Tanabe Pharma Corp.

Axoltis Pharma's phase 2 trial for amyotrophic lateral sclerosis (ALS) may have missed its primary endpoint, but the French biotech has pointed to secondary endpoints to justify persevering with the synthetic peptide. The phase 2 study enrolled 82 patients with ALS in a double-blind, randomized, multicenter trial in France. Patients received either the drug, dubbed NX210c, at 5 mg/kg or 10 mg/kg doses or a placebo three times weekly for four weeks. At six weeks, patients did not show a significant change from baseline in blood neurofilament light chain (NfL), a test used to evaluate leakage of NfL protein into the bloodstream when nerve cells are injured. While there was a trend of a decrease of NfL in the blood in 25.4% of patients in the NX210c arms of the study compared to 11.8% among the placebo-takers after four months, these results were not significant, Axoltis noted in a Sept. 15 release. Benefits in motor function scale, measured using the harmonized revised amyotrophic lateral sclerosis functional rating scale, were observed with 64% and 33% reductions in patients treated with 5 mg/kg and 10 mg/kg NX210c, respectively. The reductions suggest improvement or slowing of decline in how well a person with ALS can control their physical and limb movements, according to Axoltis. There was also a significant decrease in blood claudin-5, a protein released into the bloodstream when the blood-brain barrier (BBB) is disrupted, suggesting restoration of the BBB among the patients treated with 10 mg/kg of NX210c compared to those who received the placebo, the biotech said. “The observed effect of NX210c in reducing plasma claudin-5 opens new avenues for the use of NX210c to treat a broad range of indications where blood-brain barrier dysfunction is observed, among them Alzheimer’s disease, Parkinson’s disease and multiple sclerosis,” Axoltis CEO Yann Godfrin, Ph.D., argued in the release. ALS, also known as Lou Gehrig’s disease, is a neurodegenerative disease that destroys the nerve cells in the brain and spinal cord. As these nerve cells die, they stop sending signals to your muscles leading to impaired walking, talking, chewing and breathing. The BBB is the brain’s protective wall responsible for blocking toxins and other pathogens from entering the brain while allowing nutrients to pass through to the organ. The barrier is made of endothelial cells that are packed very tightly so that forbidden molecules cannot pass. If a molecule wants to enter the brain, it has to either be small enough to diffuse through the tightly packed cells or use a transporter, such as claudin-5, a tight junction protein that is one of the main gatekeepers of the brain. Axoltis' NX210c has a three-pronged approach of targeting BBB restoration, protecting neurons and enhancing intercellular communication. The therapy contains sequences from the subcommissural organ (SCO)-spondin, which is a glycoprotein essential for the process of neuron development that the SCO produces during embryonic development. “These results are promising and contribute to generating evidence that NX210c’s properties are beneficial in neurodegenerative diseases, especially ALS,” Axoltis Chief Medical Officer Annette Janus, M.D., said in the release. “These findings support advancing NX210c to the next stages of clinical development.” Available ALS therapies include Biogen's genetically targeted therapy Tofersen, which is used by patients with the SOD1 mutation. There's also Mitsubishi Tanabe's central nervous system agent Radicava—for which Shionogi acquired the rights last year—and the glutamate antagonist riluzole. Other potential therapies are in the works. Coya Therapeutics finally received the go-ahead last month from the FDA to kick off a phase 2 study of COYA 302—which combines the biotech's IL-2 candidate with a CTLA4-Ig—while Corcept Therapeutics unveiled promising survival data in June from its mid-stage study in ALS despite a failure to slow ALS progression . Eli Lilly also became a player in the space last year after it penned a $415 million deal to license Alchemab’s early-stage ALS drug program.

LEO Pharma has agreed to acquire worldwide rights to dersimelagon from Tanabe Pharma. Dersimelagon is an oral, once-daily small MC1R agonist being developed for EPP and XLP. EPP and XLP are rare and severe genetic diseases that cause severe sunlight-induced skin damage and can significantly limit patients’ ability to spend time outdoors. Symptoms may include skin rash, swelling, redness, burning sensations and, in some cases, liver damage. Dersimelagon is designed to increase skin melanin, helping reduce sunlight penetration and protect against phototoxic reactions. “Patients living with EPP or XLP face the devastating lifelong burden of severe reactions to sunlight, and there is a clear need for treatment options that can make a meaningful difference in their everyday lives,” said Christophe Bourdon, CEO of LEO Pharma. “By addressing a clear unmet need in a rare skin disease, dersimelagon represents a compelling opportunity to expand our rare dermatology pipeline with a late-stage oral therapy candidate.” If approved, dersimelagon would represent the first oral therapy for the treatment of EPP and XLP and help address an area of significant unmet need. Under the terms of the agreement, LEO Pharma will acquire worldwide rights to dersimelagon from Tanabe Pharma for up to $435m in up front and near-term milestone payments together with potential down- stream milestones and tiered royalties on net sales. Dersimelagon has been granted both US FDA Fast Track Designation and Orphan Drug Designation, and an NDA for the treatment of EPP and XLP was submitted to the US FDA for regulatory review in June 2026, but has not yet been approved by the FDA or any other regulatory agency. “EPP and XLP are devastating lifelong diseases that can severely limit patients’ ability to live everyday lives free from sunlight-induced pain, and dersimelagon has the potential to become an important new treatment option,” said Akihisa Harada, CEO of Tanabe Pharma. Earlier this year, Tanabe Pharma announced results from the global, randomised, double-blind, placebo-controlled phase 3 INSPIRE study of EPP and XLP that showed dersimelagon demonstrated statistically significant and clinically meaningful outcomes across primary and secondary endpoints, including key functional outcomes such as a significant prolongation of average daily sunlight exposure time to first prodromal symptoms. The phase 3 data was presented as a late breaker at the 2026 American Academy of Dermatology Annual Meeting (AAD).

China's investigator-initiated trials, once praised for their speediness, have been drawn into scrutiny for a lack of transparency. AstraZeneca and Daiichi Sankyo's Enhertu earned a win in a phase 3 non-small cell lung cancer trial. Tanabe Pharma secured a $435 million payout for global rights to its dermatology rare disease treatment, dersimelagon. And more. 1. After 2 undisclosed gene therapy deaths, can China’s alternative regulatory path still be trusted? The high-speed regulatory pathway credited with fueling China’s biotech boom is under international scrutiny after several previously undisclosed deaths—beginning with two in young children with rare genetic conditions—were uncovered in a matter of weeks. China’s investigator-initiated trials (IIT) allow Chinese hospitals and academics to launch early-stage human trials under a separate regulatory pathway with less oversight than the traditional drug approval process. Fierce talked to experts about what the patient deaths and poor public disclosure could mean for the IIT system. 2. AstraZeneca touts Enhertu, Orpathys dual lung cancer wins amid bispecific failure In a pair of phase 3 readouts for niche populations in non-small cell lung cancer, AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu bested Keytruda in first-line HER2-mutant nonsquamous NSCLC, while its Huchmed-partnered Orpathys-Tagrisso combination outperformed platinum-based chemo in Tagrisso-pretreated EGFR-mutated disease. The wins, however, were overshadowed by a phase 3 flop from the investigational bispecific volrustomig, which failed to outdo Keytruda and chemo this week in first-line NSCLC patients whose tumors express PD-L1 at below 50%. 3. Biokin bispecific ADC iza-bren hits goal in lung cancer trial, boosting case for BMS’ global test Biokin Pharma’s phase 3 trial for iza-bren, a first in-class antibody drug conjugate that targets both EGFR and HER3, met its primary endpoint in an interim analysis, demonstrating significant progression-free survival benefits. The success of the trial conducted in China increases the chances of success for the drug’s global trial, which launches this fall and is spearheaded by global partner Bristol Myers Squibb. 4. Leo ‘really strengthening’ pipeline with $435M deal for Tanabe rare skin disease drug awaiting FDA nod Tanabe Pharma is set to receive $435 million in upfront and near-term milestone payments after a deal with Leo Pharma. In exchange, Leo gets global rights to dersimelagon, a treatment in development for the rare genetic dermatology conditions erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP). Tanabe announced a phase 3 win for the drug in both indications earlier this year and now awaits an approval decision from the FDA. 5. Sandoz strikes $322M biosimilars deal with Shanghai Henlius Amid growing biosimilar opportunities, Sandoz has entered a collaboration with Shanghai Henlius and procured rights to three early development biosimilars. Per the deal, which could cover as many as 10 biosimilars, Sandoz is paying $100.5 million for the initial assets upfront, and up to $322 million if development milestones are achieved. Third death in China's popular but opaque trials revealed, this time in CAR-T (Endpoints) SK strikes settlement with MSN over future Xcopri generic: reports Kolon TissueGene shrinks workforce after pivotal osteoarthritis trial miss Contractor SK Pharmteco heralds completion of 2 API plants in Ireland Aurobindo unit, China API maker dinged in latest round of FDA warning letters China's top drugmaker Hengrui sees profit growth squeezed by bulk-buying programs (Reuters) Sino Biopharmaceutical rebrands to SBP Group (release)