On January 9, 2026 Ascendis Pharma A/S (Nasdaq: ASND) reported a business and strategic roadmap update, including planned 2026 key corporate milestones. Ascendis President and CEO Jan Mikkelsen will present this update on Monday, January 12, during the 44th Annual J.P. Morgan Healthcare Conference.

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"With our proven TransCon technology platform, strong R&D capabilities, and a maturing commercial infrastructure and financial profile, Ascendis is rapidly transforming into a leading global biopharma company," said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. "Over the next year, this inflection will be driven by further global penetration and commercial expansion, a third potential TransCon product approval with blockbuster potential, and an advancing R&D pipeline of highly differentiated internal and external TransCon product candidates, all of which create the foundation for sustainable long-term growth."

Selected Key Updates and Milestones

TransCon PTH
(palopegteriparatide, marketed as YORVIPATH)
YORVIPATH fourth-quarter 2025 and full-year 2025 unaudited preliminary revenue expected to be ~€187 million and ~€477 million, respectively.
More than 5,300 unique U.S. patient enrollments, with nearly 2,400 unique prescribing healthcare providers at year end.
Outside the U.S., now available commercially or through named patient programs in more than 30 countries, with full commercial launches anticipated in 10 additional countries by year end 2026.
Confirmed product profile for once-weekly TransCon PTH targeting patients receiving stable YORVIPATH doses.
Ongoing label expansion trials through PaTHway60 (adults) and PaTHway Adolescent.
TransCon hGH
(lonapegsomatropin, marketed as SKYTROFA)
SKYTROFA fourth-quarter 2025 and full year 2025 unaudited preliminary revenue expected to be ~€53 million and ~€206 million, respectively.
Received first label expansion in July 2025 with U.S. Food & Drug Administration (FDA) approval for adult growth hormone deficiency (GHD).
Initiated Phase 3 basket trial for additional indications: idiopathic short stature (ISS), SHOX deficiency, Turner syndrome, and small for gestational age (SGA).
TransCon CNP
(navepegritide)
In the U.S., PDUFA goal date of February 28, 2026 for pediatric achondroplasia.
Submitted marketing authorization application (MAA) to the European Medicines Agency (EMA) in October 2025, with a regulatory decision on potential use in pediatric achondroplasia anticipated in the fourth quarter of 2026.

TransCon CNP + TransCon hGH Combination Therapy
(navepegritide plus lonapegsomatropin)

On January 8, 2026, announced Week 52 topline results from Phase 2 COACH Trial, which demonstrated improvements in annualized growth velocity (AGV) across both TransCon CNP treatment-naïve and TransCon CNP-treated children that exceeded the 97th percentile of average stature children, along with improvements in body proportionality and arm span, and a safety profile consistent with those observed for monotherapies of TransCon CNP and TransCon hGH.
In Q4 2025, Ascendis submitted a protocol and held an end of Phase 2 meeting with the FDA regarding a Phase 3 trial of TransCon CNP and TransCon hGH in pediatric achondroplasia.
Week 78 COACH data update anticipated in second quarter of 2026.
Planned new trials to support TransCon CNP + TransCon hGH treatment in additional indications, such as hypochondroplasia.

TransCon IL-2 β/γ
(onvapegleukin alfa)

Expect to report median overall survival (OS) data for a cohort of 70 patients with late-line platinum-resistant ovarian cancer (PROC) from the IL-Believe Trial of TransCon IL-2 β/γ + weekly paclitaxel in the second quarter of this year.
Strategic Collaborations & Investments

Novo Nordisk A/S
Ongoing multi-product collaboration with Novo Nordisk for TransCon technology-based therapies in obesity and metabolic diseases.
Lead program TransCon semaglutide remains on track to enter the clinic as anticipated.
Eyconis, Inc.
Ascendis has granted Eyconis exclusive rights to develop and commercialize TransCon ophthalmology products globally and holds a 41% equity position in the company.
Lead program TransCon aVEGF (EYC-0305) in development for wet AMD and other retinal diseases anticipated to enter the clinic in 2026.
VISEN Pharmaceuticals
Ascendis has granted VISEN Pharmaceuticals exclusive rights to develop and commercialize TransCon hGH, TransCon PTH, and TransCon CNP in Greater China and maintains a 39% ownership interest in the public company.
Biologic License Application (BLA) for TransCon hGH (lonapegsomatropin) as a potential treatment for pediatric growth hormone deficiency (PGHD) was accepted by China’s National Medical Products Administration (NMPA) in March 2024, with an approval decision anticipated in the first quarter of 2026.
Teijin Limited
Ascendis has granted Teijin Limited an exclusive license agreement for the further development and commercialization of TransCon hGH, TransCon PTH, and TransCon CNP for rare endocrinology diseases in Japan.
In August 2025, YORVIPATH received MAA approval from Japan’s Ministry of Health, Labour, and Welfare.

Financial Update

Unaudited preliminary estimate of 2025 financial results:
Total full-year 2025 product revenue of ~€683 million:
YORVIPATH full-year 2025 revenue of ~€477 million
SKYTROFA full-year 2025 revenue of ~€206 million
Total full-year 2025 revenue of ~€720 million
Full-year 2025 gross margin expected to be ~87%
Total full-year 2025 operating expenses of ~€762 million
December 31, 2025 unaudited preliminary cash balance of ~€616 million
Expect operating cash flow of ~€500 million in 2026, based on current plans, exchange rates and excluding any contribution from TransCon CNP
Planned $120 million share repurchase program in 2026

Share Repurchase Program
The Board of Directors of Ascendis has authorized a $120 million Share Repurchase Program. Purchases under the Share Repurchase Program may be made from time to time through a variety of methods, which may include open-market purchases, privately negotiated transactions, or other methods permitted under applicable securities laws. The timing and amount of any repurchases pursuant to the Share Repurchase Program will be determined based on market conditions, share price and other factors. The Share Repurchase Program does not require Ascendis to repurchase any specific number of shares, and may be modified, suspended or terminated at any time without notice.

Presentation at J.P. Morgan Healthcare Conference on Monday, January 12
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 10:30 a.m. Eastern Time / 7:30 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 Investors & News website at View Source

(Press release, Ascendis Pharma, JAN 9, 2026, View Source [SID1234661888])

On January 9, 2026 Aptevo Therapeutics Inc. (NASDAQ:APVO), a clinical-stage biotechnology Company focused on developing novel immune-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported that the Company has entered into a $60 million equity line of credit (ELOC) agreement with Yorkville Advisors Global, LP, strengthening the Company’s financial flexibility as it continues to advance its clinical and preclinical pipeline of multispecific anti-cancer agents that are differentiated by design.

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The facility provides Aptevo with access to capital, allowing the Company to raise funds incrementally, at its discretion, and under market-based conditions. Further, an ELOC offers affordable capital financing with minimal fees and no warrants. Proceeds from the ELOC will be used to support ongoing clinical development, advance preclinical programs, and fund general corporate purposes.

"This agreement offers added flexibility and control over how and when we access capital," said Daphne Taylor, Chief Financial Officer at Aptevo. "Importantly, the fully leveraged facility plus cash on hand is sufficient to fund us for three years, into 2029. During this time, we plan to continue generating clinical data and advancing differentiated assets across the portfolio focusing on execution and value creation."

The ELOC complements Aptevo’s existing capital resources and is designed to support the Company’s disciplined approach to balance sheet management, while preserving optionality around future strategic and growth decisions.

Under the terms of the agreement, Aptevo has the right, but not the obligation, to sell shares to the counterparty from time to time, subject to customary conditions and limitations. The Company is not required to draw on the facility and retains full discretion over its use.

Aptevo’s portfolio includes five CD3-engaging assets anchored by mipletamig, a first-in-class CD123 x CD3 bispecific currently being evaluated in RAINIER, a Phase 1b/2 trial for frontline AML. In total, mipletamig has been evaluated in more than 100 patients across three trials, where mipletamig has consistently demonstrated high remission rates and a favorable safety and tolerability profile, with no observed events of cytokine release syndrome in frontline patients treated to date.

Building on this clinical validation, Aptevo has built a portfolio of tumor-directed CD3 programs, including bispecific candidates APVO442 (PSMA × CD3) for prostate cancer and APVO455 (Nectin-4 × CD3) for solid tumors, as well as trispecific candidates APVO451 and APVO452. All incorporate the Company’s proprietary application and unique use of the CRIS-7-derived CD3 binding domain, designed to enable tumor-focused immune activation with the potential for meaningful antitumor activity while also prioritizing safety and tolerability.

(Press release, Aptevo Therapeutics, JAN 9, 2026, View Source [SID1234661905])

On January 9, 2026 Biolexis Therapeutics, a clinical-stage biotechnology company pioneering an all-oral infrastructure for metabolic disease, reported it will present at the 44th Annual J.P. Morgan Healthcare Conference on Thursday, January 15, 2026 at 9:00am PST.

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The presentation will spotlight Biolexis’ differentiated strategy to overcome the maintenance, side effect, scalability, and access crises with existing market therapies for weight loss. To solve these issues, Biolexis has two clinical-stage programs:

• BLX-7006 — A first-in-human oral small-molecule allosteric GLP-1 receptor agonist based on a new chemical scaffold that is not an orforglipron or danuglipron analog like other existing market solutions. Phase 1 data demonstrates a clean safety profile and predictable once-daily pharmacokinetics, and the asset is now advancing to Phase 2

• BLX-0871 — A first-in-class γ3 isoform-selective AMPK activator designed to preserve lean muscle mass and metabolic durability, entering Phase 1 in January 2026

BLX-7006: Redefining GLP-1 Access

BLX-7006 binds a distinct allosteric pocket on the GLP-1 receptor, enabling cooperative activation while reducing receptor desensitization—a key limitation of peptide-based therapies. As a small molecule, BLX-7006 offers transformative advantages: oral administration without fasting requirements, no cold-chain logistics, and a fundamentally lower cost-of-goods structure enabling global-scale deployment.

BLX-0871: Solving the Muscle Loss Problem

Current GLP-1 therapies produce significant weight loss, but up to 40% of lost mass is lean muscle, driving metabolic adaptation and weight regain. BLX-0871 selectively activates γ3-containing AMPK complexes enriched in skeletal muscle, engaging exercise-associated metabolic pathways while sparing cardiac isoforms. The result: preserved lean mass, enhanced glucose uptake, and sustained metabolic rate.

The All-Oral Combination Advantage

Biolexis is developing the only all-oral dual-mechanism combination currently in clinical development. By pairing GLP-1-mediated appetite suppression with muscle-selective AMPK activation, the company aims to deliver what injectable monotherapies cannot: durable weight loss with preserved muscle mass and metabolic function.

"The GLP-1 revolution has proven we can treat obesity—but injectable peptides alone won’t get us to a billion patients," said David J. Bearss, PhD, Co-founder and Chairman of Biolexis Therapeutics. "Our all-oral platform isn’t an incremental improvement—it’s infrastructure for global scale. And by adding muscle-selective AMPK activation, we’re not just helping patients lose weight, we’re helping them keep it off. That’s the durability breakthrough the field has been waiting for."

Conference Availability

Biolexis management will be available throughout the conference for meetings with pharmaceutical companies, institutional investors, and strategic partners to discuss Phase 2 clinical plans, combination development strategy, and partnership opportunities.

(Press release, Biolexis Therapeutics, JAN 9, 2026, View Source [SID1234661889])

On January 9, 2026 Phanes Therapeutics, Inc. (Phanes), a clinical stage biotech company focused on innovative drug discovery and development in oncology, reported the first clinical data evaluating spevatamig in combination with chemotherapy in frontline (1L) treatment of metastatic pancreatic ductal adenocarcinoma (mPDAC). The data was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI) 2026; Abstract #709. This marks the first public release of Phanes’ clinical trial data from their ongoing U.S. multi-center study with spevatamig.

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The TWINPEAK study (NCT05482893) is an ongoing multi-cohort Phase 1 monotherapy dose escalation, and Phase 2 combination expansion and dose optimization study in patients with GI carcinomas. Combination expansion cohorts include various combinations with chemotherapy and/or an immune-checkpoint inhibitor. As of December 12, 2025, 107 patients have been treated with spevatamig in the US collectively in monotherapy and combination settings. Of these patients, 42 with 1L mPDAC have been treated with spevatamig + GnP across several dosing regimens. Data from the 2 mg/kg weekly (QW) + GnP regimen was presented at ASCO (Free ASCO Whitepaper) GI 2026, with data from dosing regimens >2 mg/kg QW spevatamig + GnP still maturing. Of note:

Spevatamig has demonstrated a favorable safety profile. In monotherapy, no CRS or DLTs were observed. The maximum tolerated dose (MTD) has not been reached in either the monotherapy or combination therapy setting. No Grade ≥ 3 treatment-emergent anemia, neutropenia or thrombocytopenia were observed during the study.
At 2 mg/kg QW spevatamig + GnP dose level, the rates of anemia, neutropenia and thrombocytopenia were comparable to those observed in the GnP treatment arms from pivotal trials. No Grade ≥ 3 treatment-emergent nausea or vomiting events were reported, and no dose reductions or treatment discontinuations due to nausea or vomiting occurred. No CRS was observed.
In the 2 mg/kg QW spevatamig + GnP 1L mPDAC dosing regimen (n=15), the DCR was 93% and the ORR was 40% (6/15 achieved partial response, with 1 patient pending confirmation).
The median progression-free survival (mPFS) was 7.3 months with a 6-month PFS rate of 59%. The median overall survival (mOS) was 13.2 months and still maturing while 6-month OS rate was 93%.
Responses were observed across CLDN18.2 scores ≥ 10% (≥ 2+ staining). Of note, 85% of patients screened met this CLDN18.2 threshold requirement.
Phanes’ data of spevatamig featured at ASCO (Free ASCO Whitepaper) GI 2026 can be found here: View Source

ABOUT SPEVATAMIG
Spevatamig is a first-in-class native IgG-like bispecific antibody (bsAb) targeting claudin 18.2 and CD47. It was granted orphan drug designation (ODD) for the treatment of pancreatic cancer by the FDA in 2022 and was granted Fast Track designation for the treatment of patients with metastatic claudin 18.2-positive pancreatic adenocarcinoma in 2024. In 2023, Phanes entered into a clinical collaboration agreement with Merck (known as MSD outside the US and Canada) to study spevatamig in combination with Merck’s anti-PD-1 therapy, pembrolizumab.

The multi-center Phase 1/2 clinical trial of spevatamig (NCT05482893), known as the TWINPEAK study, is currently evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of spevatamig in patients with advanced gastric, gastroesophageal junction, pancreatic ductal or biliary tract adenocarcinomas. The Phase 2 study of spevatamig has begun in China.

(Press release, Phanes Therapeutics, JAN 9, 2026, View Source [SID1234661906])

On January 9, 2026 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the development of targeted radiotherapeutics for cancer, reported plans to highlight the company’s 2026 strategic initiatives at the upcoming Biotech Showcase, taking place January 12-15, 2026, in San Francisco during the 44th Annual JP Morgan Healthcare Conference. James Caruso, president and CEO of Cellectar, will present a corporate update on Tuesday, January 14, 2025, at 1:30 pm Pacific Time.

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2025 Achievements

Received feedback from the SAWP supporting a Conditional Marketing Authorization (CMA) filing for iopofosine I 131 in WM based upon our CLOVER WaM study
U.S. Food and Drug Administration (FDA) granted Break Through Designation (BTD) for iopofosine I 131 in relapsed/refractory WM
Confirmed traditional accelerated approval pathway with the FDA for iopofosine I 131 in WM
Presented compelling data from the CLOVER-2 Phase 1b study in relapsed/refractory pediatric high-grade glioma (pHGG), demonstrating extended progression-free survival and favorable safety profile
Initiated a Phase 1b clinical study for CLR 125, an Auger-emitting iodine-125 program for the treatment of triple-negative breast cancer (TNBC)
Strengthened supply chain through strategic supply agreements for our next generation Auger- and alpha-emitting radiotherapeutics
Raised approximately $15.2 million through financings and warrant exercises to support pipeline development and regulatory milestones

CEO Commentary

"Our priorities are clear: secure European conditional marketing approval for iopofosine I 131 for WM in early 2027 with commercialization to follow, further validate our PDC platform by executing on our Phase 1b study evaluating CLR 125 for TNBC, and advance our regulatory strategy with the FDA for iopofosine I 131 approval in the U.S. With an established iopofosine I 131 regulatory strategy for both EMA and FDA, and promising data across our pipeline, we are entering 2026 with strong momentum and multiple opportunities," said James Caruso, president and CEO of Cellectar. "We believe these initiatives position Cellectar to deliver meaningful therapies for patients and create significant value for shareholders."

2026 Strategic Initiatives

Regulatory Milestones:
Submit CMA application to EMA for iopofosine I 131 in WM in 3Q 2026, with potential European market approval in early 2027
Advance NDA preparations for U.S. accelerated approval
Clinical Development:
Enroll patients in the Phase 1b study of CLR 125 in TNBC with interim data expected in mid-2026
Present final findings and subset analysis from the CLOVER WaM Phase 2 study of iopofosine I 131
Prepare actinium-based CLR 225 for first-in-human trials in pancreatic cancer
Pipeline Expansion:
Progress additional PDC-based radiotherapeutics into preclinical and IND-enabling studies
Partnerships:
Evaluate strategic collaborations for commercialization of iopofosine I 131
Financial Strategy:
Continue disciplined capital management and explore non-dilutive funding opportunities

"Looking ahead, we are focused on receiving a conditional marketing approval in 2027 from the EMA impacting approximately thirty countries, which collectively possess a larger WM population than the U.S. In parallel, we remain committed to advancing our regulatory strategy with the FDA fully understanding the tremendous incremental value it potentially represents for all Cellectar stakeholders. The FDA-recommended post-BTKi indication positions iopofosine I 131 as a treatment option as early as the second line, substantially expanding the available patients in the U.S. market. Additionally, we are actively recruiting patients for our Phase 1b study evaluating CLR 125, our Auger-based radiotherapeutic for the treatment of TNBC, which builds on strong preclinical data showing growth inhibition and tumor volume reduction in this challenging-to-treat cancer," concluded Mr. Caruso.

Mr. Caruso’s Biotech Showcase presentation will be live webcast and can be accessed HERE. A replay of the presentation will be available on the Events section of the company’s Investor Relations website.

(Press release, Cellectar Biosciences, JAN 9, 2026, View Source [SID1234661890])