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.

On November 14, 2024 Affimed N.V. (Nasdaq: AFMD) ("Affimed" or the "Company"), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer, reported financial results and provided an update on clinical and corporate progress for the quarter ended September 30, 2024 (Press release, Affimed, NOV 14, 2024, View Source [SID1234649279]).

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"Our innate cell engager therapies are meeting the critical needs of cancer patients who often come to us after exhausting nearly all other treatment options. The responses we’ve observed, even in the heavily pre-treated populations, give us tremendous confidence," said Dr. Shawn Leland, Chief Executive Officer of Affimed. "With clinical proof of concept established for all our innate cell engagers, the demonstrated activity across both hematological and solid tumors, combined with a strong safety profile, underscores the potential of our platform. We are refining our strategy to focus on clinical priorities and commercial viability, ensuring that our therapies deliver meaningful, long-term impact. I’m also very excited about expanding our efforts to seek collaborations with potential partners who share our vision to drive success for both patients and stakeholders."

Pipeline Highlights:

AFM24 (EGFR / CD16A)
In the AFM24-102 trial (combination with atezolizumab):

The cohort of NSCLC EGFRwt patients who failed chemotherapy and PD-1/PD-L1 has completed enrollment. ORR and safety data for this cohort will be presented on a Company conference call on December 17, 2024.
As previously reported at ASCO (Free ASCO Whitepaper) 2024, in 17 EGFRwt NSCLC patients who failed chemotherapy and PD-1/PD-L1 treatment, 1 complete response (CR), 3 partial responses (PRs) and 8 stable diseases (SDs) had been observed. Disease control was 71%. At the time, 3 of the 4 responses were ongoing for more than 7 months and median progression free survival (PFS) was 5.9 months.
PFS data from the EGFRwt and ORR and PFS data from the EGFR mutant (EGFRmut) cohorts expected to be presented at a major scientific conference in H1 2025.
Acimtamig (AFM13) (CD30 / CD16A)

Updated clinical data from the four cohorts of the run-in phase of the LuminICE-203 study will be shared in a poster session at ASH (Free ASH Whitepaper) 2024.
As previously reported on the Company’s Q2 2024 earnings call, in the phase 2 LuminICE-203 trial, 12 patients with advanced, treatment-refractory Hodgkin Lymphoma in cohorts 1 and 2, treated with a combination of the CD30-targeting innate cell engager acimtamig (AFM13) and AlloNK, demonstrated high efficacy, with an ORR of 83.3% and a CRR of 50%.
AFM28 (CD123 / CD16A)

An oral presentation at ASH (Free ASH Whitepaper) 2024 will feature updated clinical data including efficacy and safety data from the AFM28 phase 1 dose escalation study in relapsed/refractory acute myeloid leukemia (AML).
A poster with preclinical data will highlight the in vitro efficacy of AFM28 in combination with both patient-derived autologous NK cells and healthy volunteer-derived allogeneic NK cells against leukemic blasts.
As previously reported, initial data on the six patients treated at dose level 6 at 300 mg in the multi-center phase 1 open-label, dose-escalation study (AFM28-101), of AFM28 monotherapy in CD123-positive r/r AML, showed 1 patient with a CR, 2 patients with a CRi for a composite complete remission (CRc) rate (defined as CR+CRi) of 50% (3/6).
An additional six patients have been enrolled in the 300 mg cohort.
Upcoming Milestones:

LuminICE-203: Efficacy update of four cohorts to be presented at ASH (Free ASH Whitepaper) 2024.
AFM24-102: ORR and safety data from the EGFRwt cohort to be presented at a Company conference call on December 17, 2024.
AFM28-101: Updated data from the dose escalation to be presented at ASH (Free ASH Whitepaper) 2024.
AMF24-102: ORR and PFS data from the EGFRmut and PFS data from the EGFRwt cohorts expected to be presented at a major scientific conference in H1 2025.
Third Quarter 2024 Financial Highlights
Affimed’s consolidated financial statements are prepared in accordance with International Financial Reporting Standards (IFRS) as issued by the International Accounting Standard Board (IASB). The consolidated financial statements are presented in Euros (€), the Company’s functional and presentation currency.

As of September 30, 2024, cash, cash equivalents and short-term investments totaled €24.1 million. Based on current operating and budget assumptions, the Company expects that cash, cash equivalents and investments, together with anticipated proceeds from its ATM program and the sale of AbCheck, will finance its operations into Q4 of 2025.

Net cash used in operating activities for the quarter ended September 30, 2024, was €11.1 million compared to €18.3 million for the quarter ended September 30, 2023. The decline was mainly due to lower research and development expenditure and personnel expenses.

Total revenue for the quarter ended September 30, 2024, was €0.2 million compared with €2.0 million for the quarter ended September 30, 2023. Revenue in 2024 only related to a platform license provided to Genentech and 2023 predominantly related to the Roivant research collaborations for which all work has been completed.

Research and development expenses for the quarter ended September 30, 2024, were €10.1 million compared to €21.5 million in 2023. The decrease was primarily a result of lower expenses associated with the development of acimtamig and AFM24, due to a decrease in procurement of clinical trial material, clinical trial costs and manufacturing costs, decrease in head count due to the corporate restructuring.

General and administrative expenses for the quarter ended September 30, 2024, were €4.3 million compared to €5.4 million for the quarter ended September 30, 2023. The decrease was due to the decline in headcount, in legal and consulting expenses, insurance expenses and share-based payment expenses.

Net loss for the quarter ended September 30, 2024, was €15.1 million, or €0.94 loss per common share compared with a net loss of €24.4 million, or €1.63 loss per common share, for the quarter ended September 30, 2023.

The weighted number of common shares outstanding for the quarter ended September 30, 2024, was 16,100,185 shares.

Additional information regarding these results will be included in the notes to the consolidated financial statements as of September 30, 2024, included in Affimed’s filings with the U.S. Securities and Exchange Commission (SEC).

Note on International Financial Reporting Standards (IFRS)
Affimed prepares and reports consolidated financial statements and financial information in accordance with IFRS as issued by the IASB. None of the financial statements were prepared in accordance with U.S. Generally Accepted Accounting Principles. Affimed maintains its books and records in Euro.

Conference Call and Webcast Information
Affimed will host a conference call and webcast on November 14, 2024, at 8:30 a.m. EST / 14:30 CET to discuss third quarter 2024 financial results and corporate developments.

The conference call will be available via phone and webcast. The live audio webcast of the call will be available in the "Webcasts" section on the "Investors" page of the Affimed website at View Source To access the call by phone, please use link: https://register.vevent.com/register/BIb9251d6bc6504a4da37a53cdb1b0ac94, and you will be provided with dial-in details and a pin number.

Note: To avoid delays, we encourage participants to dial into the conference call 15 minutes ahead of the scheduled start time. A replay of the webcast will be accessible at the same link for 30 days following the call.

On November 14, 2024 Merck, a leading science and technology company, accelerated its growth in the third quarter of 2024 (Press release, Merck KGaA, NOV 14, 2024, View Source [SID1234648982]). All three business sectors achieved organic increases in sales and earnings. Accordingly, the company remains on course to return to profitable growth in fiscal 2024, as previously announced.

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On November 14, 2024 HOOKIPA Pharma Inc. (NASDAQ: HOOK, "HOOKIPA", the "Company"), a clinical-stage biopharmaceutical company developing next generation immunotherapeutics for the treatment of cancer and serious infectious disease, reported financial results for the third quarter ended September 30, 2024 and provided recent business highlights, including an update on the progress of the lead clinical program, eseba-vec (Press release, Hookipa Pharma, NOV 14, 2024, View Source [SID1234648545]).

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"HOOKIPA made excellent progress across each program in our pipeline in the third quarter," said Malte Peters, MD, Chief Executive Officer of HOOKIPA. "We advanced the development of eseba-vec for HPV16+ head and neck squamous cell carcinoma (HNSCC), through the start of an investigator-initiated study in the new clinical setting of adjuvant care, and presented continued positive, updated data from our Phase 2 study in first-line recurrent/metastatic disease as a late breaking abstract at SITC (Free SITC Whitepaper) 2024. In parallel, we have made significant progress with our review of the business strategy and operations, having implemented a number of initiatives to optimize spending and ensure prioritization of resources."

Recent Developments

Oncology
Eseba-vec: Pivotal-trial ready immunotherapy for human papilloma virus type 16 positive (HPV16+) cancers, including head and neck squamous cell carcinoma (HNSCC) and oropharyngeal squamous cell carcinoma (OPSCC). HOOKIPA owns all rights to this program.

Enrollment completed in the Phase 2 H200-001 study for eseba-vec in combination with pembrolizumab in HPV+ HNSCC with 68 patients enrolled as of October 2024.
First Patients Dosed in Phase 2 Adjuvant Therapy IIT with MSKCC: On October 30, 2024, HOOKIPA announced that researchers at Memorial Sloan Kettering Cancer Center (MSKCC) dosed the first patients in an investigator-initiated trial (IIT) evaluating eseba-vec in patients with minimal residual disease positive (MRD+) HPV-16+ driven, locally advanced HNSCC, following treatment for curative intent. The study could pave the way to broaden the eseba-vec HNSCC opportunity into adjuvant care.
Updated Phase 2 Data Presented as SITC (Free SITC Whitepaper) 2024 Late-Breaker: On November 9, 2024, HOOKIPA’s clinical collaborator, Alan Ho, MD, PhD, Chief of the Head and Neck Oncology Service at MSKCC, presented a late-breaking poster at the 39th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (SITC 2024). The presentation provided updated results, including data from additional patients enrolled in the Phase 2 trial evaluating eseba-vec plus pembrolizumab as 1L treatment of recurring/metastatic HPV16+ HNSCC in patients with PD-L1 CPS levels of greater than or equal to 20. Eseba-vec treatment resulted in continued, durable clinical responses and antigen-specific T cell responses.
HB-700: The HB-700 program is a novel Phase 1-ready immunotherapy for KRAS-mutated cancers, including pancreatic, colorectal and lung cancer. The investigational therapy was designed to target the most prevalent KRAS mutations of these cancers in a single therapy. HOOKIPA owns all rights to this program and the Investigational New Drug Application (IND) received FDA clearance in April 2024.

Presentation of Strong Preclinical Dataset: On September 25, 2024, HOOKIPA presented the preclinical proof-of-concept dataset for HB-700 at the 6th Annual RAS-Targeted Drug Development Summit. The dataset demonstrated that HB-700 induced target-specific CD8+ T cells and target cell killing in several different animal and translational models.
Infectious Diseases
HOOKIPA is advancing two independent anti-viral programs (HB-400 for HBV and HB-500 for HIV-1) through a collaboration and license agreement with Gilead Sciences, Inc. (Gilead).

HB-400: An investigational therapeutic vaccine for the treatment of chronic hepatitis B (CHB).

Enrollment Completed in Phase 1a/1b Clinical Trial: The vaccine is being evaluated in a Phase 1a/1b clinical trial (NCT05770895) in 83 subjects to assess the safety and immune response induced by HB400 in healthy participants and in participants with CHB on oral antiviral therapy.
HB-500: An investigational therapeutic vaccine for the treatment of human immunodeficiency virus-1 (HIV-1).

Ongoing Phase 1b Clinical Trial: The vaccine is being evaluated in a Phase 1b clinical trial (NCT06430905) to assess the safety and magnitude of cellular immune response against HIV-1 induced by HB-500 in people living with HIV who are taking anti-retroviral treatment. Under the collaboration agreement with Gilead, HOOKIPA received a $5 million milestone payment associated with dosing of the first subject in this trial in July 2024.
Corporate and Financial Updates

Corporate Highlights

Board of Director Changes: On August 30, 2024, Director Julie O’Neill was appointed to be Non-Executive Chair of the Company’s Board of Directors, succeeding Jan van de Winkel, who decided to step down from the Board due to increased time commitments from other executive responsibilities. Tim Reilly also stepped down from the Board to dedicate more time to his other professional responsibilities. HOOKIPA is grateful for the years of service each Director dedicated to the Company.

Leadership Changes: On July 22, 2024, the Board of Directors appointed Malte Peters, MD, as Chief Executive Officer and Terry Coelho as Executive Vice President and Chief Financial Officer to lead the Company through its next phase of development and to realize the significant opportunity of HOOKIPA’s pipeline.

Board Appointment: On July 22, 2024, Sean Cassidy was appointed to the Board of Directors. Mr. Cassidy serves as the chair of the Audit Committee and as a member of the Compensation and the Nominating and Corporate Governance Committees.

Reverse Split: On July 9, 2024, the Company effected a reverse stock split of the outstanding shares of its common stock on a one-for-ten (1:10) basis. The reverse stock split is part of the Company’s plan to regain compliance with the minimum bid price requirement for continued listing on the Nasdaq Capital Market.
Financial Highlights: Milestone Payments

Gilead: In July, HOOKIPA received a $5.0 million milestone payment under its collaboration and license agreement with Gilead. The success-based milestone payment was achieved in connection with the dosing of the first subject in the Phase 1b clinical trial of HB-500 for the treatment of HIV-1, initiated on July 1, 2024.
Third Quarter 2024 Financial Results

Cash Position: HOOKIPA’s cash, cash equivalents and restricted cash as of September 30, 2024 was $60.0 million, compared to $117.5 million as of December 31, 2023. The decrease was primarily attributable to cash used in operating activities, partially offset by cash received relating to milestone achievements under our collaboration agreements with Roche and Gilead.

Revenue: Revenue was $4.7 million for the three months ended September 30, 2024, compared to $6.9 million for the same period in 2023. The decrease was primarily due to lower partnering revenues as a result of the termination of the Roche collaboration agreement.

Research and Development Expenses: HOOKIPA’s research and development expenses were $15.6 million for the three months ended September 30, 2024, compared to $24.6 million for the same period in 2023. The primary changes in research and development expenses were lower personnel-related and laboratory-related expenses as well as lower manufacturing and research expenses resulting from the pause in development activities related to HB-300 announced in January 2024, partially offset by higher clinical study expenses for the eseba-vec program.

General and Administrative Expenses: General and administrative expenses amounted to $6.7 million for the three months ended September 30, 2024, compared to $4.9 million for the same period in 2023. The primary drivers of the increase in general and administrative expenses were an increase in personnel-related expenses and an increase in professional and consulting fees incurred in connection with management transitions during the third quarter of 2024.

Restructuring Expenses: Restructuring expenses amounted to $0.9 million for the three months ended September 30, 2024, and resulted from severance and other personnel costs as well as professional fees related to a reduction in workforce and related activities conducted in the third quarter of 2024.

Impairment Expenses: Impairment expenses amounted to $0.2 million for the three months ended September 30, 2024, and resulted from write-downs related to laboratory equipment.

Net Loss: HOOKIPA’s net loss was $13.8 million for the three months ended September 30, 2024, compared to a net loss of $19.1 million for the same period in 2023.

On November 14, 2024 Nerviano Medical Sciences S.r.l. (NMS) – a part of NMS Group S.p.A. (NMS Group) and Nerviano Medical Sciences, Inc., a wholly owned subsidiary of NMS Srl – a global leader in oncological innovation with a leading position in PARP1 inhibitor clinical development in brain cancer, reported the acquisition of atamparib, the first selective PARP7 inhibitor (Press release, Nerviano Medical Sciences, NOV 14, 2024, View Source [SID1234648503]). This transaction highlights NMS’s commitment to solidify its leadership in PARP biology and expanding its pipeline with transformative cancer therapies designed to meet significant unmet medical needs.

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Strategic Enhancement of Oncology Portfolio: Atamparib is currently in Phase II with the potential to be first-in-class. This PARP7 inhibitor enriches NMS’s diverse range of small molecules in the portfolio developed for various types of cancers. PARP7, a crucial target in oncology, is upregulated in response to cellular stress, promoting cancer cell survival. By targeting PARP7 in tumor cells, atamparib has been shown to inhibit cellular proliferation. It is an orally bioavailable small molecule with high potency on pre-clinical models, and a favorable safety and tolerability profile in PhI/II studies.

Leveraging Expertise in PARP Biology: NMS brings extensive knowledge in PARP biology, demonstrated through the development of NMS-293, an oral potent and selective PARP1 inhibitor with non-trapping effects and brain penetrant properties. NMS-293 has shown promising clinical activity in a Ph I/II study in combination with temozolomide in recurrent glioblastoma and astrocytoma patients. Building on this expertise and foundational research conducted by Ribon Therapeutics, NMS will advance the atamparib program into an innovative and effective therapy. This initiative reinforces NMS leadership in the development of PARP inhibitors.

Dr. Hugues Dolgos, CEO of Nerviano Medical Sciences, commented: "We are excited about the addition of atamparib to our pipeline. NMS has been working on PARP7 for a long time, therefore, we believe that we are in a unique position to leverage our knowledge during the clinical development of atamparib. The very recent scientific evidence of the PARP7 biology is directing us to a different setting in which we believe we can demonstrate proof of concept in the clinical trials we plan to initiate in 2025".