On January 12, 2025 Ascendis Pharma A/S (Nasdaq: ASND) reported a business and strategic roadmap update, including planned 2025 key corporate milestones. Ascendis President and CEO Jan Mikkelsen will present this update tomorrow, January 13, during the 43rd Annual J.P. Morgan Healthcare Conference (Press release, Ascendis Pharma, JAN 12, 2025, View Source [SID1234649615]).

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"Ascendis is well-positioned for rapid revenue growth with the launch of our first two Endocrinology Rare Disease medicines, SKYTROFA and YORVIPATH, and, following our pre-NDA meeting, planned filings for our third, TransCon CNP for achondroplasia," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "Our expanding TransCon technology platform and partnerships demonstrate that our business model built on fast, successful drug development can create substantial stakeholder value."

Selected Key Updates and Milestones

TransCon hGH
(lonapegsomatropin, marketed as SKYTROFA)
SKYTROFA full-year 2024 revenue, with a single indication in pediatric GHD, expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).
U.S. SKYTROFA volume (mg) increased 84% in 2024 year-over-year resulting in an estimated 6.5% market share of the total U.S. growth hormone market for 2024 (based on third party prescription data).
Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025 for FDA review of supplemental BLA for the treatment of adults with growth hormone deficiency; pending approval, U.S. commercial launch planned in the fourth quarter of 2025.
During the third quarter of 2025, plan to submit an Investigational New Drug (IND) application or similar for a basket trial evaluating TransCon hGH in additional indications.
TransCon PTH
(palopegteriparatide, marketed as YORVIPATH)
YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million.
YORVIPATH commercially available for prescription in Germany and Austria since January 2024. Outside Germany and Austria, providing product through early access routes, such as ‘named patient,’ until commercial reimbursement established. ~700 patients on treatment in our Europe Direct and International Markets at the end of 2024.
YORVIPATH commercially available for prescription since late December 2024 in the U.S. As of January 9, 2025, 324 patients enrolled into the Ascendis Signature Access Program or direct with specialty pharmacy, with over half of prescriptions for patients new to YORVIPATH.
Expect commercial launch in at least five additional Europe Direct countries in 2025.
TransCon CNP
(navepegritide)
Following pre-NDA meeting with FDA, plan to submit New Drug Application (NDA) for the treatment of children with achondroplasia during the first quarter of 2025, and submit Marketing Authorisation Application to the European Medicines Agency during the third quarter of 2025.
Presented new data demonstrating significant improvements in leg bowing, a common complication in achondroplasia, observed with TransCon CNP compared to worsening observed with placebo in pivotal ApproaCH Trial.
Topline Week 26 results from Phase 2 COACH Trial (TransCon CNP in combination with TransCon hGH) expected in the second quarter of 2025.
During the fourth quarter of 2025, plan to submit an IND or similar for the treatment of hypochondroplasia.
Expanding the TransCon Platform & Pipeline
New TransCon protein degrader platform designed to enable efficient clearance of hormones, cytokines, and other targets. First planned TransCon protein degrader product candidate designed to normalize excess FGF-23 hormone levels for patients with X-linked hypophosphatemia.
Financial Update
Unaudited preliminary estimate of total full-year 2024 product revenue of ~€226 million:
SKYTROFA full-year 2024 revenue expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).
YORVIPATH full-year 2024 unaudited preliminary revenue estimate
of ~€29 million.
Unaudited preliminary estimate of total full-year total 2024 revenue of ~€364 million
Includes $100 million Novo Nordisk milestone payment as non-product revenue.
December 31, 2024 pro forma cash balance of ~€655 million (based on unaudited preliminary estimate of December 31, 2024 cash balance of €560 million plus expected payment from Novo Nordisk of $100 million).
Presentation at J.P. Morgan Healthcare Conference on Monday, January 13
A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at View Source The presentation will begin at 11:15 a.m. Eastern Time / 8:15 a.m. Pacific Time. A webcast replay will be available for 30 days.

The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at View Source

On January 10, 2025 Defence Therapeutics Inc. ("Defence" or the "Company"), (CSE: DTC, OTCQB: DTCFF, FSE: DTC) a Canadian biopharmaceutical company developing radiopharmaceuticals and ADC products using its proprietary platform and drug delivery technologies in addition to novel immune-oncology vaccines, reported its participation to the Biotech ShowcaseTM conference next week in San Francisco during the J.P. Morgan Healthcare Conference to meet with industry leaders, partners and potential investors (Press release, Defence Therapeutics, JAN 10, 2025, View Source;utm_medium=rss&utm_campaign=defence-to-connect-with-industry-leaders-and-potential-partners-during-the-jp-morgan-healthcare-conference-in-san-francisco-plans-to-expand-the-next-generation-of-radio-immuno-conjugates-in-2025 [SID1234649649]).

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The Biotech Showcase is a premier investor conference committed to creating a platform for biotech companies, offering them a unique opportunity to showcase their innovation and engage one-to-one with investors and biopharmaceuticals executives. The life science conference is taking place from January 13-16 in San Francisco during the JP Morgan Healthcare Conference which is the premier healthcare investment symposium attracting thousands of industry leaders and investors.

"Our CSO, Dr. Maxime Parisotto, and I will be actively participating in the healthcare conference and meetings related in San Francisco to meet with global industry leaders, potential partners and investors to reinforce our network in the US ecosystem and more importantly to clearly demonstrate the strength and importance of our proprietary Accum technology platform in the US life science market. The Accum technology platform has a great potential to play a pivotal role to improve ADCs and in the development of targeted radiopharmaceutical therapies, which we are fully dedicated to expanding in 2025" says Mr. Plouffe, CEO and president of Defence Therapeutics.

The Company is also pleased to announce the closing of the 1st tranche of its previously announced non-brokered private placement (the "Offering") of units of the Company (the "Units") at a price of $0.60 per Unit for aggregate gross proceeds of $300,000 (the "Closing"). Each Unit consists of one common share in the capital of the Company (each, a "Share") and one common share purchase warrant (each whole, a "Warrant"). Each Warrant is exercisable to acquire one additional Share at an exercise price of $0.75 per Share for a period of 24 months from the date of the Closing (the "Warrant Expiry Date").

In connection with the Closing, the Company paid a cash finder’s fee of $24,000 and issued 40,000 finder’s warrants (the "Finder’s Warrants") to certain qualified arm’s length finder. Each Finder’s Warrant is exercisable into one Share at an exercise price of $0.75 per Share on or before the Warrant Expiry Date.

The Company intends to use the net proceeds of the Offering to advance its preclinical and clinical programs and for general working capital. All securities issued in connection with the Offering are subject to a statutory hold period of four months plus a day from their date of issue in accordance with applicable securities legislation.

On January 10, 2025 Naveris, Inc., the leader in precision oncology diagnostics for viral-induced cancers, reported the expanded commercial availability of the NavDx test for Molecular Residual Disease (MRD) detection in anal squamous cell carcinoma (ASCC) patients (Press release, Naveris, JAN 10, 2025, View Source [SID1234649613]). NavDx, Naveris’ proprietary flagship blood test, is the first and only clinically validated circulating tumor TTMV-HPV DNA blood test that provides a non-invasive and precise method that can detect MRD before there is clinical or radiographic evidence of cancer recurrence.

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The commercial expansion coincides with the publication of clinical validity and utility data in the journal Cancers1 that demonstrates the NavDx test’s ability to accurately and reliably detect MRD in HPV-positive anal cancer patients in a multi-center, real-world cohort.

Dr. Barry M. Berger, Chief Medical Officer of Naveris, commented: "With an impressive per-test positive predictive value (PPV) of 98% and a negative predictive value (NPV) of 95%, the NavDx test demonstrates exceptional clinical value in detecting and ruling out disease recurrence in ASCC patients. This offers real utility for physicians, allowing them to accurately assess whether a patient has residual disease and implementing the most appropriate course of care without unnecessary delays. This expanded use of NavDx represents a critical step forward in advancing both precision medicine and the overall management of HPV-related anal cancer."

This new data further reinforces the clinical value of TTMV-HPV DNA testing in HPV-positive cancers, as supported by more than 30 peer reviewed publications validating use of the NavDx test in head and neck cancers.

The expansion of the NavDx test in HPV-positive anal cancer patients also reflects Naveris’ commitment to increasing access to high-quality care. As a blood test, the NavDx test offers a simpler, non-invasive, and more accessible compliment to traditional institution-based surveillance methods and is anticipated to increase healthcare access for those who face challenges in receiving timely, effective cancer care.

"Certain populations, notably those living with HIV and of lower socioeconomic status, are much more likely to develop anal cancer. These populations often face barriers to accessing care under current recurrence surveillance protocols," noted Alice L. Pomponio, Managing Director of the American Cancer Society’s BrightEdge impact investing arm and a Naveris Board Observer. "By bringing the NavDx test to clinic for anal cancer MRD testing, Naveris is addressing an urgent need for early cancer detection solutions and providing health equity for individuals that face challenges to accessing care. Our shared mission of impact investing, leveraging innovation to improve care for underserved populations, can be achieved through a test that will improve clinical care for all patients."

On January 10, 2025 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported its updated strategy for company growth (Press release, Innate Pharma, JAN 10, 2025, View Source [SID1234649612]). This comprehensive plan is anchored in three key pillars designed to drive sustainable growth, foster innovation, and deliver transformative therapies to patients worldwide.

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"Our updated strategy underscores Innate Pharma’s unwavering dedication to innovation, collaboration, and delivering transformative therapies for patients worldwide," said Jonathan Dickinson, Chief Executive Officer of Innate Pharma. "With a focus on our ANKET platform and ADC programs, we are poised to redefine the landscape of immunotherapy, addressing unmet needs in oncology and autoimmune diseases. This roadmap reflects our steadfast commitment to advancing science, strengthening partnerships, and maximizing value for patients and shareholders alike as we look toward a brighter future in immuno-oncology."

Three Pillars of Growth

Drive innovation with first-in-class ANKET Platform: A new era in NK cell therapeutics is at the heart of Innate Pharma’s strategy, based around its validated, proprietary Antibody-based NK Cell Engager Therapeutics (ANKET) platform. This cutting-edge technology with anticipated applications in hematologic malignancies, solid tumors, and autoimmune diseases leverages the advantages of harnessing NK cell effector functions and can create proliferation of NK cells. By advancing its clinical pipeline, Innate aims to exploit therapeutic possibilities in oncology and autoimmune diseases. IPH6501, Innate’s proprietary ANKET is currently being investigated in a Phase 1/2 study in patients with CD20-expressing non-Hodgkin’s Lymphoma.

Accelerate development of differentiated Antibody-Drug Conjugates (ADCs): Innate Pharma is advancing its ADC programs to develop differentiated and highly targeted treatments that combine the specificity of monoclonal antibodies with the potency of cytotoxic drugs. IPH4502, Innate’s lead ADC, a novel and differentiated topoisomerase I inhibitor ADC conjugated to exatecan targeting Nectin-4, is being investigated in a Phase 1 trial in patients with advanced solid tumors.

Advance current late-stage assets through partnerships: Partnerships will continue to play a critical role in the Company’s strategy, for broader impact with Innate’s established antibody assets, including lacutamab and monalizumab. Innate is actively seeking a partner to progress lacutamab for patients with advanced forms of T cell lymphomas. Monalizumab, currently in a Phase 3 trial PACIFIC-9 led by AstraZeneca in non-small cell lung cancer, will see readouts by end of 2026. By entrusting these promising therapies to strategic partners, Innate ensures their ongoing development and potential commercialization, maximizing their therapeutic potential and reach.

With this multi-faceted strategy, Innate Pharma believes it is positioned to accelerate its growth trajectory and reinforce its leadership in the immuno-oncology space. The Company’s robust scientific expertise, strategic collaborations, and focus on patient outcomes create a strong foundation for success.

On January 10, 2025 MAIA Biotechnology, Inc., (NYSE American: MAIA) ("MAIA", the "Company"), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, reported that Chief Executive Officer Vlad Vitoc, M.D. will present at the Biotech Showcase 2025 investor conference taking place in San Francisco, California January 13-15, 2025 (Press release, MAIA Biotechnology, JAN 10, 2025, View Source [SID1234649611]). Dr. Vitoc will also host one-on-one meetings with investors throughout the 3-day event. The live event will be followed by a virtual 2-day event on January 21-22, 2025.

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At the conference, Dr. Vitoc will discuss MAIA’s recent clinical developments and targeted value-driving milestones for 2025 and beyond, including:

Expansion of MAIA’s THIO-101 pivotal Phase 2 clinical trial to further assess the efficacy of telomere-targeting agent THIO in advanced non-small cell lung cancer (NSCLC) patients receiving third line (3L) therapy.
Multiple THIO trials planned for additional cancer indications.
Significant market opportunity for THIO in the top tumor types globally.
Biotech Showcase conference details:

Dates:

Live event January 13-15, 2025; Digital event January 21-22, 2025

MAIA presentation:

03:00PM PST on Monday, January 13, 2025*

Conference registration:

All-access and Digital passes available on the conference website

One-on-One Meetings:

Bookings available on the conference website

Webcast:

Join here for live presentation and virtual event streaming

On-demand playback:

Available here 2 hours after the presentation, accessible for 6 months

*Please note that the presentation date and time are subject to change. Attendees can refer to the program agenda for conference updates.

About THIO

THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine (THIO) induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with THIO followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. THIO is presently developed as a third line of treatment for NSCLC for patients that are resistant to checkpoint inhibitors and chemotherapy.