Lumos Pharma announces OraGrowtH210, OraGrowtH212 meet all endpoints

Lumos Pharma announced that topline results from its Phase 2 OraGrowtH210 dose-finding trial and its Phase 2 OraGrowtH212 Pharmacokinetic/Pharmacodynamic trial met all primary and secondary endpoints. Data from the OraGrowtH210 Trial demonstrated annualized height velocity, or AHV, on the 1.6 mg/kg dose of orally administered LUM-201 of 8.2 cm/yr at six months and 8.0 cm/yr at 12 months on treatment, in line with historical data in moderate pediatric growth hormone deficiency, or PGHD, patients and within the targeted 2 cm/yr margin of the comparator injectable recombinant growth hormone, or rhGH arm. Data also provided preliminary validation of the predictive enrichment marker, or PEM, strategy, with prespecified primary and secondary outcomes met, de-risking our patient selection for our Phase 3 program. Data from the OraGrowtH212 Trial confirmed that LUM-201’s unique pulsatile mechanism produces an increase in growth rates while restoring growth hormone secretion and IGF-1 to within normal ranges , with levels substantially below those produced by exogenous injectable rhGH. Additionally, data from a small subset of 10 subjects combined 1.6 and 3.2 mg/kg dosage of LUM-201 in both OraGrowtH210 and OraGrowtH212 trials demonstrated the sustained effectiveness of AHV up to 24 months. Furthermore, the safety profile for LUM-201 remained clean throughout both Phase 2 studies, with no safety concerns identified in either of our Phase 2 trials conducted thus far. Dosage at 1.6 mg/kg demonstrates highest LUM-201 AHV at six months and 12 months 1.7 cm/yr difference between 1.6 mg/kg LUM-201 dose and rhGH comparator arm at 12 months falls within historical non-inferiority Phase 3 margins LUM-201 AHVs align with historical growth rates of rhGH in patient populations with similar characteristics. 12-month AHV data available for 50/81 subjects: Growth rates durable at 12 months. The mean AHVs at six months and 12 months observed in the 1.6 mg/kg dose LUM-201 arm were 8.2 cm/yr and 8.0 cm/yr, respectively. These AHVs were in line with the Company’s expectations for 8.3-8.6 cm/yr AHV observed after 12 months of rhGH treatment in a moderate PGHD patient population. The higher than anticipated AHV seen in this moderate PGHD population treated in the rhGH control arm of the OraGrowtH210 Trial was inconsistent with multiple historical trials which predicted growth in the 8.3-8.6 cm/yr range for moderate PGHD1-4. This distinctive growth pattern observed in the daily GH arm of this study is likely due to a higher dosage and the presence of outliers. We anticipate that in a larger, more statistically robust Phase 3 trial, the AHV associated with rhGH treatment will align more closely with historical values for the moderate patient population. The OraGrowtH210 Trial met the prespecified percent responder enrichment providing preliminary validation of the PEM strategy. Additionally, we have achieved a 100% success rate in meeting the predetermined outcome for positive PEM specification classification reproducibility. The topline results from the OraGrowtH212 Trial reveal that LUM-201 achieved an expected AHV with only 20% of the growth hormone, or GH, concentration observed using injectable rhGH. This outcome was achieved through LUM-201’s natural pulsatile mechanism, promoting growth in moderate PGHD subjects that align with historical norms. Notably, LUM-201 raised circulating GH to levels closer to normal physiological ranges, whereas treatment with injectable rhGH has been shown to elevate GH levels to four to five times that of typical healthy children. Furthermore, it’s important to highlight that during the first 12 months of LUM-201 treatment, no IGF-1 values exceeded 2 standard deviations from the mean. Eighteen and 24-month growth data were available for 10 subjects from the OraGrowtH210 and OraGrowtH212 Trials who met AHV criteria per protocol at 12 months. Combined data from the 1.6 mg/kg and 3.2 mg/kg cohorts of both trials demonstrate sustained AHVs from 12 to 24 months without a considerable decline in growth velocity compared to the previously reported ~20% decline in AHV on rhGH from 12 to 24 months observed in the Pfizer Phase 4 KIGS dataset. The topline results from both the OraGrowtH210 and OraGrowtH212 trials have shown a clean safety record, characterized by an absence of treatment-related Serious Adverse Events, or SAEs, no instances of participants discontinuing treatment due to adverse events, or AEs, and the absence of any significant safety concerns in various parameters such as laboratory values, adverse event data, or in electrocardiogram readings.