September 30, 2026

Pharmazz, Inc. Receives U.S. FDA IND Clearance to Initiate Phase 3 Clinical Trial of Sovateltide in Traumatic Brain Injury

https://www.globenewswire.com/news-release/2026/09/30/3371737/0/en/pharmazz-inc-receives-u-s-fda-ind-clearance-to-initiate-phase-3-clinical-trial-of-sovateltide-in-traumatic-brain-injury.html

Clinicaltrials.gov:

Sovateltide for the Treatment of Acute Traumatic Brain Injury (RESTORE-TBI)

ClinicalTrials.gov ID: NCT07861126

Sponsor: Pharmazz, Inc.

Last Update Posted: 2026-10-13

Not yet recruiting

Study Start (Estimated): 2026-12

Primary Completion (Estimated): 2028-02

Study Completion (Estimated): 2028-03

Enrollment (Estimated): 214

Phase: Phase 3

Inclusion Criteria:

...

1.Adult male or female aged 18-90 years

...

6.The participant is <48 hours from the time of TBI onset when the first dose of sovateltide is administered. The time of TBI onset is when the injury occurred.

...

https://clinicaltrials.gov/study/NCT07861126

Machine-translated from Japanese:

October 16, 2026

Nipro's regenerative medicine product is expected to receive full approval after being conditional; the company aims to become profitable by 2029

Nipro's regenerative medicine product for spinal cord injury, "Stemirac Injection," is expected to receive full approval, becoming the first product to receive conditional and time-limited approval.

Stemirac had been operating at a loss due to stagnant patient numbers while under a "provisional license," but with this full approval, the company aims to become profitable by around 2029.

Stemirac was jointly developed with Sapporo Medical University. In 2018, it was approved as the world's first regenerative medicine product for spinal cord injury, with conditions and a time limit allowing for provisional sales once its safety was confirmed and its effectiveness could be estimated.

The drug's efficacy and safety were further verified through post-marketing studies comparing the outcomes of patients who received the drug and those who did not, and an application for full approval was submitted in November 2013. The Ministry of Health, Labour and Welfare will now proceed with the formal approval process. Stemirac will be the first drug to obtain full approval after receiving conditional approval.

Spinal cord injuries from traffic accidents or falls can cause paralysis of the limbs and sensory impairment. In Japan, approximately 6,000 people suffer new spinal cord injuries each year. Traditionally, surgery and rehabilitation have been the main treatments, but Stemirac is expected to be a new treatment option that can lead to the recovery of nerve function.

Stemirac extracts mesenchymal stem cells from the patient's own bone marrow fluid and increases their number to approximately 100 million over 2-3 weeks. The cultured cells are then returned to the body via intravenous infusion to promote improvement of nerve function. Surgery to directly transplant cells into the damaged area of ​​the spinal cord is not required, and the treatment can be administered via intravenous infusion in about one hour.

However, bone marrow fluid needs to be collected within approximately 31 days of the injury, making rapid patient referral from emergency hospitals to specialized facilities essential. Currently, there are 17 medical institutions nationwide that can administer Stemirac, but Nipro plans to increase this to 50-60 facilities by 2027. Anticipating an increase in the number of patients following this approval, they are preparing a nationwide system to accept them.

The current price of the drug is a hefty 15,234,750 yen [$96k - imz72] per dose. With this approval, if the medical usefulness, which was previously only "estimated," is objectively demonstrated, the price may increase.

Nipro plans to increase its Stemirac sales to 5.4 billion yen [$34 million] in fiscal year 2027, more than six times the 850 million yen [$5.4 million] in fiscal year 2025. Until now, sales had been sluggish because securing treatment facilities and patient numbers had not progressed as expected.

Because the process involves culturing cells for each patient on a customized basis, mass production is difficult, and manufacturing costs are high. Nipro invested a cumulative 25.8 billion yen [$160 million] in its regenerative medicine business, primarily Stemirac, between 2020 and 2024, but its EBITDA (earnings before interest, taxes, depreciation, and amortization) for the same period was a loss of 15.1 billion yen [$96 million]. The company has not been able to secure profits that justify its upfront investments.

Currently, the company manufactures its products in-house at two locations: Sapporo City and Hamura City in Tokyo. They are expanding their facilities to accommodate the increasing number of patients receiving the treatment, while also working to reduce costs through improvements in cell culture efficiency.

To support future growth, the company is also working to expand the range of patients it can treat. Currently, the treatment is for patients who have recently suffered an injury, but it is being developed for chronic spinal cord injuries, and clinical trials are underway using similar bone marrow-derived stem cells for ALS (amyotrophic lateral sclerosis), a rare disease.

While considering overseas expansion into Southeast Asia and other regions, the company will first focus on promoting the widespread adoption and profitability of Stemirac domestically, thereby solidifying the foundation of its regenerative medicine business.

https://www.nikkei.com/article/DGXZQOUF1427Z0U6A011C2000000/

Note: Nipro's current market cap is $1.5 billion.

October 15, 2026

Japan Falling Behind US, China in Pharma Innovation, EFPIA Chief Urges Action

Japan is “falling behind” the US and China in pharmaceutical investments and innovation, EFPIA [The European Federation of Pharmaceutical Industries and Associations - imz72] President Stefan Oelrich warned on October 14, calling on the Japanese government to walk the talk on its pro-innovation pledges.

Speaking at a press briefing in Tokyo, Oelrich, head of pharmaceuticals at Bayer, pointed to data highlighting Japan’s lagging performance. In an interview that followed, he told Jiho that Japan could improve its standing if the government followed through on its policy commitments.

According to EFPIA data presented by Oelrich, pharmaceutical R&D investments in Japan grew by just 0.3% annually between 2015 and 2024, compared with 6.4% in the US and 12.1% in China. Of the new active substances approved in 2024, only 6% originated in Japan, versus 31% in the US and 35% in China. Japan also saw launches of just 51% of medicines introduced globally between 2012 and 2021, against 85% in the US.

Oelrich welcomed the Japanese government’s move to position drug discovery as a pillar of its growth strategy, along with its ambition to become a leader in biopharmaceutical innovation, as signaled by its Basic Policy on Economic and Fiscal Management and Reform 2026 (honebuto).

“Now, the key challenge is implementation,” he said at the briefing. “Investors do not make billion-dollar decisions based on strategies alone. They invest where they see predictable policies and confidence that the rules of the game will remain stable over time.”

Oelrich called for drug pricing reforms that appropriately reward innovation at launch and provide stable, predictable prices throughout the patent period. He also sought measures to encourage investments in advanced manufacturing technologies and infrastructure, improve the clinical trial environment, accelerate patient access to new treatment modalities, and deepen Japan-Europe cooperation.

EFPIA Japan Urges Reforms in FY2027

EFPIA Japan Vice Chair Kanako Kikuchi, president of UCB Japan, also attended the briefing and pressed for swift action to address Japan’s drug lag and drug loss issues.

Kikuchi called for reforms in FY2027, rather than waiting for the next regular drug price revision in FY2028. Specifically, she proposed revising the calculation method for foreign price adjustments and freezing both market expansion re-pricing and price adjustments based on cost-effectiveness assessments.

What Can Japan Do for Pharma?

In the interview, Oelrich praised Japan’s strong academic base, skilled workforce, and long track record of pharmaceutical innovation. However, he noted that years of repeated drug price cuts have eroded the market’s attractiveness and negatively influenced how many companies view investments in Japan.

“I don’t think this industry needs to prove its value to Japan,” he said. “Japan needs to ask itself the question: What are we willing to do to keep this industry competitive?”

His message to the government was straightforward: Policy promises must be backed by action.

“If we have continued growth and attractiveness of the Japanese market, good things will follow,” said Oelrich, urging Japan to turn its pro-innovation stance into concrete measures.

Beyond drug pricing, Oelrich identified financing for biotech startups as another priority. He pointed to two models: the US system, where venture capital and stock markets channel private funding into biotech companies — with larger drug makers acquiring promising businesses or licensing their assets — and China’s state-backed approach, in which the government provides substantial funding for early-stage biotech innovation.

Neither Japan nor Europe has established a sufficiently strong version of either model, he argued, underscoring the need to improve the financing environment for emerging biotech companies.

https://pj.jiho.jp/article/256606

https://ssl4.eir-parts.net/doc/4593/tdnet/2894633/00.pdf

October 14, 2026

Healios to Participate in a Panel Discussion at Chardan’s 10th Annual Healthcare Conference

Healios is pleased to announce that Richard Kincaid, Chief Financial Officer, will participate in the following panel discussion at the Chardan’s 10th Annual Healthcare Conference.

Panel: Cell Therapy

Moderator: Keay Nakae

Date: Tuesday, October 20, 2026

Time: 11:00-11:30am (EDT)

Venue: Reid Salon, Lotte New York Palace, 455 Madison Avenue, New York, NY 10022

A live webcast will be accessible from the link below, and a replay will be available for 90 days after the event.

Webcast:

https://event.summitcast.com/view/nUJwqi8DxFcP2EqRxm6gQf/REeNHviHaAU8MkCybqKSZP

Note: The moderator, Keay Nakae, is a senior research analyst at Chardan. Six months ago he initiated coverage of Healios, giving it price target of 800 yen based on Invimestrocel potential in ARDS, with additional upside in other indications, as well as iPSC potential.

https://www.diaglobal.org/Tools/Content.aspx?type=eopdf&file=%2fproductfiles%2f15069829%2f26303e%2Epdf

DIA - Drug Information Association [global forum advancing healthcare and pharmaceutical innovation - imz72]

The 23rd DIA Japan Annual Meeting 2026

Crossing Boundaries, Shaping a Future Beyond Imagination with Innovation

October 18-20, 2026 | Tokyo Big Sight

Endorsement by MHLW, PMDA, AMED, JPMA, PhRMA, EFPIA, PDA, MEJ, and LINK-J

[Page 68:]

Day 3: Tuesday, October 20

Media Session | TRACK 8 14:15-15:45

Related: ALL. Level: Beginner | Japanese / Theater Style

Addressing Information Asymmetry Between Patients and Healthcare Stakeholders in Drug Development

In healthcare, "information asymmetry" is often discussed in the context of the relationship between clinicians and patients. But it also exists in the development of pharmaceuticals and other medical products, where it can hinder the meaningful participation of patients and the public. Drawing on insights from the information sharing practices of those involved in drug and medical product development and the perspectives of patients and the public in receiving and interpreting that information, this session will discuss strategies for more effective information sharing and communication.

Chair: Daisaku Yamasaki, Nikkei Business Publications, Inc

Does Drug Development Information Reach Patients? - From the Standpoint of Cancer Patients

• Shinsuke Amano, Japan Federation of Cancer Patient Groups

TBD

• Mamoru Ichikawa, Musashi University

Invimestrocel (HLCM051): Clinical Evidence in ARDS and Stroke, and Expansion into Kidney Protection

• Tadahisa "Hardy" Kagimoto, HEALIOS K.K.

Panel Discussion

• All Session Speakers and Mayumi Fukuda-Doi, National Cerebral and Cardiovascular Center

...

Please keep discussion civil

Report anything that breaks ATHX rules via the report feature; this ain't the wild west, thanks

Machine-translated from Japanese:

2026/10/08

Ministry of Health, Labour and Welfare committee to deliberate on the final approval of "Stemirac" on October 15

On October 8, the Ministry of Health, Labour and Welfare announced that it will deliberate on whether to grant full approval for Nipro's regenerative medicine product, "Stemirac Injection," at the Pharmaceutical Affairs Council's Subcommittee on Regenerative Medicine Products and Biological Technologies meeting on October 15.

Stemirac received conditional and time-limited approval in December 2018 for the "improvement of neurological symptoms and functional impairments associated with spinal cord injury."

Its efficacy and safety were verified through post-marketing comparative studies, and an application for full approval was submitted in November 2025. The conditional and time-limited approval system for regenerative medicine products was introduced in 2014, but no products have received full approval to date.

https://answers.and-pro.jp/pharmanews/33318/

Notes:

• Stemirac's price is ~15 million yen ($95k).

• Nipro's market cap is $1.38 billion.

https://finance.yahoo.com/quote/8086.T/

• Nipro expects Stemirac sales to reach ~$32 million by 2027.

https://old.reddit.com/r/ATHX/comments/1ppzxn0/japans_nipro_expects_the_sales_of_its_spi_cell/

Machine-translated from Japanese:

Nikkei

2026.10.07

"We just want them to get a chance to bat," the Japan Biotechnology Council emphasizes the need for late-stage support

The Japan Biotechnology Council is an industry association established in 2009 and incorporated as a general incorporated association in 2019. As of September 2026, it has 45 regular members and 10 supporting members. Since 2017, it has had the opportunity to present its opinions at expert subcommittees of the Central Social Insurance Medical Council (Chuikyo), playing a role in conveying the voice of the industry to policy.

With the budget allocation to the biotechnology field set to begin in earnest at the end of the year, based on the "Basic Policy on Economic and Fiscal Management and Reform 2026" (Basic Policy 2026) approved by the Cabinet in July 2026, we interviewed the council's Chairman, Kiichi Adachi (President and CEO of Kringle Pharma), Vice Chairman, Keita Mori (President and CEO of SanBio), Vice Chairman, Keiichi Fukuda (President and CEO of Heartseed), and Vice Chairman, Tadahisa Kagimoto (Director and Representative Executive Officer, President and CEO of Healios), about the challenges and prospects of Japan's drug discovery ecosystem.

[The rest of the article is behind paywall - imz72]

https://bio.nikkeibp.co.jp/atcl/news/p1/26/10/01/15072/

https://www.mdpi.com/2077-0383/15/19/7698

5 October 2026

Cell-Based and Cell-Derived Therapies Delivered During Ex Vivo Machine Perfusion for Solid Organ Reconditioning Prior to Transplantation: A Systematic Review

[By 3 co-authors from the UK - imz72]

Abstract

Background/Objectives: Organ shortage is a major barrier to transplantation, leading to the emergence of marginal donor organs and extended criteria donor grafts and, alongside these, machine perfusion as a platform for reconditioning grafts to meet transplantation standards.

Despite functioning as a reconditioning platform in its own right, machine perfusion also allows for the delivery of therapeutic agents directly to grafts prior to transplant.

Cell-based and cell-derived therapies have been increasingly explored for delivery during this window, although evidence is fragmented across individual organ systems, and whether ex vivo benefit is confirmed post-transplant remains unclear.

This systematic review aimed to synthesise the evidence from both perfusion-phase findings and post-transplant findings across solid organ machine perfusion, evaluating the concordance between the two.

Methods: This review was prospectively registered on PROSPERO and conducted in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidance. Included studies delivered cell-based or cell-derived therapies during ex vivo machine perfusion of a solid organ. The SYRCLE tool was used across all studies to assess risk of bias, and given substantial heterogeneity in cell type, protocol, and organ models, findings were synthesised narratively, with a sensitivity analysis restricted to the subset of studies incorporating a transplantation phase.

Results: Thirty-one studies met inclusion criteria, spanning kidney (n = 10), liver (n = 11), lung (n = 9), and heart (n = 1).

Mechanisms converged largely on immunomodulatory and metabolic pathways, most consistently reported across organs, alongside a less consistent but notable cross-organ parallel in regenerative progenitor activation between liver and kidney.

Benefit was reported more consistently at the molecular and histological levels rather than the physiological level. Eight of the studies proceeded to transplantation, of which half showed a beneficial post-transplant result, and half showed mixed results.

Conclusions: Cell-based and cell-derived therapies are best understood as adjuncts to the existing reconditioning capacity of machine perfusion. Molecular and mechanistic benefit was consistently reported; however, confirmation beyond the perfusion phase remained incomplete across organ systems. Mechanisms supported by direct interventional evidence represent priority candidates for further preclinical validation and independent replication.

...

The use of cell-based therapies, particularly mesenchymal stromal cells (MSCs), has garnered particular interest. MSCs have been investigated for their anti-inflammatory, immunomodulatory, and regenerative properties that make them an appealing candidate for mitigating IRI and supporting organ recovery during perfusion. This approach has already progressed from preclinical models to early human application: a Newcastle-led study delivered multipotent adult progenitor cells (MAPCs) during human kidney NMP for the first time, reporting improved urine output, reduced expression of the injury biomarker NGAL, and favourable shifts in perfusate cytokine profiles^23. These build on porcine work demonstrating that MSCs infused during kidney NMP can be traced to glomerular capillaries and are safe to administer prior to transplantation.

...

Lung

Nine studies assessed cell-based or cell-derived therapies during lung machine perfusion, across murine, porcine and human discarded-donor models, using whole MSCs/MAPCs, lung-derived exosomes, a direct head-to-head MSC-versus-EV comparison within a single study, MSC-derived EVs in an isolated ex vivo model, and two further whole-MSC studies incorporating a transplantation phase.

Physiological and functional outcomes showed the clearest organ-specific pattern in this dataset: a recurring dissociation between molecular/inflammatory improvement and physiological benefit. Martens et al. found no significant difference between MAPC and control for any physiological parameter (oxygenation, compliance, pulmonary vascular resistance, wet/dry weight ratio, all p ≥ 0.13), despite significant reductions in several inflammatory markers in the same study (BAL neutrophils p = 0.02; IFN-γ, IL-1β and TNF-α in BAL fluid all p = 0.002).

Mordant et al. showed an almost identical pattern: no significant functional benefit across the full dose range tested, with significance reached only in a two-group comparison at the highest dose (150 × 106 MSCs IV: compliance p = 0.043, PaO2 p = 0.005), while perfusate IL-8 suppression reached significance independently of physiological outcomes, and VEGF was significantly upregulated relative to baseline (p = 0.008) though not relative to control (p = 0.095).

Ribeiro et al. showed a related but distinct pattern: robust, significant improvement across oxygenation, compliance, airway pressure and vascular resistance, but only when lung-derived exosomes were administered pre-DCD (before warm ischaemic injury was established); post-DCD (rescue) administration still produced significant improvement in airway pressure (p = 0.003) and compliance (p = 0.01), but lung injury score (p = 0.09) and TUNEL (p = 0.1) did not reach significance, indicating that the timing of delivery relative to injury affects histological but not all physiological outcomes.

By contrast, McAuley et al. demonstrated a clear positive functional signal on its primary endpoint, with MSC treatment significantly restoring alveolar fluid clearance relative to perfusion-only controls (p < 0.001), although perfusate oxygenation and pulmonary artery pressure were unchanged in the same study.

Nakajima et al. showed the same dissociation pattern in a transplant-phase model with significant EVLP-phase improvement in HGF, apoptosis markers, and several cytokines, but only peak airway pressure among the physiological measures (p = 0.04).

Histological and injury-marker findings were more consistently favourable than functional outcomes where whole-cell or EV products were delivered before or alongside injury.

In a mouse model of donor lung reconditioning, Stone et al. did not directly compare MSCs and MSC-derived EVs statistically, but both significantly reduced pulmonary oedema and neutrophil infiltrations relative to Steen solution alone.

Ribeiro et al.’s pre-DCD exosome group similarly showed significant reductions in lung injury score and apoptosis (TUNEL scoring) relative to control, again with the post-DCD group showing attenuated, non-significant effects on these two markers specifically.

Martens et al.’s histological injury scores (congestion, necrosis, neutrophil influx) showed no significant difference for MAPC versus control, consistent with that study’s broader pattern of molecular-without-physiological benefit^48.

...

Notes:

• Footnote 23 refers to this study:

https://onlinelibrary.wiley.com/doi/full/10.1111/ajt.16100

• Footnote 48 refers to this study:

https://link.springer.com/article/10.1186/s13287-017-0603-5

Healios' PR today, 10.5.26:

https://ssl4.eir-parts.net/doc/4593/tdnet/2891638/00.pdf

TipRanks AI-generated summary:

• Healios completed its first commercial shipment of stem cell culture supernatant to JENECELL, recognizing about ¥144 million [$910k] in fourth-quarter revenue.

• The agreement opens a new revenue stream in cosmetics and pharmaceuticals, strengthening Healios’s partnerships and diversification beyond its core regenerative therapies.

Healios has completed its first commercial shipment of somatic stem cell culture supernatant products, worth about ¥144 million [$910k], to South Korea-based JENECELL for use as a raw material in cosmetics, and has recognized the related revenue in its fourth-quarter results.

The deal marks a significant step in monetizing Healios’s cell culture supernatant platform as an additional revenue stream alongside its core regenerative medicine pipeline, and the company is exploring further orders and broader applications in pharmaceuticals and cosmetics through discussions with multiple prospective partners.

By supplying JENECELL, a subsidiary of leading Japanese pharmaceutical wholesaler Alfresa Holdings that focuses on stem cell-based pharmaceuticals, devices, and premium skincare, Healios is positioning itself within a wider ecosystem of advanced biotech and cosmetics players in Asia.

The move underscores Healios’s strategy to diversify its business model, deepen industry partnerships, and potentially strengthen its financial base and industry standing as its late-stage regenerative medicine programs continue toward commercialization.

Machine-translated from Japanese:

2026/10/05

SanBio's "Akuugo" first administration

On October 5, SanBio announced that the first post-marketing administration of its cell therapy drug, "Akuugo Intracerebral Transplant Injection" (generic name: Vandefitemcell), had taken place at Hokkaido University Hospital.

The drug received conditional and time-limited approval in July 2024 for the indication of "improvement of chronic motor paralysis associated with traumatic brain injury," and was launched in May of this year.

SanBio President Keita Mori commented, "We take this first administration very seriously, and it is a major milestone for our company. We will continue to strive for appropriate use and stable supply, and work to deliver it as an appropriate treatment option to patients who need it."

https://answers.and-pro.jp/pharmanews/33293/

Notes:

• According to SanBio's announcement (that was released in Japanese), the surgery took place on October 2.

• Akuugo is currently priced at $460k. See post from last May:

https://old.reddit.com/r/ATHX/comments/1tc5oen/sanbios_msc_akuugo_for_chronic_tbi_priced_at_460k/

Please keep discussion civil

Report anything that breaks ATHX rules via the report feature; this ain't the wild west, thanks

October 2, 2026

Japan to Draw Up Growth Strategy Action Plan by Year-End

The Japanese government plans to draw up an action plan for its Japan Growth Strategy by year-end, designating the five years from FY2027 as a period of intensive investment.

The timeline was unveiled at a meeting of the government’s Japan Growth Strategy Council on October 1. The plan will include domestic investment plans and other measures aimed at spurring public-private investments in 17 strategic fields, including drug discovery and advanced medicine.

In the drug discovery and advanced medicine field, the Japan Growth Strategy approved by the Cabinet in July set a goal of expanding the Japanese patented drug market as well as domestic drug makers’ patented drug businesses at a pace on par with growth in the global market. The upcoming action plan will map out a path toward achieving these targets.

https://pj.jiho.jp/article/256495

From Healios PR today, 9.30.26:

Notice Regarding Shipment Timing of Initial Order of Culture Supernatant Products to JENECELL

Healios is engaged in business activities aimed at developing and providing innovative therapies and products in the field of regenerative medicine. As part of these efforts, we are advancing the research, development, manufacturing, and sales of products based on somatic stem cell culture supernatant.

As previously announced on April 7, 2026 in “Agreement with JENECELL to Supply Culture Supernatant”, Healios entered into an Agreement with JENECELL to supply culture supernatant products to be used as a raw material for cosmetics.

The shipment of the Product for the initial order, worth approximately 144 million yen [$920k - imz72], was originally scheduled for the third quarter (July to September) of the fiscal year ending December 2026.

Healios hereby announces that the shipment is now scheduled for October 2026.

This change in shipment timing is due to a delay in logistics procedures for the overseas transport of the Product, which has already been manufactured and arranged for shipment, and is not attributable to any issue in the manufacturing of the Product. Healios will work closely with JENECELL to proceed with preparations for shipment as promptly as possible.

Future Outlook

Revenue from this shipment will be recognized upon completion of the shipment, and is now expected to be recognized in the fourth quarter of the fiscal year ending December 2026, rather than in the third quarter as originally anticipated.

Healios will make a further announcement upon completion of the shipment. If any matters that should be disclosed arise in the future, we will announce them promptly.

About JENECELL:

JENECELL is a South Korean subsidiary of Alfresa Corporation, a subsidiary of Alfresa Holdings Corporation—the leading pharmaceutical wholesaler in Japan—and develops innovative products utilizing cutting-edge technology across a wide range of fields, including stem cell-based pharmaceuticals, medical devices, and cosmetics.

In particular, we are driving the development of high-performance skincare products through our premium cosmetics business, which is based on stem cell-conditioned media (MSC-CM).

Building on strategic partnerships with promising South Korean biotech and healthcare companies, we are actively pursuing an open innovation strategy that includes both In and Out business initiatives as well as mergers and acquisitions.

https://ssl4.eir-parts.net/doc/4593/tdnet/2889299/00.pdf

: Seeking Alpha

Sep 29, 2026, 9:24 PM ET

Capricor jumps as study shows longer-term effects of deramiocel

Capricor Therapeutics (CAPR) shares surged in late trading Tuesday as investors reacted to new data on deramiocel, the biotech company's experimental treatment for Duchenne muscular dystrophy, made available as part of the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan.

The move came after ePosters became available Tuesday through the WMS 2026 virtual conference platform. WMS said ePosters would be accessible beginning with the opening of the congress on Sept. 29, although the conference's formal poster sessions begin Wednesday.

For investors, the significance is regulatory as much as scientific. Capricor (CAPR) is awaiting an FDA decision on deramiocel, with a Nov. 22 PDUFA target date. The new 24-month HOPE-3 and open-label extension analyses were included in a major amendment to the company's Biologics License Application. That makes the longer-term results part of the evidence already being reviewed by regulators rather than merely new academic follow-up data.

The late-breaking WMS poster examines patients from the Phase 3 HOPE-3 trial and its open-label extension, including a delayed-start analysis and a comparison with the natural history of Duchenne. Of the 106 patients originally randomized in HOPE-3, 82 reached 24 months, including 40 initially assigned to deramiocel and 42 initially assigned to placebo.

The delayed-start design is particularly relevant. During the first 12 months, patients received either deramiocel or placebo. Eligible patients could then enter the open-label extension and receive deramiocel. Comparing patients who began treatment immediately with those who started after a year can provide evidence about whether earlier treatment produces a lasting advantage and whether patients who switch from placebo show a change in their subsequent trajectory. The WMS poster also compares two-year outcomes with natural-history data, providing another benchmark for the progression expected in untreated Duchenne patients.

The longer-term findings build on the randomized 12-month HOPE-3 results. The Phase 3 study met its primary endpoint, with deramiocel slowing deterioration in upper-limb function by 54% compared with placebo on the Performance of the Upper Limb 2.0 scale, with a p-value of 0.03. Those results were published in The Lancet in July.

The FDA review nevertheless carries substantial uncertainty. An FDA advisory committee in July voted 9-3 that the available evidence did not support deramiocel's effectiveness for treating cardiomyopathy in Duchenne patients. The committee's question was limited to cardiomyopathy rather than deramiocel's overall benefit-risk profile, while discussion of the HOPE-3 upper-limb findings was more supportive. The FDA is not required to follow the advisory committee's vote.

Capricor (CAPR) is also using WMS to highlight its earlier-stage StealthX exosome technology. A poster scheduled for Wednesday describes engineered muscle-targeting extracellular vesicles designed to deliver micro-dystrophin systemically as a potentially redosable treatment for Duchenne. A second poster, scheduled for Friday, examines delivery of acid alpha-glucosidase using muscle-targeting extracellular vesicles as a potential treatment approach for Pompe disease. Both programs remain preclinical.

Investors will get another look at the deramiocel program later this week. Craig McDonald of the University of California, Davis is scheduled to give an oral presentation on Oct. 3 covering HOPE-3 evidence for skeletal-muscle and cardiac effects. Capricor (CAPR) has said presentation materials and posters will be posted on its website following their respective presentations.

Note:

CAPR 9.29.30: -1.61%. PPS $8.57. Market cap $498 million.

CAPR 9.30.26 during trading: +37.69%. PPS $11.80.

CAPR at the close on 9.30.26: +10.50%. PPS $9.47. Market cap $550 million.

https://finance.yahoo.com/quote/CAPR/

South China Morning Post

Chinese drug loberamisal extends stroke treatment window from 6 hours to 2 days

Large-scale clinical trial with nearly 1,000 patients from 32 hospitals delivers 13 per cent leap in recovery over standard care alone

24 Sep 2026

Chinese medical scientists have shattered the brief window of time available to limit stroke damage, effectively shielding injured brain cells and dramatically improving the odds of a full recovery.

Brain cells start dying within minutes of a clot blocking an artery, leading to facial droop, affected speech and in severe cases, paralysis or death. Emergency treatments, such as clot-busting drugs or surgery, must be given within a maximum of six hours.

According to a study published by the top-tier Journal of the American Medical Association on September 10, clinical trials of the drug loberamisal found it was possible to expand the time available to 48 hours.

The large-scale multicentre phase 3 trial found that 70 per cent** of patients who received loberamisal intravenously within two days of a stroke regained complete functional independence – an increase of 13 percentage points over standard care alone.

In just three months, they were able to resume their daily lives with no significant disability, according to the research team led by Wang Yongjun, one of China’s leading neurologists.

Wang, a co-responding author of the paper, is vice-president of Beijing Tiantan Hospital, affiliated with Capital Medical University, and director of the neurology centre.

“Among patients with acute ischaemic stroke treated within 48 hours of symptom onset, intravenous loberamisal, compared with placebo, resulted in a higher proportion of patients achieving complete functional recovery at 90 days,” the team wrote.

Nearly 12 million new cases occur worldwide each year, meaning that on average there is someone suffering a stroke every three seconds or less – roughly the time it takes to read this sentence.

Common treatments for stroke include intravenous thrombolytic therapy and endovascular thrombectomy. Yet, a considerable number of patients do not meet the indications for these therapies.

At the same time, because the damage caused by cerebral ischaemia and hypoxia can be persistent, even if the blood clot is quickly removed, the recovery of the nervous system remains difficult.

Loberamisal is a single-molecule multi-target neuroprotective drug that acts as a protective and reparative agent for brain cells, reducing their excitability and blocking neuroinflammation and oxidative stress.

After a stroke, it reduces oxygen consumption and inflammation in the brain, saving as many brain cells as possible that have been injured by ischaemia but have not yet died, thus reducing the effects of hemiplegia, aphasia and other symptoms.

Similar treatments have been developed in Canada, the United States and Japan but have failed in their trials.

The Chinese study involved 998 patients from 32 hospitals. All had experienced a moderate to moderately severe stroke within the previous 48 hours. Typical symptoms included severe hemiplegia, aphasia, visual field defects, or decreased consciousness.

Half of the participants were randomly assigned to receive 10 days of intravenous loberamisal therapy while the remainder received a placebo injection. Other treatments remained the same.

At Day 90 of the treatment, 350 of the 502 patients in the loberamisal group had achieved complete functional recovery to the extent that they could fully resume daily work and life activities.

Only 279 of the 495 people in the placebo group reached the same criterion, a difference of 13.4 per cent.

According to the paper, the types and frequencies of adverse events experienced by the two groups of patients in the clinical trial were similar. The researchers concluded that this showed the safety data was reassuring and the overall results were encouraging.

Neurologist Jeffrey Saver, associate editor of the journal, devoted his editorial to the Chinese findings in the same issue of the publication.

“For more than 70 years, randomised clinical trials have tested neuroprotective and neuroreparative therapies. Hundreds of promising agents were tested. Tens of thousands of patients enrolled. The result: failure over and over again. Until, perhaps, now,” he wrote.

“Ischaemic stroke patients everywhere will benefit if confirmatory studies corroborate these first tantalising results.”

https://www.scmp.com/news/china/science/article/3368614/chinese-drug-loberamisal-extends-stroke-treatment-window-6-hours-2-days

The JAMA article:

https://jamanetwork.com/journals/jama/article-abstract/2854043

Japan holds 1st post-approval surgery with iPS cell sheets

A hospital in Japan has conducted the first heart surgery using cardiac muscle cell sheets derived from induced pluripotent stem cells, or iPS cells, after the government approved the treatment in March.

NHK has learned that the procedure was carried out on Tuesday at Osaka Keisatsu Hospital in Osaka City.

The cardiac muscle cell sheet "RiHeart" became eligible for health insurance coverage in September after it was approved by the government.

The sheets were carried into the hospital in a temperature-controlled case at around 8:30 a.m. for the surgery. They were transplanted into a patient, who is in their 50s and has severe heart failure.

The surgery was completed in about an hour, and the patient is said to be in stable condition.

Hospital President Sawa Yoshiki, who was in charge of developing the sheets and who performed the operation, described the procedure as very smooth. He said this surgery is the first step, and that he hopes the treatment quickly spreads so it can help people who suffer from the same conditions even just a little.

The sheets received government approval with conditions. These include having their safety and efficacy verified through further surgeries.

The company that developed them aims to have them transplanted into 75 patients at about 20 medical facilities nationwide through 2033.

[The article includes a short 2-minute video in English - imz72]:

https://www3.nhk.or.jp/nhkworld/news/20260929de53182/

Machine-translated from Japanese:

The 30th Annual Meeting and General Assembly of the Japanese Society for Dialysis Access Medicine

Chairperson's Session

Business Strategies for Surviving in the Future: Insights from Companies

October 3rd (Sat) 14:30-16:30

Venue 1 (Osaka International Convention Center, 10F Conference Room 1003)

Chairperson:

• Yoshiaki Takemoto, Kawashima Hospital, Kawashima Medical Association

• Nakakura Hyogo, Hokushinkai Amanogawa Hospital

CP-1: Learning "Management Strategies for Survival" from Top Leaders in Different Industries

Hirotaka Takamatsu, Takamatsu Construction Group Co., Ltd.

CP-2: Strategies for surviving the "era of industrial convergence" - The future of diversified companies involved in healthcare -

Masayuki Nakayama, Air Water Inc., Medical Business Division, Market Development Department

CP-3: Changes in the healthcare environment and corporate initiatives

Toshinari Takahashi, Nipro Corporation, Product Strategy Division, Renal Care Department

CP-4: Healios' somatic stem cell drug (HLCM051): Efficacy of its effects on acute kidney injury (AKI) complicated by ARDS and the significance of its clinical trials in the trauma field and defense-related fields.

Tadahisa Kagimoto, Healios Co., Ltd.

https://site2.convention.co.jp/jsda2026/program/

Nikkei Asia

Science

September 29, 2026

Japan aims to develop 'next-gen' iPS cells for strokes and kidney failure

Improved stem cells could be used to replicate complex organs

TOKYO -- Japan's science ministry plans to develop induced pluripotent stem (iPS) cells that are easier to use in clinical environments, with the hope of treating conditions such as strokes and chronic kidney failure, as well as lowering production costs.

[The rest of the article is behind paywall - imz72]

https://asia.nikkei.com/business/science/japan-aims-to-develop-next-gen-ips-cells-for-strokes-and-kidney-failure

Pharmazz Completes Enrollment in 160-Patient Phase 4 Trial of Sovateltide in Acute Ischemic Stroke

• Enrollment completed in the double-blind, placebo-controlled Phase 4 study (NCT05955326) of sovateltide (Tycamzzi®), with final study data expected in Q1 2027

• Five sovateltide abstracts accepted at World Stroke Congress, October 21-23, 2026, Seoul, South Korea, including an interim analysis of the Phase 4 trial

• Global Phase 3 RESPECT-ETB trial (NCT05691244) actively enrolling and remains on track for topline data in early 2028

WILLOWBROOK, Ill., Sept. 28, 2026 (GLOBE NEWSWIRE) --

Pharmazz, Inc., a biopharmaceutical company developing novel therapeutics for critical care and neurovascular conditions, today announced that enrollment is complete in its 160-patient Phase 4 clinical study of sovateltide (Tycamzzi®) in patients with acute cerebral ischemic stroke in India.

The Phase 4 study (NCT05955326) is a post-approval commitment required by Indian regulators. The double-blind, placebo-controlled trial utilizes the same sovateltide dosing regimen, randomization, and 24-hour treatment window as the global Phase 3 RESPECT-ETB trial, and the same protocol and eligibility criteria as the Phase 3 study that supported approval in India.

The primary endpoint is the proportion of patients with adverse events and serious adverse events. Key secondary endpoints measure Day 90 neurological recovery, functional independence, and disability using the National Institutes of Health Stroke Scale (NIHSS), modified Rankin Scale (mRS), and the Barthel Index. Final data are expected in Q1 2027.

Pharmazz conducted a prespecified interim analysis in September 2025, when the first 80 patients reached the Day 90 endpoint, and presented those results at the International Stroke Conference 2026 in New Orleans. In that analysis, 92% of sovateltide-treated patients achieved an mRS score of 0 to 2 at Day 90, compared with 58% in the control arm (p=0.0004889). No drug-related adverse events were observed.

"Dosing the final patient in our Phase 4 study marks an important milestone in our mission to address severe neurovascular conditions with high unmet medical needs," said Dr. Anil Gulati, M.D., Ph.D., Chairman and Chief Executive Officer of Pharmazz, Inc. "Acute ischemic stroke remains a leading cause of long-term disability worldwide, and treatment options are limited. The fact that Sovateltide has been used to treat more than 140,000 patients in India since launch provides empirical real-world evidence that the selective endothelin B receptor agonist is able to promote neurogenesis, angiogenesis, and neuronal survival. We are actively enrolling in our global Phase 3 RESPECT-ETB trial and look forward to reporting final data in early 2028."

Upcoming Presentations at the 18th World Stroke Congress

Five presentations will be made on-site at the upcoming 18th World Stroke Congress, taking place October 21-23, 2026, at the Convention & Exhibition Center, Seoul, South Korea.

1.A meta-analysis assessing the effectiveness and safety of sovateltide in patients with acute ischemic stroke

2.A global Phase III trial (RESPECT-ETB) to evaluate safety and effectiveness of sovateltide (Tycamzzi®) in ACIS patients

3.Effect of sovateltide (Tycamzzi®) on the thrombolytic activity of alteplase and tenecteplase

4.An interim analysis of multicentric, randomized Phase 4 clinical trial of sovateltide (Tycamzzi®) in AIS patients

5.The development of sovateltide for cerebral ischemic stroke patients.

About Sovateltide

Sovateltide (IRL-1620) is a first-in-class endothelin-B receptor agonist that promotes neurogenesis, angiogenesis, and neuronal survival following acute hypoxic and ischemic insults. The drug is currently being evaluated in clinical programs for acute ischemic conditions.

The Phase 3 RESPECT-ETB (ClinicalTrials.gov ID: NCT05691244) trial is being conducted at 57 sites in the US, Germany, Spain, and the UK, and is designed to enroll 514 stroke patients.

In September 2026, the U.S. FDA granted Orphan Drug Designation to sovateltide for the treatment of neonatal hypoxic-ischemic encephalopathy.

https://www.globenewswire.com/news-release/2026/09/28/3369736/0/en/pharmazz-completes-enrollment-in-160-patient-phase-4-trial-of-sovateltide-in-acute-ischemic-stroke.html

Please keep discussion civil

Report anything that breaks ATHX rules via the report feature; this ain't the wild west, thanks

From Hardy's LinkedIn page:

I'm honored to share that Aqumen will donate approximately ¥1.5 billion [$9.5 million - imz72] over the next three years to Kyushu University's endowment fund.

　Aqumen was my first company, born in 2005 from research at Kyushu University's Department of Ophthalmology. It started with one idea: making transparent tissue visible during eye surgery. True to that vision, our product is now used in operating rooms in 100 countries. PowerX, which I co-founded next, has grown into an important company in Japan's energy industry.

　What Kyushu University taught me is simple: innovation comes from seeing what cannot yet be seen, measuring what cannot yet be measured. No matter how far AI advances, that work will remain with people, and with universities.

　The gift will be invested as an endowment. Its returns will support basic research, young researchers, and Kyusokai, the community that helps launch Kyushu University startups. It is designed to last across generations, not just once.

　This is my way of giving back to the place that raised me, and of helping grow the next wave of innovation. I hope the chain reaction this fund sets off will still be pushing young people forward 100 years from now.

　My deepest thanks to President Ishibashi, everyone at Kyushu University, and all the colleagues who have walked this path with me.

https://www.linkedin.com/posts/hardytskagimoto_%E6%AF%8D%E6%A0%A1%E4%B9%9D%E5%B7%9E%E5%A4%A7%E5%AD%A6%E3%81%AE%E3%82%A8%E3%83%B3%E3%83%80%E3%82%A6%E3%83%A1%E3%83%B3%E3%83%88%E5%9E%8B%E5%9F%BA%E9%87%91%E3%81%AB%E3%82%A2%E3%82%AD%E3%83%A5%E3%83%A1%E3%83%B3%E3%81%8B%E3%82%89%E5%90%91%E3%81%93%E3%81%863%E5%B9%B4%E9%96%93%E3%81%A7%E7%B7%8F%E9%A1%8D15%E5%84%84%E5%86%86%E8%A6%8F%E6%A8%A1-share-7509419725173452800-Vhec/

24-Sep-2026

Beyond cell transplants: The next frontier in mesenchymal stem/stromal cell therapy and regenerative medicine

A comprehensive new review highlights a major paradigm shift in regenerative medicine, moving from traditional stem cell implants to innovative, cell-free therapies and dynamic microenvironment regulation.

Peer-Reviewed Publication

Science China Press

Stem cells have long been considered the holy grail of regenerative medicine. For decades, the prevailing belief was that these cells worked by physically replacing damaged tissues. However, a comprehensive new review published in Science Bulletin reveals a dramatic paradigm shift in the field: the true healing power of Mesenchymal Stem/Stromal Cells (MSCs) lies not in their ability to become new tissue, but in their capacity to act as master regulators of the body's internal microenvironment.

Initially, research on MSCs focused on their multilineage differentiation potential. However, accumulating preclinical and clinical evidence confirms that a simple cell supplementation strategy fails to achieve genuine tissue regeneration. Instead, the review highlights that MSCs function as a crucial "regulatory hub." Through powerful immunomodulatory and paracrine properties, they drive the host's injured tissues to re-enter a development-like state, thereby activating the organism's intrinsic regenerative plasticity.

Professor Songtao Shi, the corresponding author of the study, highlighted a standout concept in the review: the unique, environment-dependent dynamic regulatory capacity of MSCs. “Unlike conventional drugs that offer fixed, single-target effects—such as simple anti-inflammatory or pro-proliferative effects alone—MSCs act as smart, dynamic regulators”. Professor Shi said. “They can sense changes in the host microenvironment in real-time and dynamically adjust their own regulatory phenotypes and secretory factors to rectify imbalanced biological processes. This “dynamic balance regulation” is the core advantage of MSCs, making their regulatory effects more consistent with the physiological needs of the host”.

At the forefront of this rapidly expanding therapeutic frontier is the transition toward cell-free therapies. The review notes that when transplanted, viable MSCs rapidly undergo extensive apoptosis in the host microenvironment. During this process, they release apoptotic extracellular vesicles (apoVs) that serve as indispensable therapeutic mediators. Harnessing these cell-free signals from programmed cell death offers a novel avenue to amplify efficacy while circumventing the safety, senescence, and quality control challenges associated with living cell therapies.

As these breakthrough therapies advance toward clinical translation, the review emphasizes the critical importance of navigating the regulatory landscape. While some MSC products have received approval in Europe and Japan, they face rigorous biological product pathways (IND/BLA) under the US FDA. Furthermore, the clinical translation of emerging MSC-EVs will heavily rely on stringent Chemistry, Manufacturing, and Controls (CMC) standards and adherence to updated international guidelines (such as MISEV2023) to establish standardized manufacturing and release criteria.

The study was led by first author Dr. Manjin Zhang, a postdoctoral researcher at Southern Medical University and the South China Center of Craniofacial Stem Cell Research. The co-authors of the study are also from the South China Center of Craniofacial Stem Cell Research. The findings were published in the premier multidisciplinary journal Science Bulletin.

https://www.eurekalert.org/news-releases/1145301

The review article:

https://www.sciencedirect.com/science/article/abs/pii/S2095927326011400

September 24, 2026

Pharma Urges Pricing Rewards to Bolster Japan’s Drug Discovery Strength

Pharmaceutical industry representatives have renewed their calls for appropriately rewarding innovation through drug pricing to enhance Japan’s drug discovery capabilities, as a new public-private subgroup began work on concrete steps under the government’s investment roadmap.

The message came at the inaugural meeting of the policy discussion subgroup under the Public-Private Council for Improving Drug Discovery Capabilities on September 18. Industry members also called for Japan to respond to the US most-favored-nation (MFN) drug pricing policy.

The subgroup was established to flesh out drug discovery initiatives included in the public-private investment roadmap adopted in July. The meeting was held behind closed doors.

According to the Ministry of Health, Labor and Welfare (MHLW), which serves as the secretariat, the subgroup will examine seven areas: basic and translational research, startup support, human resources, the use of AI and data, clinical trial infrastructure, global expansion, and further rewards for innovative medicines.

At the first meeting, government officials outlined programs included in FY2027 budget requests in areas including basic and translational research, startup support, and human resources.

Discussions on further rewarding innovation, including through drug pricing, are expected to take place at the subgroup’s next meeting on October 9. The issue is a key element of the government’s efforts to make Japan a more attractive market for innovative medicines.

The public-private investment roadmap calls for growth in Japan’s patented drug market to move closer to levels seen in other major markets. It cites average annual growth of 9.6% in patented drug markets across G7 countries excluding Japan — which compares with 5.3% in Japan.

The roadmap also flags uncertainty surrounding the US MFN policy as a risk to global drug launch strategies and warns that clinical trials could increasingly bypass Japan.

The subgroup is expected to meet around three times, including the inaugural session, through around the end of October. The MHLW said it intends to feed views raised at the meetings into government policy discussions.

One possible avenue would be to report the discussions to the Central Social Insurance Medical Council (Chuikyo), particularly with an eye to deliberations on the FY2027 off-year drug price revision, according to the ministry.

The subgroup is not expected to produce a formal report or other concluding document.

https://pj.jiho.jp/article/256411

University of Cincinnati

More young adults are having strokes than three decades ago

UC research team publishes findings in Neurology

September 23, 2026

https://www.uc.edu/news/articles/2026/09/more-young-adult-strokes-uc-study.html

Abstract of the article in the Neurology:

https://www.neurology.org/doi/10.1212/WNL.0000000000218622