On September 3, 2024 Galectin Therapeutics, Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins, reported that Joel Lewis, Chief Executive Officer and Khurram Jamil, M.D., Chief Medical Officer, will be attending the H.C. Wainwright 26th Annual Global Investment Conference being held September 9-11, 2024 in New York, NY (Press release, Galectin Therapeutics, SEP 3, 2024, View Source [SID1234646317]).

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Dr. Jamil will be providing a company presentation on September 9, 2024 at 12:00 PM ET. The webcast of the presentation can be accessed here or through the Company’s website on the Events & Presentation page of the investors section.

Additionally, Galectin management team will be available for one-on-one meetings during the conferences. Interested investors should contact their representative at H.C. Wainwright.

On September 3, 2024 Pacylex Pharmaceuticals Inc. (Pacylex) reported that it is seeking partners to develop its large family of NMT inhibitors as a new class of Antibody Drug Conjugate (ADC) payloads (Press release, Pacylex Pharmaceuticals, SEP 3, 2024, View Source [SID1234646334]). To introduce its ADC payload molecules, Pacylex will participate in the upcoming ADC & Radiopharmaceuticals Pharma & Biotech Partnering Summit on September 9-10 at the Revere Hotel Boston Common, Boston, Massachusetts, and the 15th World ADC Summit, November 4-7 at the Town & Country Resort in San Diego, California.

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ADCs have emerged as a major category of therapeutics, particularly in the field of oncology. Despite this, only a very limited number of ADC payloads have been used to kill cancer cells. This new class of payload, NMT inhibitors, simultaneously affect multiple processes critical to cancer cell growth and survival and have been shown to regress solid tumor cancers in animal models when coupled with ADCs. Pacylex has exclusive license to 503 NMTis, 27 of which have single-digit nM IC50s against human NMT1. The lead molecule in Pacylex’s NMTi collection, zelenirstat, a first-in-class myristoylation inhibitor, has been shown to inhibit the myristoylation required for assembly, translocation, and/or function of validated targets, such as B-cell receptor, Flt3-cKit complex, EGFR, and VEGFR. Zelenirstat also blocks respiratory Complex I formation in the mitochondria of cancer cells, thereby shutting down oxidative phosphorylation, which is critical for metastasis and cancer stem cells.

Zelenistat is the only clinically validated NMTi; it is well tolerated in Phase 1 patients with refractory/relapsed (r/r) lymphoma and refractory solid tumors. Phase 1 safety, PK, and drug exposure results with oral zelenirstat in 24 heavily pretreated NHL and solid tumor patients showed no dose-limiting toxicities. Patients receiving the recommended Phase 2 dose (RP2D) had significantly better progression-free and overall survival than those treated at lower doses; 57% had stable disease or better for six months or longer, despite cancers originating in 5 different organs. A patient with colorectal cancer who only achieved short-term benefits from the 5 prior lines of therapy, received the RP2D of zelenirstat for 475 days with reductions of approximately 50% in carcinoembryonic antigen (CEA) and tumor volumes. A Phase 2a patient with DLBCL has achieved a partial response and is on their 13th 28-day cycle of zelenirstat.

"The ability of NMTis to interrupt many critical cancer processes simultaneously makes them exciting drug candidates but also excellent potential ADC payloads", said Dr. Michael Weickert, CEO of Pacylex. "Our collection of potent NMTis is one of only two families of molecules shown independently to be potent and on target, and zelenirstat is the only clinically validated NMTi. We want to establish ourselves as a partner of choice for those seeking novel and effective payloads for their ADC programs."

On September 3, 2024 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the second quarter ended June 30, 2024, and provided a business update (Press release, Ascendis Pharma, SEP 3, 2024, View Source [SID1234646284]).

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"The recent FDA approval of YORVIPATH demonstrates why our unrelenting focus on helping patients suffering from hypoparathyroidism and other serious diseases with considerable unmet need is so important for Ascendis," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "Already, two out of our three Endocrine Rare Disease TransCon product candidates have been approved by the FDA and European Commission. Our first, SKYTROFA, has achieved U.S. market value leadership, and, we believe, now with broader market access, remains well positioned to reach blockbuster status in the U.S. alone. Looking forward, we are preparing for our second U.S. launch with YORVIPATH and are on track to report pivotal data in the coming weeks for our third TransCon product candidate, TransCon CNP."

Select Highlights & Anticipated 2024 Milestones

TransCon hGH:
(lonapegsomatropin, marketed as SKYTROFA)
SKYTROFA revenue for the second quarter of 2024 totaled €26.2 million, a 27% year-over-year decrease compared to €35.9 million during the same period in 2023. 134% year-over-year volume growth was offset by the cost associated with broader market access for SKYTROFA which also resulted in a negative adjustment to prior period sales deductions of €27.1 million, where €19.5 million and €7.6 million were attributable to the three months ended March 31, 2024, and periods prior to January 1, 2024, respectively.
SKYTROFA revenue for the first half of 2024 totaled €91.2 million, a 35% year-over-year increase compared to €67.4 million during the same period of 2023. 159% year-over-year volume growth was offset by the cost associated with broader market access for SKYTROFA which also resulted in a negative adjustment to prior period sales deductions of €7.6 million, which were attributable to periods prior to January 1, 2024.
On track to submit a supplemental Biologics License Application to the FDA for adult growth hormone deficiency in the third quarter of 2024.
Topline results from Phase 2 New InsiGHTS Trial in Turner syndrome expected in the fourth quarter of 2024.

TransCon PTH:
(palopegteriparatide, marketed as YORVIPATH)
Received U.S. FDA approval for TransCon PTH, under the brand name YORVIPATH, for the treatment of hypoparathyroidism in adults.
Completing manufacturing of commercial product for the U.S. market and anticipate initial supply will be available in the first quarter of 2025. The Company is in dialogue with the FDA about commercialization of existing manufactured product, which if agreed, could be introduced in the U.S. in the fourth quarter of 2024.
Second quarter YORVIPATH revenue totaled €5.2 million, reflecting the first full quarter of commercial launch in Germany and Austria as well initial revenue in International Markets. Initial revenue in France expected starting in the fourth quarter of 2024.
TransCon CNP
(navepegritide)
Topline data from pivotal ApproaCH Trial expected in the coming weeks, and, if successful, plan to submit a New Drug Application to FDA for children with achondroplasia (age 2-11 years) in the first quarter of 2025.
Plan to complete enrollment in the combination TransCon hGH and TransCon CNP COACH trial of children with achondroplasia (ages 2-11 years) during the third quarter of 2024; topline Week 26 data expected in the second quarter of 2025.
Expect to initiate teACH, a Phase 2 trial in adolescents with achondroplasia, in the fourth quarter of 2024.
Oncology Programs
Presented new and updated results from the ongoing Phase 1/2 IL-Believe Trial of TransCon IL-2 β⁄γ in a poster presentation at ASCO (Free ASCO Whitepaper) 2024. As of the April 16, 2024, data cutoff, 40% of efficacy-evaluable patients (2 out of 5) in the initial cohort of patients with anti-PD-1 refractory melanoma treated with TransCon IL-2 β⁄γ in combination with TransCon TLR7/8 Agonist exhibited confirmed clinical responses with no new safety signals.
Initial results from the Phase 2 dose expansion cohort of the IL-Believe Trial of TransCon IL-2 β⁄γ in combination with chemotherapy in platinum-resistant ovarian cancer (PROC) will be presented later this month at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2024 Congress in Barcelona, Spain.

Financial Update and Outlook Based on Current Plans
As of June 30, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €259 million, compared to €399 million as of December 31, 2023.
Full year 2024 SKYTROFA revenue expected to be €220 million to €240 million.
Expect total operating expenses (SG&A and R&D) to be approximately €600 million for 2024.
Pending launch timing of YORVIPATH in the U.S., expect to be operating cash flow breakeven on a quarterly basis in 2024 or 2025
Subsequent to the quarter end, entered into a $150 million capped synthetic royalty funding agreement with Royalty Pharma relating to net sales of YORVIPATH in the United States. More information on this funding can be found in a separate press release issued today and available here on the Investors & News section of the Ascendis Pharma website.
Second Quarter 2024 Financial Results
Total revenue for the second quarter of 2024 was €36.0 million, compared to €47.4 million during the same period for 2023. Results in the quarter were primarily impacted by a negative adjustment to prior periods’ estimates and assumptions for sales deductions of €27.1 million, where €19.5 million and €7.6 million were attributable to the three months ended March 31, 2024, and periods prior to January 1, 2024, respectively. This was partially offset by increased demand for SKYTROFA in the U.S. and revenue contribution from YORVIPATH. In addition, non-product revenue was €4.6 million in the second quarter of 2024, compared to €11.5 million during the same period for 2023.

Total Revenue
(In EUR’000s) Three Months Ended
June 30, Six Months Ended
June 30,
2024 2023 2024 2023
Revenue from external customers
Commercial sale of products 31,389 35,895 97,888 67,446
Licenses 869 589 25,639 1,203
Other 3,740 10,909 8,365 12,333
Total revenue from external customers 35,998 47,393 131,892 80,982
Research and development (R&D) costs for the second quarter of 2024 were €83.5 million, compared to €105.0 million during the same period in 2023. The decline was largely tied to lower external development costs for TransCon TLR 7/8 Agonist and lower costs for TransCon PTH, as well as lower employee costs as a result of the Eyconis spin-off.

Selling, general, and administrative (SG&A) expenses for the second quarter of 2024 were €74.3 million, compared to €70.3 million during the same period in 2023. The increase was primarily due to higher employee costs, including the impact from commercial expansion.

Total operating expenses for the second quarter of 2024 were €157.8 million compared to €175.3 million during the same period in 2023.

Net finance income for the second quarter of 2024 was €29.4 million compared to a net finance income of €26.4 million during the same period in 2023.

For the second quarter of 2024, Ascendis Pharma reported a net loss of €109.4 million, or €1.91 per share (basic and diluted) compared to a net loss of €121.4 million, or €2.16 per share (basic and diluted) for the same period in 2023.

As of June 30, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €258.7 million compared to €399.4 million as of December 31, 2023. As of June 30, 2024, Ascendis Pharma had 58,231,484 ordinary shares outstanding, including 881,730 ordinary shares represented by ADSs held by the company.

Conference Call and Webcast Information
Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its second quarter 2024 financial results.

Those who would like to participate may access the live webcast here, or register in advance for the teleconference here. The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at View Source A replay of the webcast will be available on this section of the Ascendis Pharma website shortly after conclusion of the event for 30 days.

On September 3, 2024 Nkarta, Inc., a biopharmaceutical company developing engineered natural killer (NK) cell therapies, reported its participation in the following investor conference (Press release, Nkarta, SEP 3, 2024, View Source [SID1234646301]):

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H.C. Wainwright 26th Annual Global Investment Conference
September 10, 2024
12:30 p.m. ET – fireside chat

A simultaneous webcast of each event will be available on the Investors section of Nkarta’s website, www.nkartatx.com, and a replay will be archived on the website for approximately 90 days.

On September 3, 2024 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, cardiovascular and metabolic, autoimmune, ophthalmology and other major diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its PD-1/IL-2α Bispecific Antibody Fusion Protein (R&D code: IBI363) for the treatment of unresectable locally advanced or metastatic melanoma (except choroidal melanoma) in patients who have progressed after at least one line of systemic therapy, which must include a PD-1/L1 inhibitor (Press release, Innovent Biologics, SEP 3, 2024, View Source [SID1234646318]). Phase 1/2 clinical trials are currently underway in China, the U.S., and Australia to assess IBI363’s efficacy and safety in various advanced malignant tumors.

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At the ESMO (Free ESMO Whitepaper) Plenary meeting on June 14, 2024, Innovent presented promising efficacy signals in melanoma patients who had previously undergone immunotherapy: 37 patients with melanoma who had previously received immunotherapy received 1mg/kg of IBI363 and underwent at least one tumor evaluation after baseline, and 11 patients achieved objective responses, including 1 CR and 10 PR, with ORR and DCR of 29.7% and 73.0%, respectively. (Link)

Dr. Hui Zhou, Senior Vice President of Innovent, said, "Melanoma is the most common fatal skin cancer in Europe and the United States. In China, while melanoma is a rare malignant tumor, it has a high fatality rate, and its incidence is steadily increasing each year. Despite the success of immune checkpoint inhibitors in the treatment of melanoma, there is currently no drug approved for immunotherapy failed melanoma around the world, and the ORR of traditional chemotherapy ± anti-vascular therapy for immunotherapy failed melanoma is only 3.8% to 6.8%, with a median PFS of less than 3 months, and the benefit is very limited[1]-[2]. Therefore, there is an urgent clinical need for patients who have previously failed immunotherapy. As a First in-class PD-1/IL-2α-bias bispecific antibody fusion protein, IBI363 monotherapy has shown encouraging efficacy and a favorable safety profile in melanoma subjects who have previously received immunotherapy. We will continue to explore the efficacy and safety of IBI363 in melanoma to provide more effective clinical treatment for patients with immune-resistant melanoma."

Fast Track Designation (FTD) is a rapid review process designed to facilitate the clinical development of a drug that may treat serious conditions and fulfill an unmet medical need. According to regulations, drug candidates that obtain FTD qualifications will have more opportunities to communicate with the FDA during subsequent drug development and review processes, which will help speed up the clinical development and approval of the drug.

About Melanoma

Melanoma is a malignant tumor that develops from melanocytes and is the fifth most common cause of cancer in the United States[3]. Although melanoma represents only 3% of all skin cancer cases, it has the highest mortality rate and is the most prone to metastasize. In China, both the incidence and mortality rates of melanoma have been steadily rising over the years. According to the classification of the disease site, melanoma is mainly divided into skin melanoma, acral and mucosal melanoma. Chinese melanoma differs greatly from European and American Caucasian melanoma in pathogenesis, biological behavior, histological morphology, treatment and prognosis[4]. For advanced cutaneous and acral melanomas, for those carrying BRAF V600 mutation, BRAF inhibitor combined with MEK inhibitor is the preferred molecular targeted therapy. For patients without a BRAF V600 mutation, comination of chemotherapy and anti-angiogenic drugs may be considered as the first-line treatment. Immunotherapy has not been approved as the first-line treatment indication for advanced melanoma in China. For second-line treatment, therapies not used in the first-line are recommended. If a PD-1 monoclonal antibody was not administered initially, it can be selected for the second-line. In advanced mucosal melanoma, chemotherapy or a combination of PD-1 monoclonal antibody and anti-angiogenic drugs may be considered as first-line options. For patients with BRAF V600 mutation, a BRAF inhibitor ±MEK inhibitor can be chosen. Currently, posterior treatment options for melanoma are very limited[5].

About IBI363 (PD-1/IL-2α)

IBI363 is a first-in-class drug candidate independently developed by Innovent Biologics. Its active ingredient is PD-1/IL-2 bispecific antibody fusion protein. The IL-2 arm of IBI363 has been engineered to maximize efficacy and reduce toxicity, whereas the PD-1 binding arm achieves PD-1 blockade and selective IL-2 delivery. Therefore, IBI363 functions by simultaneously blocking the PD-1/PD-L1 pathway and activating the IL-2 pathway, enabling more precise and efficient targeting and activation of tumor specific T cells. IBI363 has demonstrated notable anti-tumor activity across various tumor-bearing pharmacological models and showed significant antitumor efficacy in both PD-1 resistant and metastatic models. Additionally, IBI363 exhibited a favorable safety profile in preclinical models. Clinical studies of IBI363 are currently underway in China, the United States, and Australia to evaluate its safety, tolerability and preliminary efficacy in subjects with advanced malignancies.