SOUTH SAN FRANCISCO (dpa-AFX) - This week's biotech landscape features key FDA approvals, clinical holds and go-aheads, trial discontinuations, and clinical trial data readouts across key therapeutic areas such as Endometrial cancer, Colorectal cancer, Weight Management, Acne, and Duchenne muscular dystrophy.
Let us unpack the key developments and milestones in the biotech space this week.
FDA Approvals & Rejections
Intellia Gets FDA Green Light to Resume MAGNITUDE-2 Phase 3 Trial
Intellia Therapeutics, Inc. (NTLA) said the U.S. FDA has lifted the clinical hold on its MAGNITUDE-2 Phase 3 trial of nexiguran ziclumeran (nex-z) for hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), allowing enrollment and dosing to restart.
The hold was issued in October 2025 after a patient in a study, dubbed MAGNITUDE ATTR-CM, experienced Grade 4 liver enzyme elevations. Intellia worked with the agency on enhanced safety monitoring, including more frequent liver lab assessments, which supported the FDA's decision to clear MAGNITUDE-2.
MAGNITUDE-2 will now proceed with an updated protocol, increasing target enrollment to 60 patients from the original 50. Nex-z, a CRISPR/Cas9-based therapy designed to permanently inactivate the TTR gene, has shown deep and durable TTR reductions in earlier studies and holds Orphan Drug and RMAT designations.
Intellia said it will resume MAGNITUDE-2 enrollment promptly while continuing discussions with the FDA to resolve the ongoing hold on the ATTR-CM trial.
NTLA closed Thursday's (January 29, 2025) trading at $13.09, up 0.58%.
Outset Medical Secures FDA Clearance for Next-Generation Tablo Hemodialysis System
Outset Medical, Inc. (OM) said the U.S. FDA has granted 510(k) clearance for its next-generation Tablo Hemodialysis System, making it the first dialysis device to meet the agency's strengthened cybersecurity standards.
The upgraded platform features modernised software, hardware enhancements, and improved durability, while maintaining Tablo's integrated water purification and on-demand dialysate production for use across hospital and home settings.
Outset plans to begin shipping the new system in the second quarter of 2026, with upgrade options available for existing customers.
OM closed Thursday's trading at $5.07, up 0.90%.
OKYO Reaches Alignment With FDA for Urcosimod Phase 2b/3 trial
OKYO Pharma Limited (OKYO) reported that the U.S. FDA provided positive Type C meeting feedback and endorsed the Phase 2b/3 trial design for Urcosimod, its lead candidate for neuropathic corneal pain.
The agency confirmed the proposed VAS pain-reduction endpoint, agreed that a two-point or greater improvement is clinically meaningful, supported the study's powering and OPAS use, and raised no material CMC issues.
OKYO plans to begin the 120-patient multi-dose Phase 2b/3 trial in the first half of 2026.
OKYO closed Thursday's trading at $2.04, down 6.85%.
REGENXBIO Hit by FDA Clinical Holds on MPS I and MPS II Gene Therapy Programs
REGENXBIO Inc. (RGNX) announced that the U.S. FDA has placed clinical holds on its RGX-111 and RGX-121 gene therapy programs for MPS I and MPS II after a preliminary analysis identified a single case of an intraventricular CNS tumor in a child treated with RGX-111 four years earlier.
Genetic testing of the resected tumor showed an AAV vector integration event involving PLAG1, though causality has not been established, and the participant remains asymptomatic. No similar findings have been reported among nine other RGX-111 patients or the 32 patients treated with RGX-121.
The company expressed surprise at the hold on RGX-121, citing its long-term safety profile, and is awaiting the FDA's full clinical hold letter for further details.
RGNX closed Thursday's trading at $11.60, up 5.36%.
Almirall Wins NMPA Approval for Seysara in China
Almirall, S.A. (LBTSF) announced that China's National Medical Products Administration has approved Seysara (sarecycline hydrochloride) for the treatment of inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients 9 years of age and older. The company also finalized a licensing agreement with Sinomune, which will commercialize and distribute the therapy in China.
Seysara is an oral, tetracycline-derived antibiotic developed specifically for dermatology, and the approval marks a key expansion of Almirall's medical dermatology portfolio in the region.
LBTSF closed Thursday's trading at $15.47.
Deals
YD Bio Signed LOI To Acquire Safe Save Medical
YD Bio Limited (YDES) announced that it has entered into a binding letter of intent to acquire all shares, assets, and business of Safe Save Medical Cell Sciences & Technology Co., Ltd., a Taiwan-based dendritic cell immunotherapy platform, which includes corporate restructuring for a total consideration of $26.87 million in a cash and share deal.
Under the terms of the letter of intent, YD Bio Ltd intends to acquire 100% of the equity interests of Safe Save Cell Holdings Limited (KY), an offshore holding company to be established through a Safe Save Medical Cell Sciences & Technology's (SSMC) corporate restructuring that will result in the KY Company holding 100% of SSMC's shares, for a total consideration of NT$839.83 million or approximately $26.87 million.
The consideration for the acquisition is expected to be a combination of newly issued YD Bio Ltd shares and cash.
To be precise, shareholders holding more than 10% of SSMC's shares, including its founder Mr. Yang Wen-Guang and his affiliates will receive new YD Bio Ltd shares through a share swap. And other shareholders will have the option to receive cash or elect to receive new YD Bio Ltd shares.
The parties aim to complete the process by March 20, 2026, with the closing of the transaction anticipated within 30 days thereafter, pending satisfaction of all conditions.
Safe Save Medical Cell Sciences Technology (SSMC) specialises in innovative cell-based medical technologies, with a focus on immunocell therapy platforms.
The acquisition is expected to strengthen YD Bio's capabilities in developing next-generation treatments and broaden its portfolio in the field of advanced cellular therapeutics.
YDES closed Thursday's trading at $11.03, down 9.89%.
Clinical Trials - Breakthroughs
Aprea's APR-1051 Showed Early Clinical Activity In Endometrial Cancer
Aprea Therapeutics, Inc. (APRE) reported early clinical proof-of-concept in its ongoing ACESOT-1051 dose-escalation trial evaluating APR-1051, a WEE1 inhibitor in patients with advanced solid tumours.
APR-1051 is an orally bioavailable, highly potent, and selective small molecule inhibitor of WEE1, a protein kinase that helps regulate the cell cycle by preventing abnormal progression.
The ACESOT-1051 trial is a first-in-human, open-label, multicenter study evaluating APR-1051 in patients with advanced solid tumours. The study is assessing safety, tolerability, and anti-tumour activity across escalating dose levels.
In a patient with PPP2R1A-mutated uterine serous carcinoma, a form of endometrial cancer, treatment using APR-1051,150 mg dose level, achieved a 50% reduction in target lesion size at the 8-week imaging assessment per RECIST v1.1 criteria, along with a marked decline in CA-125 levels.
APRE closed Thursday's trading at $0.59.
Fractyl Health's Revita Demonstrated Durable Weight Maintenance in REMAIN-1 Midpoint Cohort
Fractyl Health, Inc. (GUTS) announced positive six-month randomized results from its REMAIN-1 Midpoint Cohort, a blinded, sham-controlled study evaluating Revita for weight maintenance following GLP-1 drug discontinuation.
Revita is Fractyl Health's lead product candidate, designed to remodel the duodenal lining via a minimally invasive endoscopic procedure to restore healthy nutrient sensing and signalling disrupted by chronic metabolic diseases.
The REMAIN-1 Midpoint Cohort included 40 patients, with five excluded per protocol due to diet and lifestyle noncompliance. Revita-treated patients showed a 4.5% weight regain compared to 7.5% in the sham arm at six months.
Among patients with above median GLP-1-associated weight loss, Revita treatment resulted in approximately 70% less post-GLP-1 weight regain versus sham.
Patients treated with Revita experienced sustained weight maintenance, improved cardiometabolic profiles, and reduced food cravings compared with sham at six months, with continued excellent safety and tolerability.
GUTS closed Thursday's trading at $0.58.
Ascletis Reported Positive Phase 3 Safety Results of Denifanstat in Moderate-to-Severe Acne
Ascletis Pharma Inc. (1672.HK) announced positive topline results from its Phase 3 study for Denifanstat in moderate-to-severe acne vulgaris, a common dermatological condition.
Denifanstat is a once-daily oral fatty acid synthase (FASN) inhibitor, and is licensed from Sagimet Biosciences Inc (SGMT) for exclusive rights in Greater China.
The phase 3 study was an open-label, multicenter trial in China designed to evaluate the long-term safety of Denifanstat (ASC40), in 240 patients with moderate-to-severe acne vulgaris. All patients, previously treated with Denifanstat or placebo for 12 weeks, received Denifanstat once daily for 40 weeks.
1672.HK closed Friday's trading at HK$13.8, up 1.62%.
GRI Bio Reports New Phase 2a Gene Expression Data Supporting Anti-Fibrotic Activity Of GRI-0621 in idiopathic pulmonary fibrosis
GRI Bio Inc. (GRI) reported new gene-expression findings from Phase 2a study of GRI-0621 in idiopathic pulmonary fibrosis. GRI-0621 is an oral immune-modulating candidate designed to target key biological drivers of fibrosis.
The new RNA-sequencing data showed significant improvements across genes associated with lung injury, myofibroblast activation, extracellular matrix deposition, fibrosis progression, and epithelial repair compared with placebo.
In addition, GRI Bio noted that in the loss of alveolar epithelial cells and basement-membrane destruction associated with IPF, the new gene-expression data supported basement-membrane repair and AT2-to-AT1 epithelial transition, which are the key hallmarks of lung regeneration.
GRI closed Thursday's trading at $3.32, down 19.22%.
CalciMedica Discontinues Phase 2 KOURAGE Trial Of Auxora Study
CalciMedica, Inc. (CALC), announced the discontinuation of its Phase 2 KOURAGE clinical trial that evaluates Auxora in patients with Stage 2 or Stage 3 acute kidney injury with associated acute hypoxemic respiratory failure, following a recommendation from the Independent Data Monitoring Committee.
The recommendation was based on a safety concern that prompted a re-evaluation of the study design, particularly the patient enrollment criteria.
Now, CalciMedica has discontinued the trial and plans to perform a comprehensive review assessing the impact of baseline characteristics, disease severity, concomitant therapies on patient outcomes.
CALC closed Thursday's trading at $1.02, down 18.40%.
Cardiff Reports Positive Colorectal Cancer Trial Results
Cardiff Oncology, Inc. (CRDF) announced encouraging clinical results from its CRDF-004 Phase 2 dose-finding trial on Onvansertib in combination with a standard-of-care regimen as a first-line RAS-mutated metastatic colorectal cancer.
The standard of care (SoC) regimens included FOLFIRI/bevacizumab (bev) or FOLFOX/bev.
The company observed dose-dependent improvements in multiple efficacy measures, including objective response rate and progression-free survival and found the 30 mg Onvansertib plus FOLFIRI/bevacizumab arm outperforming both standard-of-care arms.
The regimen was well tolerated, with no major or unexpected toxicities and no additive adverse events, allowing Cardiff to select a 30 mg dose to advance into a registrational program, with final data and trial design discussions expected in the first half of 2026.
CRDF closed Thursday's trading at $1.62, down 0.31%.
Genentech's CT388 Demonstrates Significant 22.5% Weight Loss in Phase 2 Study
Genentech, a biotechnology firm and member of the Roche Group (RHHBY, ROG.SW), announced positive topline results from its Phase 2 clinical trial of its lead candidate CT-388 in obesity.
CT-388 is an investigational once-weekly subcutaneous injectable, dual GLP-1/GIP receptor agonist being developed for the treatment of obesity, type 2 diabetes, and other obesity-related comorbidities.
In the phase 2 study, dubbed CT388-103, once-weekly subcutaneous injections of CT-388 at 24 mg achieved a statistically significant placebo-adjusted weight loss of 22.5% at 48 weeks, without reaching a weight loss plateau.
According to the company, at Week 48, among participants receiving the 24 mg dose of CT-388, 95.7% lost at least 5% of their body weight, 87% lost at least 10%, 47.8% lost at least 20%, and 26.1% lost at least 30%.
Among participants receiving the 24 mg dose, 54% achieved resolution of obesity (BMI <30 kg/m2), compared with just 13% in the placebo group.
The Phase 3 clinical trial program of CT-388 in obesity, dubbed Enith1 and Enith2, is expected to start this quarter.
RHHBY closed Thursday's trading at $57.02, down 4.84%.
Sarepta's ELEVIDYS showed positive topline three-year results in Ambulatory Duchenne Patients EMBARK Study
Sarepta Therapeutics Inc. (SRPT) announced positive topline three-year results from its EMBARK study, showing that ELEVIDYS significantly slowed disease progression across key functional measures in ambulatory patients with Duchenne muscular dystrophy.
ELEVIDYS is a single-dose AAV-based gene therapy designed to deliver a micro-dystrophin transgene to skeletal muscle. It is currently the only approved gene therapy for Duchenne, available to ambulatory individuals aged 4 and older under an updated FDA label issued at the end of 2025.
The three-year data come from Part 1-treated patients in EMBARK, Sarepta's global, randomized, placebo-controlled Phase 3 trial evaluating ELEVIDYS in ambulatory boys aged 4-7 at the time of dosing.
EMBARK is a two-part, crossover Phase 3 study in which participants received either ELEVIDYS or a placebo in Part 1, then crossed over in Part 2.
The study's primary endpoint was change from baseline in the North Star Ambulatory Assessment (NSAA) at Week 52, with long-term follow-up extending through multiple years.
As per the three-year data, the ELEVIDYS-treated patient group's NSAA mean score remained above baseline at Year 3, while the external control group declined with time to rise (TTR), depicting 73% slowing of disease progression. Also 10-meter walk/run (10MWR) based score showed 70% slowing of disease progression.
SRPT closed Thursday's trading at $21.24, down 0.35%.
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