Recombinant growth hormone-albumin fusion protein(Zhejiang Younuojin Biological Engineering Co Ltd)

A 1.6-mg/kg daily dose of LUM-201 was linked to an 8-cm increase in height over a year. Lumos Pharma's stock jumped Wednesday morning as a successful phase 2 trial maintained the Texan biotech’s hopes of bringing the first oral drug for pediatric growth hormone deficiency (PGHD) to market. The phase 2 OraGrowtH210 trial assessed LUM-201 in 82 participants with moderate PGHD. These participants were selected to be most likely to respond to the therapy, judged by predictive enrichment marker (PEM) criteria such as having a baseline insulin-like growth factor 1 (IGF-1) value above 30 ng/ml. Of the three doses tested, a 1.6-mg/kg daily dose of LUM-201 was linked to an 8-cm increase in height over a year, the biotech reported. This was within a 2-cm margin of comparable injectable growth hormones despite LUM-201 increasing growth hormone levels to just 20% of the concentration of these approved treatments, the company noted. Preliminary data from both the 1.6-mg/kg and 3.2-mg/kg dose cohorts suggested the drug maintained its effectiveness up to 24 months, the biotech added.  However, Lumos stressed that the main purpose of the study was not to prove that the therapy was effective. Instead, the primary endpoint—which the trial hit—was simply to validate its PEM test. The biotech said the success of this criterion will “de-risk” the selection of patients for the phase 3 trial, which the company is now looking toward. Lumos also reported the results of a separate phase 2 trial of 24 participants, dubbed OraGrowtH212, which the company said confirmed that LUM-201's mechanism of action produces an increase in growth rates by restoring growth hormone secretion and IGF-1 to within normal ranges. The safety profile for LUM-201 in both trials “remained clean” with no “safety concerns identified … thus far,” the company added. Investors appeared impressed, sending the biotech’s stock up over 30% in premarket trading to $4.77 per share from a Tuesday closing price of $3.64. “Our data indicates that LUM-201 can enhance annualized height velocity in line with established standards for moderate PGHD patients undergoing recombinant growth hormone therapy, demonstrating a robust and durable response,” CEO Rick Hawkins said in the release. “We look forward to discussing these data and finalizing our plans for a phase 3 pivotal trial with the FDA in our end of phase 2 meeting anticipated in the first half of 2024,” he added. The biotech expects the $42.7 million in the bank as of the end of September will be enough to fund the planning and launch of this late-stage trial.