovarian, and pancreatic cancer. BP1003 is a neutral liposome incorporated with a nuclease resistant P-ethoxy antisense oligodeoxynucleotide targeting the STAT3 mRNA and its unique design enhances stability, cellular uptake, and target affinity.

Reported Solid Tumor Patient Response Supporting BP1001-A’s Compelling Potential as Treatment for Advanced Solid Tumors. Bio-Path’s first patient in the second dose cohort in its Phase 1/1b advanced solid tumor clinical trial experiencing a positive response may signal that this analog of prexigebersen has potential as a new treatment for advanced solid tumors. The patient appears to be doing well on study after failing extensive chemotherapy and surgical treatment for gynecologic cancer, demonstrating a 15% reduction in her primary tumor through six cycles of treatment. Moreover, it appears that these positive outcomes may have contributed to allow her to continue with rigorous exercise and improved quality of life.

The dose finding portion of the Phase 1/1b trial is comprised of BP1001-A monotherapy with no accompanying chemotherapy. This clinical trial of BP1001-A in patients with advanced or recurrent solid tumors has successfully completed the initial prescribed dose in the first cohort of 60 mg/m2 and began enrollment in the higher dose cohort of 90 mg/m2. The Phase 1b portion of the study is expected to commence after completion of three planned BP1001-A monotherapy dose level cohorts and is intended to assess the safety and efficacy of BP1001-A in combination with paclitaxel in patients with recurrent ovarian or endometrial tumors. Phase 1b studies are also expected to be opened in combination with gemcitabine in late-stage pancreatic cancer.
Closed $4.0 Million Private Placement. In October, Bio-Path announced closing of a private placement priced at-the-market under Nasdaq rules for the issuance and sale of an aggregate of 4,597,702 shares of its common stock (or common stock equivalents in lieu thereof), series A warrants to purchase up to 6,407,657 shares of common stock and short-term series B warrants to purchase up to 6,407,657 shares of common stock at a purchase price of $0.87 per share of common stock (or per common stock equivalent in lieu thereof) and accompanying warrants in a private placement. The gross proceeds to the Company from the offering were approximately $4.0 million, before deducting the placement agent’s fees and other offering expenses payable by the Company, and excluding the proceeds, if any, from the exercise of the warrants.
Financial Results for the Third Quarter Ended September 30, 2024

The Company reported a net loss of $2.1 million, or $0.70 per share, for the three months ended September 30, 2024, compared to a net loss of $3.2 million, or $6.36 per share, for the three months ended September 30, 2023.

Research and development expense for the three months ended September 30, 2024 decreased to $1.3 million, compared to $2.3 million for the three months ended September 30, 2023 primarily due to decreased manufacturing expenses related to drug product releases as well as a decrease in expense related to our clinical trial for BP1001 in AML due to timing of patient enrollment during the quarter.

General and administrative expense for the three months ended September 30, 2024 increased to $1.3 million, compared to $1.0 million for the three months ended September 30, 2023 primarily due to increased legal fees and salaries and benefits expense.

As of September 30, 2024, the Company had cash of $0.6 million, compared to $1.1 million as of December 31, 2023. Net cash used in operating activities for the nine months ended September 30, 2024 was $7.7 million compared to $9.7 million for the comparable period in 2023. Net cash provided by financing activities for the nine months ended September 30, 2024 was $7.2 million.
Conference Call and Webcast Information

Bio-Path Holdings will host a conference call and webcast today at 8:30 a.m. ET to review these third quarter 2024 financial results and to provide a general update on the Company. To access the conference call please dial (844) 481-3014 (domestic) or (412) 317-1879 (international). A live audio webcast of the call and the archived webcast will be available in the Media section of the Company’s website at www.biopathholdings.com.

On November 15, 2024 AIM ImmunoTech Inc. (NYSE American: AIM) ("AIM" or the "Company") reported its financial results for the third quarter 2024 (Press release, AIM ImmunoTech, NOV 15, 2024, View Source [SID1234648435]). Also, the Company will host a conference call and webcast today, November 15, 2024 at 8:30 AM ET (details below).

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AIM Chief Executive Officer Thomas K. Equels stated, "We are driving significant momentum across multiple clinical programs and studies that are demonstrating Ampligen’s significant potential to address high value and high need indications, especially in the pancreatic cancer space. Over the course of 2024, our team has made important progress in executing our clinical strategy, facilitating potential partnerships with big pharma and leveraging commercialization opportunities to create value. Our Board of Directors is dedicated to helping patients in need and delivering enhanced value for our shareholders."

Recent Highlights

Pancreatic Cancer:
Reported positive preliminary data in Phase 1b/2 study of Ampligen and Imfinzi as a combination therapy for late-stage pancreatic cancer.
Announced further positive findings from a study evaluating Ampligen in the treatment of pancreatic cancer.
Long-COVID:
Announced that analysis of AMP-518 complete clinical patient data underscores Ampligen’s potential to improve the post-COVID condition of fatigue.
Endometriosis: Company granted U.S. patent for Ampligen for composition of matter and method of treatment of endometriosis.
Please refer to previously released CEO Corner segments for additional highlights around the Company’s news and programs.

Summary of Financial Highlights for Third Quarter 2024

As of September 30, 2024, AIM reported cash, cash equivalents and marketable securities of $7.2 million.
Research and development expenses for the three months ended September 30, 2024 were $1.4 million, compared to $2.7 million for the same period in 2023.
General and administrative expenses were $3.1 million for the three months ended September 30, 2024, compared to $5.4 million for the same period 2023.
The net loss from operations for the three months ended September 30, 2024 was $3.7 million, or $0.06 per share, compared to $7.8 million, or $0.16 per share, for the three months ended September 30, 2023.
Please refer to the full 10-Q for complete details.

Conference Call and Webcast Details

As previously announced, the Company will host a conference call and webcast to discuss the Company’s Q3 2024 operational and financial results on November 15, 2024 at 8:30 AM ET.

The call will be hosted by members of AIM’s leadership team, Thomas K. Equels, Chief Executive Officer and Christopher McAleer, PhD, Scientific Officer. Interested participants and investors may access the conference call by dialing (877) 407-9219 (domestic) or (201) 689-8852 (international) and referencing the AIM ImmunoTech Conference Call. The webcast will be accessible on the Events page of the Investors section of the Company’s website, aimimmuno.com, and will be archived for 90 days following the live event.

On November 14, 2024 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the third quarter ended September 30, 2024, and provided a business update (Press release, Ascendis Pharma, NOV 14, 2024, View Source [SID1234649389]).

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"2024 has been another transformative year for Ascendis. Now, all three of our three endocrinology rare disease programs have delivered clinically differentiated pivotal data, each demonstrating potential ability to address major unmet medical needs and the potential for each to achieve blockbuster status. We are ready and very excited about launching YORVIPATH in the U.S. with product availability expected in mid-January of 2025," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "In addition, our new partnership with Novo Nordisk highlights our ability to extend the success of our TransCon platform and positions Ascendis to benefit patients and capture significant value in this large, high volume therapeutic areas."

Select Highlights & Anticipated 2024 Milestones

TransCon hGH:
(lonapegsomatropin, marketed as SKYTROFA)
SKYTROFA revenue for the third quarter of 2024 totaled €47.2 million compared to €47.0 million during the same period in 2023. Volume growth was offset by higher sales deductions and a negative adjustment to prior period sales deductions of €2.5 million.
SKYTROFA revenue for the first nine months of 2024 totaled €138.5 million, a 21% year-over-year increase compared to €114.4 million during the same period of 2023. Volume growth was partially offset by higher sales deductions. In addition, sales deductions attributable to periods prior to January 1, 2024 totaled €9.3 million.
Submitted U.S. Food & Drug Administration (FDA) supplemental Biologics License Application for TransCon hGH for the treatment of adults with growth hormone deficiency.
Topline results from Phase 2 New InsiGHTS Trial in Turner syndrome expected in the fourth quarter of 2024.
Expect to initiate a basket trial evaluating SKYTROFA in other established daily growth hormone indications in the first half of 2025.
TransCon PTH:
(palopegteriparatide, marketed as YORVIPATH)
YORVIPATH launching in U.S. with our expanded U.S. field teams engaging with health care providers, our Ascendis Signature Access Program accepting prescriptions and enrolling patients starting in December in preparation for product availability in mid-January 2025
Third quarter YORVIPATH revenue outside the U.S. totaled €8.5 million, a sequential quarter-over-quarter revenue increase of more than 60%, reflecting growing physician and patient demand with now over 600 patients on treatment, partially offset by accruals reflecting the end of the free pricing period in the third quarter. Final pricing in Germany is expected to be completed next year.
TransCon CNP
(navepegritide)
Announced positive topline data from pivotal ApproaCH Trial with children with achondroplasia (ages 2-11 years) treated with once-weekly TransCon CNP.
Plan to submit New Drug Application (NDA) to the FDA for TransCon CNP for the treatment of children with achondroplasia during the first quarter of 2025 and a Marketing Authorisation Application (MAA) for the treatment of children with achondroplasia to the European Medicines Agency during the third quarter of 2025.
Expect topline Week 26 data from COACH, the combination TransCon hGH and TransCon CNP trial of children with achondroplasia (ages 2-11 years) in the second quarter of 2025.
Oncology Programs
Presented first results from platinum-resistant ovarian cancer (PROC) cohort of the Phase 1/2 IL-Believe Trial at ESMO (Free ESMO Whitepaper) 2024. Initial data suggest clinical activity in heavily pre-treated PROC patients and that TransCon IL-2 β/γ in combination with chemotherapy was generally well-tolerated.
Recently, we closed enrollment to dose expansion cohorts involving TransCon TLR7/8 Agonist in the transcendIT-101 and IL Believe trials to prioritize our efforts on TransCon IL-2 β⁄γ.
Strategic Collaboration
In November, granted Novo Nordisk A/S an exclusive, multi-product, worldwide license to the TransCon technology platform to develop, manufacture and commercialize Novo Nordisk proprietary products in metabolic diseases and a product-by-product exclusive license in cardiovascular diseases. The lead program in the collaboration is a once-monthly GLP-1 receptor agonist product candidate that will initially target obesity and type 2 diabetes.
For the lead program, Ascendis will be eligible to receive total payments of up to $285 million in upfront, development, and regulatory milestone payments, plus sales-based milestone payments and escalating tiered, mid-single digit royalties on global net sales.
Novo Nordisk will be responsible for Ascendis’ early development costs as well as for clinical development, regulatory, commercial manufacturing, and commercialization activities.
Financial Update and Outlook Based on Current Plans
As of September 30, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities, totaling €626 million, compared to €399 million as of December 31, 2023.
Full year 2024 SKYTROFA revenue excluding sales deductions related to prior years expected to be €200 – €220 million.
Expect total operating expenses (SG&A and R&D) to be approximately €600 million for 2024.
Third Quarter 2024 Financial Results
Total revenue for the third quarter of 2024 was €57.8 million, compared to €48.0 million during the same period for 2023. The year-over-year increase in total revenue was primarily attributable to revenue contribution of €8.5 million from YORVIPATH following commercial launch in the first quarter of 2024. Non-product revenue was €2.1 million in the third quarter of 2024, compared to €1.1 million for the same period for 2023.