On March 20, 2024 Antennova, a clinical-stage biotech company focused on oncology reported completion of the first dosing cohort in the Phase I study for the anti-CD24 antibody, ATN-031 (also known as ATG-031) (Press release, Antengene, MAR 20, 2024, View Source [SID1234641307]). The dose escalation trial is evaluating ATN-031 in patients with advanced solid tumors or B-cell non-Hodgkin’s lymphoma (B-NHL), (NCT06028373). The PERFORM trial is being conducted at four cancer centers in the U.S., led by The University of Texas MD Anderson Cancer Center.

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!

A total of five late stage cancer patients have been enrolled based on the Bayesian Optimal Interval (BOIN) design of the trial in the first dosing cohort. To date, no dose-limiting toxicities (DLTs) have been reported among the 5 patients. Tumor shrinkage based on CT scan was observed in one heavily pre-treated patient (7 prior lines of therapy).

"Introducing the first anti-CD24 program for late stage cancer patients in the U.S. is a very important milestone for Antennova. We are especially grateful to the participation in the study by our patients and study centers, and encouraged to observe early clinical activity in this heavily pre-treated patient population based on clinical evaluation and translational data. We are working closely with study sites and investigators at MD Anderson, the University of California, San Francisco, the University of Colorado, and Yale University Cancer Center and look forward to providing periodic updates and presentations at major international medical conferences throughout the study." commented by Jay Mei, M.D., Ph. D., Founder and Chairman of the Board.

The PERFORM trial is a first-in-human, multi-center, open-label, Phase I dose-finding/escalation study of ATN-031 in patients with advanced solid tumors or B-NHL. The study’s primary objective is to evaluate the safety and tolerability of ATN-031 as a monotherapy, and determine the appropriate dose for Phase II studies. The secondary objective is to characterize the pharmacology, evaluate the immunogenicity, and assess the preliminary efficacy of ATN-031.

About ATN-031

ATN-031 is a first-in-class humanized anti-CD24 monoclonal antibody which inhibits the "don’t eat me" signal while stimulating the "eat me" signal, and enhances macrophage-mediated phagocytosis of cancer cells. Tumor cells evade the surveillance of the human immune system by over-expressing "don’t eat me" surface proteins that signal macrophages to prevent the detection and phagocytosis of cancer cells. CD24 is a prominent "don’t eat me" signal that plays a significant role in tumor immune evasion by suppressing macrophage-mediated phagocytosis. Compared to CD47, another "don’t eat me" target, CD24, has a more markedly restricted distribution in normal tissues and higher expression in cancerous tissues, especially solid tumors. Importantly, CD24 is differentiated from CD47 because it is not expressed on human red blood cells, allowing for a wider therapeutic window and minimal on-target-off-tumor toxicity.

CD24 acts as a novel innate immune checkpoint, orchestrating immune evasion through its interaction with the inhibitory receptor Siglec-10 (sialic-acid-binding Ig-like lectin 10), expressed on tumor-associated macrophages (TAMs). Preclinical data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in 2023 (AACR 2023) and the Society for Immunotherapy in Cancer Annual Meeting in 2022 (SITC 2022) demonstrated that ATN-031 can specifically bind to CD24 with nM affinity and block the interaction of CD24 and Siglec-10. Furthermore, ATN-31 induces efficient phagocytosis with a picomolar EC50 and stimulates the pro-inflammatory cytokines production by macrophages.

On March 19, 2024 Kiromic BioPharma, Inc. (OTCQB: KRBP) ("Kiromic" or the "Company") reported early efficacy results showing primary lung tumor stabilization and the complete disappearance of brain metastases in the second patient in the Company’s Deltacel-01 Phase 1 clinical trial (Press release, Kiromic, MAR 19, 2024, View Source [SID1234641267]). Deltacel-01 is evaluating Deltacel (KB-GDT-01), Kiromic’s allogeneic, off-the-shelf, Gamma Delta T-cell (GDT) therapy, in patients with stage 4 metastatic non-small cell lung cancer (NSCLC).

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!

Preliminary results obtained six weeks from the beginning of treatment show Deltacel’s efficacy in controlling tumor growth, as well as favorable safety and tolerability. Stable disease status was confirmed by CT scan. An MRI scan of the patient’s brain performed before treatment began noted new onset metastatic disease, which completely resolved on repeat MRI imaging performed six weeks from the beginning of treatment. This patient is being treated at the Beverly Hills Cancer Center (BHCC).

"These six-week results in the second patient enrolled in Deltacel-01 are extremely promising. Along with preliminary results from the first patient we previously reported, they strengthen the candidacy of Deltacel as a potential treatment for patients with advanced disease. The complete eradication of this patient’s preexisting brain metastases supports a growing body of evidence for the benefit of our therapy and could also support Deltacel-01 crossing the blood-brain barrier," said Pietro Bersani, Chief Executive Officer of Kiromic BioPharma.

"These preliminary results, especially the CT and MRI scan findings and the absence of dose-limiting toxicities, underscore the promise of Deltacel as a potentially efficacious therapy. We are cautiously optimistic given we have achieved a stable disease status for this patient. This aligns with our center’s commitment to bringing pioneering care for patients with few or no available treatment options," commented Afshin Eli Gabayan, M.D., Medical Oncologist, Medical Director, and Principal Investigator at BHCC.

Kiromic expects to announce initial safety, tolerability, and preliminary efficacy results from the third patient enrolled in Deltacel-01 by the end of March.

About Deltacel-01

In Kiromic’s open-label Phase 1 clinical trial, titled "Phase 1 Trial Evaluating the Safety and Tolerability of Gamma Delta T Cell Infusions in Combination With Low Dose Radiotherapy in Subjects With Stage 4 Metastatic Non-Small Cell Lung Cancer" (NCT06069570), patients with stage 4 NSCLC will receive two intravenous infusions of Deltacel with four courses of low-dose, localized radiation over a 10-day period. The primary objective of the Deltacel-01 trial is to evaluate safety, while secondary measurements include objective response, progression-free survival, overall survival, time to progression, time to treatment response and disease control rates.

About Deltacel

Deltacel (KB-GDT-01) is an investigational gamma delta T-cell (GDT) therapy currently in the Deltacel-01 Phase 1 trial for the treatment of stage 4 metastatic NSCLC. An allogeneic product consisting of unmodified, donor-derived gamma delta T cells, Deltacel is the leading candidate in Kiromic’s GDT platform. Deltacel is designed to exploit the natural potency of GDT cells to target solid cancers, with an initial clinical focus on NSCLC, which represents about 80% to 85% of all lung cancer cases. Data from two preclinical studies demonstrated Deltacel’s favorable safety and efficacy profile when it was combined with low-dose radiation.

On March 19, 2024 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotech company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to improving survival outcomes for patients with cancer, reported that it will present new in vitro data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024, taking place on April 5-10 in San Diego, California (Press release, MaaT Pharma, MAR 19, 2024, View Source [SID1234641268]).

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!

MaaT034, a ground-breaking full ecosystem synthetic microbiota product, is the first candidate coming​ from the​ Company’s MET-C platform. Produced using a co-culturing technology and for large scale manufacturing, MaaT034 is being developed to improve responses to immunotherapy for patients with solid tumors in combination with an ICI (Immune Checkpoint Inhibitors) treatment. The first-in-human study is planned for 2025, with clinical batches currently being produced in 2024.

The Company will detail the new preclinical data in a press release on Monday, April 8th, 2024.

AACR Poster Presentation details:

Title: Evaluation of a new co-cultured microbiome ecosystem therapy candidate (MaaT034) for clinical testing as adjuvant/neoadjuvant to immune checkpoint inhibitors in solid tumors
Session Category:Immunology
Session Title: Microbiome, Inflammation, and Cancer
Session Date and Time: Wednesday Apr 10, 2024, 9:00 AM – 12:30 PM
Location:Poster Section 2
Poster Board Number:18
Published Abstract Number:6687

On March 19, 2024 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported full year 2023 financial results and business updates (Press release, Precigen, MAR 19, 2024, View Source [SID1234641269]).

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!

"2024 is poised to be a transformative year for Precigen, as we are on track to present pivotal Phase 2 data in the second quarter and submit a BLA for PRGN-2012 in the second half, a milestone bolstered by the Breakthrough Therapy Designation and accelerated approval pathway granted by the FDA," said Helen Sabzevari, PhD, President and CEO of Precigen. "We are preparing our manufacturing facility and commercial operations in anticipation of the launch of PRGN-2012 in 2025. We are also looking forward to exciting updates from our UltraCAR-T programs, which offer a novel approach compared to traditional CAR-T therapies and have garnered significant interest from potential partners due to the safety, preliminary efficacy, and manufacturing advantages."

"With multiple value inflection points anticipated in 2024, we remain steadfastly committed to a strategy of sound financial management," said Harry Thomasian Jr., CFO of Precigen. "We are evaluating various financing opportunities to strengthen our balance sheet as we prepare our lead asset PRGN-2012 for potential commercial launch in 2025."

Key Program Highlights

AdenoVerse Immunotherapies

· PRGN-2012 in RRP: PRGN-2012 is an investigational off-the-shelf AdenoVerse immunotherapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent respiratory papillomatosis (RRP). PRGN-2012 has received Breakthrough Therapy Designation and Orphan Drug Designation from the US Food and Drug Administration (FDA) and Orphan Drug Designation from the European Commission.

o PRGN-2012 is currently under investigation in a Phase 1/2 pivotal single-arm study in adult patients with RRP (clinical trial identifier: NCT04724980).

o PRGN-2012 demonstrated strong efficacy and a favorable safety profile in the Phase 1 portion of the study with 50% of patients (N=12) in durable and ongoing Complete Response more than two years after PRGN-2012 treatment. Results of the Phase 1 portion of the Phase 1/2 study were published in the peer-reviewed journal, Science Translational Medicine, a leading publication from the American Association for the Advancement of Science (AAAS).

o Enrollment and dosing in the Phase 2 portion of the study is complete and a Phase 2 data presentation is anticipated in the second quarter of 2024.

o The Company has received agreement from the FDA that PRGN-2012 is eligible for consideration of a rolling Biologics License Application (BLA) review. A planned BLA submission under an accelerated approval pathway is anticipated in the second half of 2024.

o Commercial readiness preparations are underway for a potential launch in 2025.

· PRGN-2009 in OPSCC and Cervical Cancer: PRGN-2009 is an investigational off-the-shelf AdenoVerse immunotherapy designed to activate the immune system to recognize and target HPV-associated cancers.

o The Phase 2 study of PRGN-2009 in combination with pembrolizumab in newly diagnosed patients with HPV-associated oropharyngeal squamous cell carcinoma (OPSCC) is enrolling patients (clinical trial identifier: NCT05996523).

o The Phase 2 randomized, open-label study of PRGN-2009 in combination with pembrolizumab in patients with HPV-associated recurrent/metastatic cervical cancer is active and recruiting patients (clinical trial identifier: NCT06157151).

UltraCAR-T Cell Therapies

· PRGN-3006 in AML/MDS: PRGN-3006 is an investigational multigenic, autologous chimeric antigen receptor T cell (CAR-T) therapy engineered to simultaneously express a CAR specifically targeting CD33, membrane bound IL-15 (mbIL15), and a safety/kill switch. PRGN-3006 has been granted Orphan Drug Designation in patients with acute myeloid leukemia (AML) and Fast Track Designation in patients with relapsed/refractory (r/r) AML by the FDA.

o PRGN-3006 is currently under investigation in a Phase 1b dose expansion clinical trial (clinical trial identifier: NCT03927261) for the treatment of patients with r/r AML or higher-risk myelodysplastic syndromes (MDS).

o The first-in-human Phase 1 dose escalation study data show that PRGN-3006 was well-tolerated with no dose-limiting toxicities (DLTs) and a 27% objective response rate (ORR) in heavily pre-treated r/r AML patients infused following lymphodepletion.

o An interim Phase 1b dose expansion data presentation is anticipated in the second half of 2024.

o PRGN-3005 in Ovarian Cancer: PRGN-3005 is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR specifically targeting the unshed portion of MUC16, mbIL15, and a safety/kill switch.

o The Phase 1b dose expansion portion of the Phase 1/1b study is ongoing (clinical trial identifier: NCT03907527).

o PRGN-3007 in Advanced ROR1+ Hematological and Solid Tumors: PRGN-3007, based on the next generation UltraCAR-T platform, is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), mbIL15, a safety/kill switch, and a novel mechanism for the intrinsic blockade of PD-1 gene expression.

o The Phase 1 dose escalation portion of the Phase 1/1b study is ongoing (clinical trial identifier: NCT05694364).

o A preliminary Phase 1 dose escalation data presentation is anticipated by the end of 2024.

Financial Highlights

o Cash, cash equivalents, short-term and long-term investments totaled $62.9 million as of December 31, 2023.

o Selling, general, and administrative (SG&A) costs decreased versus the prior year, 16% for the twelve months ended December 31, 2023.

Full Year 2023 Financial Results Compared to Prior Year Period

Research and development expenses increased $1.4 million, or 3.1%, compared to year ended December 31, 2022. Salaries, benefits, and other personnel costs increased $2.8 million due to an increase in the hiring of employees to support the growth in the Company’s development activities, and to a lesser extent, increases in salaries of our continuing employees. These increases were offset by less expenses incurred related to preclinical research programs for the comparable period.

SG&A expenses decreased $7.6 million, or 15.8%, compared to the year ended December 31, 2022. This decrease was primarily driven by a reduction in professional fees of $6.5 million, due to decreased legal fees associated with certain litigation matters, and $0.7 million decreased insurance-related premiums.

Total revenues decreased $20.7 million, or 76.9%, compared to the year ended December 31, 2022. Collaboration and licensing revenues decreased $14.6 million, or 99.5%, compared to the year ended December 31, 2022, primarily due to the prior year period non-cash recognition of revenue related to historical collaboration agreements for which revenue was previously deferred. Product and services revenues decreased $5.9 million, or 48.8%, compared to the year ended December 31, 2022. This decrease is related to reductions in services performed at Exemplar as well as the recognition of revenue in the first quarter of 2022 related to agreements for which revenue was previously deferred that did not occur in 2023.

Total other income, net, increased $8.5 million, compared to the year ended December 31, 2022. This was primarily due to $6.3 million in reduced interest expense associated with the Convertible Notes as they were fully retired in the second quarter of 2023, and $3.1 million increased interest income due to higher interest rates on investments. This increase was partially offset by a $0.9 million decrease in gain recorded on the early retirement of a portion of our Convertible Notes compared to the year ended December 31, 2022.

The Company recorded a $10.4 million impairment charge in the fourth quarter of 2023 related to its Exemplar subsidiary as a result of the Company’s annual goodwill impairment test.

Loss from continuing operations was $95.9 million, or $(0.39) per basic and diluted share, compared to loss from continuing operations of $79.8 million, or $(0.40) per basic and diluted share, in year ended December 31, 2022.

On March 19, 2024 Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, reported financial results and corporate updates for the full year ended December 31, 2023 (Press release, Scholar Rock, MAR 19, 2024, View Source [SID1234641270]).

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!

"In 2023, Scholar Rock made significant progress across our industry-leading antimyostatin pipeline, and we are poised for a transformational year ahead. We remain on track to release topline data for our Phase 3 SAPPHIRE trial of apitegromab in spinal muscular atrophy in the fourth quarter of 2024, which if successful, will be the foundation of our neuromuscular franchise. Additionally, we remain on track to initiate a Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 RA in mid-2024, as we continue to move SRK-439, our novel preclinical myostatin inhibitor for obesity, toward IND," said Jay Backstrom, M.D., M.P.H., President & Chief Executive Officer of Scholar Rock. "As the only company to show clinical proof of concept in SMA with our selective antimyostatin approach, we are well positioned to further extend our leadership with a potential launch in SMA and clinical data in obesity anticipated in 2025."

Company Highlights and Upcoming Milestones

Spinal Muscular Atrophy (SMA) Program

Apitegromab is an investigational, fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle and is being developed as a potential first muscle-targeted therapy for the treatment of SMA. Apitegromab is the only muscle-targeted therapy candidate to show clinical proof-of-concept in SMA.

· On track to announce topline data from the Phase 3 SAPPHIRE clinical trial in 4Q 2024. In September 2023, the Company announced that it completed enrollment of SAPPHIRE. Scholar Rock remains on track to report topline data from the Phase 3 trial in the fourth quarter of 2024. If the trial is successful and apitegromab is approved, the Company expects to initiate a commercial product launch in 2025.

· Presented two posters at the Muscular Dystrophy Association Conference 2024 (MDA) in March 2024. Earlier this month, Scholar Rock presented 36-month extension data from the Phase 2 TOPAZ trial at MDA showing long-term substantial and sustained improvements in motor function and improvements in patient- and caregiver-reported outcomes in patients with nonambulatory types 2 and 3 SMA receiving survival motor neuron (SMN) therapy. Scholar Rock also presented a literature review that describes the ongoing burden and significant unmet needs in SMA despite advancements in treatment. In addition, the Company hosted a "Muscle Matters in SMA" lunch symposium highlighting the experiences of a person living with SMA, a neurologist, and a physical therapist.

· 12-month data from Phase 2 TOPAZ trial published in Neurology. These data demonstrated that apitegromab led to improved motor function in participants with types 2 and 3 SMA, supporting further study of apitegromab in the pivotal Phase 3 SAPPHIRE trial. The full manuscript titled, "Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3" appeared in the March 12 issue of Neurology.

· ONYX long-term extension study for patients from both the TOPAZ and SAPPHIRE studies is ongoing.

Cardiometabolic Program

SRK-439 is a novel, preclinical, investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin and maintains myostatin specificity (i.e., no GDF11 or Activin-A binding), and is initially being developed for the treatment of obesity.

· Announced FDA clearance of IND application to initiate a Phase 2 proof-of-concept trial of apitegromab to treat obesity in patients taking a GLP-1 receptor agonist (GLP-1 RA). The Phase 2 trial is a randomized, double-blind, placebo-controlled, multi-center study to evaluate the effect of apitegromab, a highly selective myostatin inhibitor, to safely preserve lean muscle mass as an adjunctive therapy in overweight and obese adults who are taking a GLP-1 RA. The clinical study is expected to commence in mid-2024 with data expected in mid-2025 and will be used to guide clinical development of SRK-439. The Company plans to file an IND for SRK-439 for the treatment of obesity in 2025.

· Presented preclinical SRK-439 data at the Keystone Symposia’s Obesity: Causes and Consequences meeting in February 2024. Preclinical data evaluating SRK-439 in combination with GLP-1 RAs supports the potential of SRK-439 to preserve lean muscle mass as part of healthy weight loss management. The Company intends to present additional preclinical SRK-439 data at upcoming conferences.

Corporate

· Appointed Katie Peng to the Board of Directors. In February 2024, Ms. Peng joined Scholar Rock’s Board of Directors. As an accomplished biotechnology executive, she brings over three decades of deep commercial experience in neurology and rare diseases, including with Evrydsi for SMA, as the Company prepares for the potential commercial launch of apitegromab. Ms. Peng is currently the Chief Commercial Officer at Denali Therapeutics.

· Promoted Mo Qatanani, Ph.D. to Chief Scientific Officer. In February 2024, Dr. Qatanani was promoted to Chief Scientific Officer of Scholar Rock. Dr. Qatanani joined Scholar Rock in 2021 and has been leading all research functions since 2022 and driving Scholar Rock’s early-stage pipeline, including SRK-439. He brings over 15 years of discovery and translational research experience in developing and advancing multiple therapeutic modalities in the neuromuscular and cardiometabolic disease areas.

· Scholar Rock to host an Investor & Analyst Day event on May 22, 2024 in New York City. Event to highlight the Company’s myostatin inhibition programs in SMA and obesity, including commercial readiness activities for apitegromab and the SRK-439 development plan.

Full Year 2023 Financial Results

For the full year ended December 31, 2023, net loss was $165.8 million or $1.99 per share compared to a net loss of $134.5 million or $2.26 per share for the full year ended December 31, 2022.

· The Company did not record any revenue for the year ended December 31, 2023. The Company recorded $33.2 million in revenue for the year ended December 31, 2022. The revenue in 2022 was attributable to recognition of the remaining revenue related to a collaboration agreement between the Company and Gilead Sciences.

· Research and development expense was $121.9 million for the year ended December 31, 2023, compared to $124.4 million for the year ended December 31, 2022. R&D spend was in line with company plans.

· General and administrative expense was $49.4 million for the year ended December 31, 2023, compared to $43.1 million for the year ended December 31, 2022. The increase was associated with planned increases in employee compensation and benefits expense as the Company increased total headcount during 2023.

· As of December 31, 2023, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $280 million, which is expected to fund the Company’s anticipated operating and capital expenditure requirements into the second half of 2025.

"We executed across our portfolio in 2023 and leveraged our world-leading expertise in myostatin inhibition with the formal launch of our cardiometabolic program. Additionally, we managed spend carefully throughout the year, and the capital raise in the fourth quarter extended our cash runway," said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. "We are off to a strong start in 2024 and are well positioned for a pivotal year, looking to the SAPPHIRE data read-out as a critical inflection point for the company."

About the Phase 3 SAPPHIRE Trial

SAPPHIRE is an ongoing randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 SMA who are receiving SMN-targeted therapy (either nusinersen or risdiplam). SAPPHIRE targeted enrolling approximately 156 patients aged 2-12 years old in the main efficacy population. These patients were randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks. An exploratory population that targeted enrolling up to 48 patients aged 13-21 years old will also separately be evaluated. These patients were randomized 2:1 to receive either apitegromab 20 mg/kg or placebo. For more information about SAPPHIRE, visit www.clinicaltrials.gov. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established.

About SRK-439

SRK-439 is a novel, preclinical, investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin and maintains myostatin specificity (i.e., no GDF11 or Activin-A binding), and is initially being developed for the treatment of obesity. Based on preclinical data, SRK-439 has the potential to support healthier weight management by preserving lean mass. The efficacy and safety of SRK-439 have not been established and SRK-439 has not been approved for any use by the FDA or any other regulatory agency.