A trial run in China just handed Cue Biopharma a stock chart that spiked more than 40% before giving almost all of it back in the same session. Longeveron’s pediatric heart trial met the opposite fate, missing its main goal just days after the company finally cleared a Nasdaq compliance deadline it had spent a year fighting. Hitting the mark and missing it both came with complications this time.
Cue Biopharma’s CUE-221 Meets Primary Endpoint in Urticaria Trial
Cue Biopharma, Inc. (Nasdaq: CUE) announced positive topline results on September 20 from a Phase 2 trial of CUE-221 in moderate to severe chronic spontaneous urticaria (CSU), a chronic hives disorder that leaves many patients undertreated by standard antihistamines, and 145 patients enrolled. The trial met its primary endpoint and key secondary endpoint with high statistical significance, and at the highest of three doses tested, CUE-221 showed a different efficacy profile than Xolair (omalizumab), the current standard treatment.
The trial was not run by Cue. It was conducted in China by Genesis Life Sciences, a related company of Ascendant Health Sciences Ltd., the firm that licensed the molecule to Cue in April for a $15 million upfront payment plus up to $676.5 million in potential milestones and tiered royalties. Cue holds rights everywhere except mainland China, Hong Kong, Macau, and Taiwan, and plans to advance CUE-221 into its own Phase 2b/3 registration study in CSU following this data, alongside a separate Phase 2 study already planned in food allergy.
Shares jumped as much as 42% in premarket trading on September 21, then reversed and fell more than 27% intraday the same day as investors weighed the license’s remaining milestone obligations against the trial’s mixed reception. Cue held $17.4 million in cash as of June 30, after one-time payments tied to the Ascendant license, and has since raised an additional $50 million through a private placement, which management says extends its runway at least twelve months from its most recent financial statements.
Longeveron’s ELPIS II Trial Misses Primary Endpoint in HLHS
Longeveron Inc. (Nasdaq: LGVN) reported topline results on September 16 from ELPIS II, its Phase 2b trial of the stem cell therapy laromestrocel in hypoplastic left heart syndrome (HLHS), a rare and often fatal pediatric heart defect with orphan-drug designation. The trial did not meet its primary endpoint: change in right ventricular ejection fraction (RVEF) at month 12 showed a difference of just -0.7 percentage points between treatment groups, with a 95% confidence interval spanning -7.3 to 5.9 and a p-value of 0.8336, far from statistical significance.
In an exploratory as-treated analysis, no patients died in the treated group over twelve months, against one death in the control group, and laromestrocel’s safety profile held up across the 644 patients Longeveron has now dosed across its programs. Longeveron announced alongside the data that it is exploring all options to maximize shareholder value and starting cost-optimization measures, language the company has not yet translated into specifics.
The company disclosed substantial doubt about its ability to continue as a going concern in its most recent quarterly filing, with $10.1 million in cash as of June 30 guided to last only into the fourth quarter of 2026. Longeveron completed a 1-for-10 reverse stock split on August 26 to lift its share price back above Nasdaq’s $1.00 minimum bid requirement, and the exchange confirmed on September 11, before this data, that the company had regained compliance and closed the matter. The exchange issue is resolved. The clinical one is not. Longeveron also changed chief financial officers earlier this year, with Nirav Jhaveri succeeding Marie Washburn, who had been appointed to the role in July.
CUE, LGVN: Forward-Looking FAQ
Cue has not given a start date. The company says it will initiate a registrational Phase 2b/3 study in CSU following review of this data, but only after the regulatory and IND-enabling steps specific to its ex-China territory are complete.
That is not stated in Cue’s disclosures. The company has not specified which milestones in the license agreement are payment triggers, only that up to $676.5 million total is possible across development, regulatory, and commercial milestones.
The company has not defined the options. Possibilities range from further discussion with the FDA on the trial’s exploratory endpoints to partnering the program or discontinuing it, and Longeveron has ruled nothing in or out.
Not on its current cash position alone. The company has said it intends to pursue additional financing, capital raises, and non-dilutive funding, but has not disclosed a specific plan or amount.
Sources
- GlobeNewswire / Cue Biopharma: Cue Biopharma Announces Positive Topline Results from CUE-221 Phase 2 Study in Chronic Spontaneous Urticaria, September 20, 2026
- SEC 8-K Exhibit / Cue Biopharma: Exclusive License Agreement with Ascendant Health Sciences Ltd. for Ascendant-221 (UB-221), April 30, 2026
- GlobeNewswire / Cue Biopharma: Cue Biopharma Reports Second Quarter 2026 Financial Results and Recent Business Highlights, August 14, 2026
- GlobeNewswire / Longeveron: Longeveron Announces Topline Results from ELPIS II Phase 2b Clinical Trial Evaluating Laromestrocel as a Potential Treatment for Hypoplastic Left Heart Syndrome (HLHS), September 16, 2026
- GlobeNewswire / Longeveron: Longeveron Announces 2026 Second Quarter Financial Results and Provides Business Update, August 12, 2026
- SEC 8-K / Longeveron: Notification of Regained Nasdaq Minimum Bid Price Compliance, filed September 14, 2026 (event dated September 11, 2026)
- BioSpace / Longeveron: Longeveron Announces 1-for-10 Reverse Stock Split, August 24, 2026
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