Maintaining Buy on Gain Therapeutics: Strong GCase Biology, Validated GluSph Biomarker, and Favorable Risk-Reward for GT-02287 in Parkinson’s Disease

In a report released yesterday, Thomas Shrader from BTIG maintained a Buy rating on Gain Therapeutics, with a price target of $5.00.

Thomas Shrader has given his Buy rating due to a combination of factors that reinforce the biological and clinical rationale behind Gain Therapeutics’ lead program, GT-02287. He views the recent KOL event and emerging data as validating that GT-02287 meaningfully boosts GCase enzyme activity, as reflected by consistent reductions in CSF glucosylsphingosine (GluSph), a toxic substrate closely linked to Parkinson’s disease pathology. The fact that GluSph levels in treated patients move toward those seen in healthy individuals, and that GluSph is considered a more reliable and stable biomarker than glucosylceramide, supports the potential for true disease modification rather than just symptomatic relief. Shrader also highlights the mechanistic argument that shifting patients from severely impaired GCase function toward a milder functional state should translate into slower clinical progression, which he views as stronger genetic and biological support than seen for many competing approaches.
In addition, Shrader emphasizes Gain’s targeted “molecular staple” strategy, which stabilizes both wild-type and mutant GCase and improves its cellular trafficking, including to mitochondria, thereby addressing multiple aspects of the disease mechanism. Preclinical results showing reduced mitochondrial stress, better neuronal survival, improved complex I activity, and enhanced mitochondrial GCase in models with severe GBA1 mutations bolster his confidence that the drug’s effects extend beyond a single pathway. With most patients now continuing in an open-label extension and additional Parkinson’s-related readouts expected ahead of 12‑month clinical outcomes, he sees a favorable risk‑reward profile at current levels. These scientific, translational, and valuation considerations underlie his decision to maintain a Buy rating on GANX.

In another report released on December 30, Maxim Group also maintained a Buy rating on the stock with a $7.00 price target.