On March 3, 2026 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine, and Merck, known as MSD outside of the United States and Canada, reported an expanded, multi-year collaboration aimed at accelerating the discovery and development of precision medicine biomarkers and supporting Merck’s oncology and potentially broader therapeutic portfolios.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This collaboration builds on our existing relationship and reflects our shared commitment to harnessing the power of multimodal datasets with AI to deliver better options for patients," said Ryan Fukushima, CEO, Data & Apps at Tempus. "We’ve spent years configuring our Lens Platform to seamlessly leverage our library of de-identified multimodal data with the necessary AI computing power to train and fine-tune specific models for healthcare. Working with the great scientists at Merck, we have exciting opportunities to translate the insights from AI models into precision medicine strategies and improve patient outcomes across oncology and beyond."

Under the terms of the agreement, Merck will use Tempus’ de-identified data along with Tempus’ Lens Platform and Workspaces environment, which offers an advanced computational configuration powered by one of the industry’s largest GPU infrastructures, which enables researchers to efficiently conduct complex analyses on training-ready multimodal datasets, generating novel insights to accelerate the development and optimization of candidate therapies at scale.

"The combination of new AI technologies and large curated multimodal data sets are transforming the way we conduct discovery research," said George Addona, Senior Vice President, Discovery, Preclinical Development and Translational Medicine, Merck Research Laboratories. "This collaboration with Tempus positions Merck to advance our precision oncology strategy through the application of the latest AI/ML capabilities to discover novel precision biomarkers, identify mechanisms of cancer cell resistance, and inform rational combinations for drugs in our early pipeline."

(Press release, Tempus, MAR 3, 2026, View Source [SID1234663269])

On March 3, 2026 AN2 Therapeutics, Inc. (Nasdaq: ANTX), a clinical-stage biopharmaceutical company developing novel small molecule therapeutics derived from its boron chemistry platform, reported its plans to expand the development of oral epetraborole into a Phase 2 proof-of-concept clinical study in adults with phlebotomy-dependent polycythemia vera (PV). PV is a blood cancer characterized by overproduction of red blood cells in the bone marrow. This overproduction increases hematocrit which can lead to serious medical complications, including arterial and venous thromboembolic events.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Company’s decision to pursue PV is supported by data from multiple clinical trials of oral epetraborole in healthy volunteers and non-PV patients in which the drug consistently demonstrated early, controlled, sustained, and dose-dependent reductions in hematocrit at potentially clinically meaningful levels for PV.

Epetraborole’s effects were characterized by the following pharmacodynamic and clinical observations:

Consistent hematocrit reductions across multiple clinical populations, including healthy volunteers and nontuberculous mycobacterial (NTM) lung disease patients, with effects sustained over a six-month treatment period
Early onset of hematocrit reduction after dose initiation with durable, stable control and reversibility at treatment cessation
No clinically relevant change in white blood cell counts and minimal change in platelet counts
Demonstrated durable hematocrit reduction in 9-month chronic non-human primate studies
A potentially differentiated mechanism of action, likely acting on globin synthesis rather than directly on heme synthesis
Epetraborole has been generally well tolerated in clinical trials to date at doses anticipated for the treatment of PV.

"We believe epetraborole may offer a differentiated hematological profile that combines hematocrit control via red-cell selectivity, early onset, titratability and oral delivery, attributes that could address key treatment objectives in polycythemia vera and offer patients a new therapeutic option where current approaches fall short," said Eric Easom, Co-Founder, Chairman, President and CEO of AN2 Therapeutics. "This program creates additional, near-term value inflection points within our current runway and broadens our pipeline, which now includes three Phase 2 studies initiating in 2026, and two preclinical oncology compounds that are expected to move into development this year."

"While current therapies are effective for some patients, many continue to have inadequately controlled hematocrit levels and rely on repeated phlebotomy or injectable treatments," said Stan Gerson, M.D., Hematologist and Oncologist at University Hospitals Cleveland Medical Center and Dean and Professor of Medicine at Case Western Reserve University School of Medicine. "As a chronic illness with no cure, PV carries a persistent risk of thrombosis and a substantial symptom burden. There remains a clear need for additional oral treatment options, including those with novel mechanisms of action, that can help manage hematocrit while minimizing treatment burden and long-term tolerability concerns."

The Company is currently proceeding through the regulatory clearance process and anticipates initiating the Phase 2 trial in the third quarter of 2026. The Company expects to provide periodic public data updates as early as the fourth quarter of 2026, subject to regulatory clearance and enrollment progress.

About Polycythemia Vera (PV)

PV is a blood cancer characterized by overproduction of red blood cells in the bone marrow. This overproduction increases hematocrit which can lead to serious medical complications, including arterial and venous thromboembolic events. If untreated, PV can be life-threatening. Despite available therapies, many patients experience uncontrolled hematocrit levels and persistent symptom burden, requiring long term management to maintain adequate disease control. PV is estimated to affect approximately 155,000 people in the U.S.

Webcast Information

AN2 will host a live webcast presentation on Wednesday, March 4, 2026 at 9:30am ET to provide an overview of the PV program. The event will feature Dr. Aaron Gerds, alongside members of the AN2 team. Dr. Gerds is the Associate Professor of Medicine at the Cleveland Clinic Lerner College of Medicine of Case Western Reserve University and serves as Deputy Director for Clinical Research at the Cleveland Clinic Taussig Cancer Institute. He is also Deputy Associate Director for Clinical Research at the Case Comprehensive Cancer Center.

The live webcast of the presentation can be accessed by registering under "Events" in the investors section of the Company’s website at View Source or View Source Upon registration, all participants will receive an email confirmation with a link that will log you in automatically and the option to add it to your calendar. It is recommended that participants log into the webcast approximately 10 minutes prior to the webcast. An archived replay will be available for 30 days following the presentation. The replay will be available on this same link beginning approximately two hours after the event.

About the Phase 2 Study of Epetraborole in PV

This planned Phase 2 study consists of an open-label epetraborole sentinel cohort, an open-label dose optimization cohort for dose selection (Part 1), followed by a double-blind, randomized, placebo-controlled cohort (Part 2), and an optional open-label extension cohort (Part 3). The study is designed to assess the efficacy of oral epetraborole in phlebotomy-dependent adults with PV and its effect on key hematological variables, to optimize a dose regimen on a by-patient level, to assess safety and tolerability, to assess patient-reported outcomes (PROs) using validated PRO instruments, and to assess other key hematological parameters.

About Epetraborole

Epetraborole is a boron-containing, orally available, small molecule that has shown dose and exposure-dependent decreases in hematocrit. Evidence suggests that it operates by reducing production of early-stage erythrocytes (red blood cells) while sparing other cell lineages in the marrow, including white blood cells and platelets. Epetraborole’s clinical data package supporting evaluation in PV is comprehensive, including 10 Phase 1 studies, two Phase 2 studies, and a Phase 2/3 study in NTM lung disease. The drug has been generally well tolerated in prior trials at doses anticipated for PV and, to date, no tolerability barriers to long-term use have been identified. Epetraborole, if approved, would represent a distinct chemical class in both PV and anti-infectives.

(Press release, AN2 Therapeutics, MAR 3, 2026, View Source [SID1234663268])

On March 3, 2026 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), a clinical stage company pioneering immuno-oncology and RNA for the treatment of high risk and advanced cancer, reported that it has entered into an exclusive, worldwide, fully paid-up royalty-free license agreement with Unleash Immuno Oncolytics, Inc. ("Unleash"). TransCode obtained the rights to develop three Unleash drug candidates, UIO-524, UIO-525 and UIO-526, which includes a license of all in-licensed rights held by Unleash to the Unleash drug candidates together with the acquisition of all rights to the drug candidates owned by Unleash. Under the terms of the exclusive license agreement, Unleash will receive a one-time payment of 1,136,364 shares of a new series of non-voting convertible preferred stock of TransCode, convertible into an equal number of shares of common stock of TransCode (the "Preferred Stock"). The Preferred Stock represents 6.8% of TransCode’s common stock on a fully diluted basis assuming conversion of all TransCode preferred stock outstanding.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The lead candidate UIO-524 complements and expands TransCode’s oncology pipeline by introducing a next-generation, biology-driven oncolytic immunotherapy platform designed to address solid tumor indications with high-unmet medical need, beginning with muscle-invasive bladder cancer (MIBC).

MIBC is a significant unmet medical need indication with poor outcomes, limited durable treatment options, and a highly immunosuppressive tumor microenvironment. Bladder cancer represents a multi-billion-dollar global market, with muscle-invasive disease accounting for a disproportionate share of treatment intensity and healthcare costs, creating what TransCode believes is a compelling opportunity for differentiated therapeutic approaches.

Dr. Philippe P. Calais, Pharm.D., Ph.D., Chairman and CEO of TransCode, stated that "As we are advancing our lead asset, TTX-MC138, into a clinical Phase 2a trial, expanding our pipeline with an innovative preclinical technology such as UIO-524 provides us with an additional shot on goal using a next generation oncolytic immunotherapy candidate intended to address a large and attractive market in more aggressive settings such as MIBC."

Tungsten Advisors acted as the exclusive financial advisor.

About Unleash and the Licensing Transaction

UIO-524 is a rationally designed oncolytic adenovirus engineered to selectively replicate within both malignant cells and cancer-associated stroma. The virus delivers a multi-cytokine immune-activating payload comprising CD40-L, 4-1BBL, and IL-21, intended to activate dendritic cells, T cells, and NK cells, and to drive a robust, systemic anti-tumor immune response. UIO-524 is regulated by a proprietary SPARC promoter that is highly active in malignant cells and cancer-associated stromal compartments and which enables biology-driven differentiation. This design enables selective viral replication and localized expression of immune-activating cytokines within the tumor microenvironment.

UIO-524 builds on CG Oncology’s CG0070, the most clinically advanced and successful oncolytic adenovirus to date, demonstrating meaningful activity in non–muscle-invasive bladder cancer (NMIBC). UIO-524 contains a structurally related oncolytic adenovirus backbone, incorporates tumor- and stroma-targeted replication and contains a more comprehensive, multi-cytokine immune payload. This design positions UIO-524 as a next-generation oncolytic immunotherapy candidate intended to address more aggressive disease settings such as MIBC.

A more detailed description of the financing and licensing agreements can be found in TransCode’s Form 8-K filed with the U.S Securities and Exchange Commission.

About TTX-MC138

TTX-MC138 is a first-in-class therapeutic candidate designed to inhibit microRNA-10b, or miR-10b, a microRNA widely believed to be critical to the emergence and progression of many metastatic cancers. TransCode’s Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at a microdose of the drug candidate, suggesting a broad therapeutic window for TTX-MC138.

(Press release, TransCode Therapeutics, MAR 3, 2026, View Source [SID1234663265])

On March 3, 2026 Biogen Inc. (Nasdaq: BIIB) reported that Uptal Patel, Head of Biogen West Coast Hub, will participate in a fireside chat during the Leerink Global Healthcare Conference 2026. The webcast will be live on Tuesday, March 10, 2026, at 1:00 p.m. ET. To access the live webcast, please visit the Investors section of Biogen’s website at investors.biogen.com. An archived version of the webcast will be available for at least 30 days following the presentation.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

(Press release, Biogen, MAR 3, 2026, View Source [SID1234663258])

On March 3, 2026 Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to dramatically improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, reported financial results for the fourth quarter and full year ended December 31, 2025, and provided an update on recent company developments.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Our highest priority is to serve children and adults living with SMA by bringing apitegromab through the regulatory review process as quickly as possible," said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. "To that end, we are encouraged by the FDA’s continued engagement and shared sense of urgency as Novo Nordisk works expeditiously to remediate its Catalent Indiana facility. We are ready to resubmit our apitegromab BLA following successful reinspection of the site by the FDA."

Mr. Hallal continued, "As we prepare to usher in the next phase of innovation for patients with SMA, we continue to strengthen our financial position while aggressively advancing our pipeline and expect 2026 to be a transformative year for Scholar Rock."

Business Highlights and Upcoming Milestones

Apitegromab

Apitegromab is an investigational fully human monoclonal antibody designed to inhibit myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. It is the first and only muscle-targeted therapeutic candidate in spinal muscular atrophy (SMA) to demonstrate a statistically significant and clinically meaningful benefit in a pivotal Phase 3 clinical trial (SAPPHIRE).

SMA Program

● BLA resubmission and U.S. launch, following approval, expected in 2026. A meeting between FDA and Catalent Indiana occurred early in the first quarter of 2026. The meeting was constructive and included a discussion of Novo Nordisk’s progress remediating the Catalent Indiana facility. No additional corrective actions were requested by FDA. Scholar Rock plans to resubmit the apitegromab BLA following a successful reinspection of the site.
● U.S. commercial team preparing for launch. The commercial team is expanding its reach and deepening relationships with key stakeholders, including SMA treatment centers and payers. The team’s focus includes educating on the importance of addressing the full motor unit, which consists of the motor neuron and the muscle.
● European Medicines Agency (EMA) regulatory review ongoing. A decision by EMA on the apitegromab Marketing Authorisation Application (MAA) is expected in mid-2026. The European team continues to engage with key stakeholders on SMA disease awareness and education initiatives. The Company is planning for an apitegromab launch in Europe in the second half of 2026, beginning with Germany.
● Advancing key activities at second fill-finish facility. Technology transfer continues at a second U.S.-based fill-finish facility to strengthen supply continuity and support future commercial demand. Engineering runs are underway with additional manufacturing runs planned through the second quarter of 2026. Scholar Rock expects to submit a supplemental BLA (sBLA) for this fill-finish facility later in 2026.
● Phase 2 OPAL clinical trial ongoing. Enrollment and patient dosing continue in the Phase 2 OPAL study (NCT07047144). The trial is designed to evaluate apitegromab in infants and toddlers with SMA under two years of age who have received an approved SMN1-targeted gene therapy or who are receiving ongoing treatment with an approved SMN2-targeted therapy.
● Development activities for subcutaneous apitegromab progressing. Scholar Rock is advancing a subcutaneous formulation of apitegromab intended to provide optionality for patients as a small volume, self- or caregiver-administered anti-myostatin antibody suitable for an autoinjector. A Phase 1 study in healthy volunteers has been completed, and further development activities are ongoing, including planned FDA and EMA regulatory engagements.
FSHD Program

● Phase 2 FORGE trial on track for initiation in mid-2026. Scholar Rock is developing apitegromab for the treatment of people with facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare, progressive neuromuscular disease characterized by muscle atrophy and functional decline, affecting approximately 30,000 individuals across the U.S. and Europe. The IND application is cleared, and the Company continues to anticipate the initiation of a Phase 2 randomized, double-blind, placebo-controlled trial, called FORGE, in mid-2026.
SRK-439

SRK-439 is a novel, investigational, subcutaneously administered myostatin inhibitor that binds to pro- and latent myostatin with high affinity and selectivity (i.e., no GDF11 or Activin A binding). Based on preclinical data, SRK-439 has the potential to potently inhibit myostatin and increase muscle mass.

● Dosing continues in Phase 1 healthy volunteer study. A Phase 1 study evaluating SRK-439 in healthy volunteers is underway, with topline data expected in the second half of 2026.
Corporate Update

● Secured new debt facility for up to $550 million in non-dilutive capital from funds managed by Blue Owl Capital (NYSE: OWL). This debt facility is expected to support commercialization of apitegromab and strategic advancement of key pipeline programs. The debt facility matures in February 2032, and consists of the following:
o

$100 million, which became available at closing and was used to retire Scholar Rock’s prior debt facility with Oxford Finance;

o

An additional $100 million to be drawn down in the first quarter of 2026;

o

Up to $150 million available upon FDA approval of apitegromab; and

o

An option for additional incremental facilities of up to $200 million at the mutual consent of Scholar Rock and Blue Owl Capital.

Fourth Quarter and Full Year 2025 Financial Results

Scholar Rock reported a net loss of $91.0 million, including stock-based compensation of $19.4 million, for the quarter ended December 31, 2025, compared to a net loss of $66.5 million, including stock-based compensation of $9.5 million, for the quarter ended December 31, 2024. Net loss per common share was $0.88 for the quarter ended December 31, 2025, compared to $0.61 per common share for the quarter ended December 31, 2024. For the full year ended December 31, 2025, Scholar Rock reported a net loss of $377.9 million, including stock-based compensation of $75.6 million, compared to a net loss of $246.3 million for the year ended December 31, 2024, including stock-based compensation of $36.6 million. Net loss per common share was $3.29 for the full year ended December 31, 2025, compared to $2.47 per common share for the full year ended December 31, 2024.

● The Company did not record any revenue for the quarters ended December 31, 2025 and 2024, or for the full years ended December 31, 2025 and 2024.
● Research and development expense was $46.9 million, including $5.3 million in stock-based compensation, for the quarter ended December 31, 2025, compared to $50.4 million, including $4.0 million in stock-based compensation, for the quarter ended December 31, 2024. For the full year ended December 31, 2025, research and development expense was $208.4 million, including $20.7 million in stock-based compensation, compared to $184.5 million, including $16.0 million in stock-based compensation, for the full year ended December 31, 2024.
● General and administrative expense was $45.0 million, including $14.1 million in stock-based compensation, for the quarter ended December 31, 2025, compared to $19.0 million, including $5.5 million in stock-based compensation, for the quarter ended December 31, 2024. For the full year ended December 31, 2025, general and administrative expense was $176.2 million, including $54.9 million in stock-based compensation, compared to $67.5 million, including $20.6 million in stock-based compensation, for the full year ended December 31, 2024.
● As of December 31, 2025, Scholar Rock had cash, cash equivalents, and marketable securities of $367.6 million. This reflects $60.4 million from the exercise of outstanding warrants for the quarter ended December 31, 2025.

Conference Call Information

Scholar Rock will host a conference call and webcast today, Tuesday, March 3, at 8:00 a.m. ET to review its fourth quarter and full year 2025 financial results and discuss recent business updates. To access the live audio webcast, please go to "Events and Presentations" in the Investors section of the Scholar Rock website at View Source

To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call. A replay of the webcast will be available on the Company’s website for approximately 90 days.

(Press release, Scholar Rock, MAR 3, 2026, View Source [SID1234663232])