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 Ouro Medicines, a biotechnology company developing immune reset therapeutics for people living with chronic immune-mediated diseases, reported its launch with $120 million in funding (Press release, Ouro Medicines, JAN 10, 2025, View Source [SID1234656164]). The company was founded by Monograph Capital in partnership with GSK plc. The Series A was co-led by TPG Life Sciences Innovations, NEA, and Norwest Venture Partners, with participation from Monograph Capital, GSK, UPMC Enterprises, Boyu/Zoo Capital, LongRiver Investments, and other unnamed investors.

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Chronic immune-mediated diseases encompass an array of conditions where the body’s own immune cells play a driving role. This category includes B cell mediated diseases, which make up the company’s immediate focus. Current treatments for such diseases generally require ongoing immunosuppressive therapy and may be minimally effective with high levels of toxicity.

Due to their high potency, specificity, and relative ease of administration and manufacture, T cell engager antibodies (TCEs) have emerged as a promising off-the-shelf therapeutic modality for targeting and depleting specific pathogenic cell populations that drive immune-mediated diseases. By depleting target cell populations, TCEs might be used to reset the immune system to a state before those cells became pathogenic. This approach has potential to create long periods of remission where immunosuppression is unnecessary.

"We see a tremendous opportunity to define the future of treatment for people with immune-mediated diseases because the standard of care today leaves a lot to be desired," said Jaideep Dudani, CEO, Ouro Medicines and Portfolio Principal, Monograph Capital. "As a class, we believe TCEs have properties that make it possible to unlock immune system resets with periods of durable remission and without the need for ongoing immunosuppression. Being able to precisely target cell populations with a high-potency therapeutic is crucial to depleting whole lineages of pathogenic cell populations so that we can achieve immune reset."

Ouro’s most advanced product candidate is OM336, a BCMA-directed, bispecific T cell engager recently licensed from Keymed Biosciences (2162.HK). BCMA is highly expressed across several B cell subtypes that play a role in driving multiple indications where high specificity and potency are needed to achieve near-complete B cell depletion.

Keymed Biosciences is currently studying OM336 (CM336) in an ongoing Phase 2 expansion study in multiple myeloma in China. Ouro holds exclusive rights outside of Greater China and intends to start its first Phase 1 study in 2025. Examples of B cell mediated diseases include systemic lupus erythematosus, scleroderma, rheumatoid arthritis, Sjögren’s, and myositis. Supportive proof of concept data has been generated from the off-label use of BCMA-directed TCEs in these indications.

"TPG has been following the theme of deep B cell depletion since initial data came from CAR-T cell therapies," said Shinichiro Fuse, PhD, Business Unit Partner with TPG Life Sciences Innovations. "In Ouro, we found a leadership team and a group of advisors who bring deep knowledge of immune-mediated diseases and the key modalities being investigated for these indications, as well as a wealth of experience in both drug discovery and clinical development. We are thrilled to support the company as it works to develop immune reset therapeutics that address serious unmet needs for patients."

Behind its lead program, Ouro intends to advance a broad set of discovery programs targeting additional dimensions of B cell biology. These programs probe unique subsets of B cells compared to existing therapies in the clinic. Additionally, Ouro’s protein engineering expertise is being applied to next-generation versions of novel TCEs to continue to drive innovative treatments for chronic immune-mediated diseases.

"Monograph has high conviction that immune reset has the potential to change the course of many autoimmune diseases," said Tim Funnell, DPhil, Member, Ouro Medicines Board of Directors and Partner, Monograph Capital. "We started Ouro with an intention of building a discovery platform to unlock immune reset therapeutics, and we have been excited to accelerate the company’s development by bringing in OM336, which we believe has great promise based on the preclinical profile and clinical data we’ve seen in oncology. The combination of learnings from the OM336 clinical program and Ouro’s discovery activities will give us a strong foundation as we aim to build the leading immune reset therapeutics company."

Ouro has assembled a leadership team with expertise in a range of relevant diseases and modalities, including:

Jaideep Dudani, PhD, Chief Executive Officer. Dudani was most recently part of the founding team of Human Immunology Biosciences (HI-Bio), where he led portfolio development and strategy prior to the acquisition by Biogen, and has played cross-functional roles across R&D, strategy, and business development throughout his career. Dudani is also a Portfolio Principal at Monograph Capital.
Neely Mozaffarian, MD, PhD, Chief Medical Officer. Mozaffarian was previously Chief Medical Officer at GentiBio and Atomwise, with large pharma experience at Johnson & Johnson, Gilead, Eli Lilly, and AbbVie, leading drug development of biologics and small molecules.
Christina Carlson, JD, Chief Administrative Officer. Carlson most recently served as General Counsel of Human Immunology Biosciences (HI-Bio) and brings nearly 20 years of pharmaceutical and legal experience to her role at Ouro.
Kevin P. Baker, PhD, Chief Development Officer. Baker was previously Chief Development Officer at Pionyr Immunotherapeutics, where he led a team developing monoclonal antibodies and proteins to modulate the tumor microenvironment, and has deep experience across research programs in immune-oncology and immunology.
Ouro’s Board of Directors includes Shinichiro Fuse, PhD (TPG), Matt McAviney, MD (NEA), Brian Matesic, MD (Norwest), Tim Funnell, DPhil (Monograph), and Bo Chen, PhD (Keymed).

Supporting the Ouro leadership team are several deeply experienced clinical advisors, including John Davis, MD, MPH, President, Norte Health Consulting and former scientific advisory board member of HI-Bio; Matthew Baker, MD, MS, Clinical Chief in the Division of Immunology and Rheumatology at Stanford University and the Co-Director of the Stanford Multidisciplinary Sarcoidosis Program; and Stanford Peng, MD, PhD, most recently President and Head of Research & Development at Alpine Immune Sciences.

On January 10, 2025 TJ Biopharma ("TJ Bio" or "Company"), a fully integrated biotech company in China that develops, manufactures, and commercializes through partnership on innovative biologics for autoimmune diseases, oncology and metabolic diseases, reported that the Biologics License Application (BLA) submission for felzartamab, an investigational differentiated CD38 antibody, has been accepted for review by China’s National Medical Products Administration (NMPA) for the treatment of multiple myeloma (MM) (Press release, TJ Bio, JAN 10, 2025, View Source [SID1234654001]). This milestone, following the recent BLA submission for eftansomatropin alfa, demonstrates TJ Bio’s proven capability to advance late-stage pipeline products through its full-fledged R&D and manufacturing infrastructure.

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In China, where multiple myeloma affects over 160,000 patients, there remains a significant unmet need for more accessible treatment options. Felzartamab’s Phase 3 registration studies have demonstrated a differentiated profile with comparable efficacy to existing therapies, while offering advantages in safety through lower and less severe infusion-related reactions and improved convenience with shorter infusion time from 6 hours to 1.5 hours, particularly benefiting elderly patients who constitute a large portion of the multiple myeloma population.

With TJ Bio’s state-of-the-art GMP facility in Hangzhou receiving the Drug Manufacturing Permit (License A) in August 2024, felzartamab is on track to become China’s first domestically manufactured CD38 therapy. This manufacturing capability ensures reliable domestic supply to meet the growing demand for innovative biologics while providing Chinese patients with more accessible treatment options.

"This BLA acceptance for felzartamab, coming shortly after eftansomatropin alfa, demonstrates TJ Bio’s strong execution capabilities and our commitment to advancing innovative therapies," said Dr. Jingwu Zang, Founder and Chairman of TJ Bio. "While this first regulatory milestone positions us to potentially deliver China’s first domestically manufactured CD38 therapy, we are particularly excited about felzartamab’s broader potential in autoimmune diseases. As we advance its development as a novel plasma cell depletion therapy across multiple autoimmune indications, these consecutive BLA submissions and our newly licensed manufacturing facility exemplify our ability to execute on our vision of bringing innovative biologics to patients in need."

About Felzartamab
Felzartamab is an investigational therapeutic human monoclonal antibody directed against CD38, a protein expressed on mature plasma cells. Scientific research suggests that anti-CD38 antibodies have therapeutic potential in multiple disease areas across immuno-oncology and autoimmune diseases. Beyond its initial indication in multiple myeloma, felzartamab has been shown in clinical studies to selectively deplete CD38+ plasma cells, which may allow applications that ultimately improve clinical outcomes in a broad range of diseases driven by pathogenic antibodies.

Felzartamab was originally developed by MorphoSys AG (now MorphoSys GmbH, a Novartis company). TJ Bio owns the exclusive rights for development and commercialization of felzartamab for all indications in Greater China, which encompasses Mainland China, Hong Kong, Macao, and Taiwan. Human Immunology Biosciences (HI-Bio) exclusively licensed the rights to develop and commercialize felzartamab across all indications in all countries and territories excluding Greater China. Biogen acquired HI-Bio in July 2024.

About Multiple Myeloma

Multiple myeloma is a hematological malignancy that affects plasma cells, with over 160,000 patients in China requiring treatment. The disease primarily affects elderly patients, who are particularly vulnerable to treatment-related complications. Despite recent therapeutic advances, there remains a significant need for treatments that combine efficacy with improved safety profiles and greater convenience.

The current treatment landscape in China faces challenges including high rates of infusion-related reactions with existing therapies, lengthy infusion times that burden both patients and healthcare systems, and limited access to innovative treatments due to cost considerations. These factors underscore the urgent need for domestically manufactured therapies that can provide effective, safer, and more accessible treatment options.

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."