Valo Health Announces Topline Results from Phase 2 SPECTRA Study of OPL-0401 in Patients with Diabetic Retinopathy

Lex­ing­ton, MA, Decem­ber 31, 2024 – Valo Health, Inc (Valo), a biotech­nol­o­gy com­pa­ny focused on uti­liz­ing large-scale data and AI-dri­ven com­pu­ta­tion to dis­cov­er and devel­op ther­a­peu­tics, today announced topline data from its Phase 2 SPEC­TRA study of OPL-0401 in patients with dia­bet­ic retinopa­thy (DR). While the study did not meet its pri­ma­ry or sec­ondary end­points based on the analy­sis in the pre­de­fined pri­ma­ry pop­u­la­tion, cer­tain dos­es test­ed in the study showed poten­tial evi­dence of pre­vent­ing dis­ease pro­gres­sion. Safe­ty and tol­er­a­bil­i­ty were in line with clin­i­cal stud­ies con­duct­ed pri­or to in-licens­ing of OPL-0401, with evi­dence now in near­ly 300 patients and healthy vol­un­teers demon­strat­ing that OPL-0401 is well-tol­er­at­ed. Giv­en Valo’s renewed strate­gic focus on lever­ag­ing the poten­tial of its unique Opal plat­form in devel­op­ing new med­i­cines, the Com­pa­ny has made the strate­gic deci­sion to sus­pend its devel­op­ment of OPL-0401 and seek a part­ner to fur­ther devel­op the program.

“There is a sig­nif­i­cant unmet med­ical need for patients suf­fer­ing from dia­bet­ic retinopa­thy, a lead­ing cause of blind­ness in the US, and cur­rent treat­ment options con­sist main­ly of inva­sive intrav­it­re­al injec­tions and laser treat­ment tar­get­ed to patients with more severe or advanced dis­ease,” said Bri­an M. Alexan­der, M.D., M.P.H., Chief Exec­u­tive Offi­cer of Valo Health and CEO-Part­ner of Flag­ship Pio­neer­ing. ​“In light of this crit­i­cal need, we are dis­ap­point­ed that OPL-0401, a poten­tial first-in-class oral option for patients, did not reach its pri­ma­ry or sec­ondary objec­tives. How­ev­er, we are encour­aged by the favor­able safe­ty pro­file and evi­dence of clin­i­cal activ­i­ty with cer­tain dos­es, sug­gest­ing the poten­tial of OPL-0401 to ben­e­fit DR patients. These data col­lec­tive­ly sup­port the fur­ther clin­i­cal eval­u­a­tion of OPL-0401.”

Dr. Alexan­der con­tin­ued, ​“While the data were intrigu­ing, OPL-0401 did not incor­po­rate ele­ments of our plat­form in dis­cov­ery or devel­op­ment. As a result, we are look­ing to find a part­ner well posi­tioned to real­ize the clin­i­cal poten­tial OPL-0401 in DR and beyond. Mov­ing for­ward, Valo’s renewed strate­gic focus will be on oppor­tu­ni­ties that lever­age our Opal plat­form to dis­cov­er new ther­a­peu­tic tar­gets in real world data, val­i­date those tar­gets in human-cen­tric mod­els, and devel­op new med­i­cines with our AI enabled closed loop small mol­e­cule design.”

Charles Wykoff, M.D., Ph.D., Direc­tor of Research at Reti­na Con­sul­tants of Texas in Hous­ton and SPEC­TRA Lead Inves­ti­ga­tor, said, ​“We sin­cere­ly thank all of the patients, inves­ti­ga­tors, and study coor­di­na­tors who par­tic­i­pat­ed in this study. DR, both NPDR and PDR, remain major caus­es of vision loss in the US and glob­al­ly, and there remains an impor­tant unmet need for safe and effec­tive oral ther­a­pies for patients. While the pri­ma­ry end­points in the cur­rent tri­al were not achieved, post hoc analy­ses sug­gest­ing pre­ven­tion of dis­ease pro­gres­sion at cer­tain dos­es is inter­est­ing. I hope to see con­tin­ued devel­op­ment of OPL-0401 to fur­ther explore this poten­tial­ly promis­ing therapy.”

SPEC­TRA (NCT05393284) was a mul­ti-cen­ter ran­dom­ized, dou­ble-blind, place­bo-con­trolled, 24- week study in patients with DR. A total of 114 patients with mod­er­ate­ly severe or severe non-pro­lif­er­a­tive dia­bet­ic retinopa­thy (NPDR) or mild pro­lif­er­a­tive dia­bet­ic retinopa­thy (PDR) were enrolled. The study’s pri­ma­ry objec­tive was to assess the effi­ca­cy of OPL-0401 com­pared to place­bo in improv­ing dia­bet­ic retinopa­thy sever­i­ty score (DRSS). The pri­ma­ry end­point (the por­tion of patients with a two or more step improve­ment of DRSS in the study eye as com­pared to place­bo), and the key sec­ondary end­point (the por­tion of patients with a three or more step change of DRSS in binoc­u­lar eyes) did not meet sta­tis­ti­cal sig­nif­i­cance. Treat­ment-relat­ed adverse events were infre­quent and mild. There were no treat­ment-relat­ed seri­ous adverse events, and no adverse events relat­ed to liv­er, kid­ney, or heart func­tion were observed. The results from one dose group test­ed in a small num­ber of patients demon­strates a con­sis­tent trend across sev­er­al key effi­ca­cy vari­ables includ­ing the DRSS change and pro­gres­sion to vision threat­en com­pli­ca­tion. This trend needs to be con­firmed in future studies.

Paul Bion­di, Exec­u­tive Chair of Valo, Flag­ship Pio­neer­ing Gen­er­al Part­ner and Pres­i­dent of Pio­neer­ing Med­i­cines, added, ​“Valo’s Opal plat­form rep­re­sents a fun­da­men­tal shift in the way we can dis­cov­er new ther­a­peu­tic tar­gets and devel­op med­i­cines against them. We look for­ward to con­tin­u­ing to real­ize the vast poten­tial of the plat­form and con­tin­u­ing our momen­tum in car­diometa­bol­ic diseases.”

The Opal Com­pu­ta­tion­al Plat­form is an AI-dri­ven tar­get and drug devel­op­ment engine designed to accel­er­ate the devel­op­ment of bet­ter med­i­cines for patients. The plat­form lay­ers an unmatched vol­ume and qual­i­ty of human data from mul­ti­ple exclu­sive and pro­pri­etary data access agree­ments with promi­nent glob­al health sys­tems on top of Valo’s dis­tinct cross-func­tion­al team of med­ical, data sci­ence, and machine learn­ing experts to gain unique insights into dis­ease patho­phys­i­ol­o­gy, iden­ti­fy new tar­gets for poten­tial ther­a­peu­tic inter­ven­tion, and devel­op new med­i­cines faster and at scale.

About OPL-0401
OPL-0401 is a non-selec­tive ROCK (Rho kinase) 1 and 2 inhibitor with unique phys­io­chem­i­cal prop­er­ties that make it suit­able as an oral treat­ment option for patients with vas­cu­lar dis­eases of the eye such as dia­bet­ic retinopa­thy (DR). Pre­clin­i­cal results in an ani­mal study show about a 200-fold high­er expo­sure in reti­na than in plas­ma at steady state fol­low­ing dai­ly oral dos­ing for 7 days. OPL-0401 has pre­vi­ous­ly been found to be gen­er­al­ly safe and well tol­er­at­ed across near­ly 300 patients and healthy vol­un­teers in 12 Phase 1 and 2 stud­ies com­bined (includ­ing those con­duct­ed pri­or to Valo’s acqui­si­tion of the pro­gram). OPL-0401 is an inves­ti­ga­tion­al agent and has not been approved by the U.S. Food and Drug Admin­is­tra­tion (FDA).

About Valo Health

Valo Health, Inc is apply­ing AI to exten­sive human data to unlock new lev­els of speed and scale in ther­a­peu­tic dis­cov­ery and devel­op­ment. The company’s Opal Plat­form™ is an AI-dri­ven drug devel­op­ment engine that inte­grates real human data across every step, enabling the bio­log­i­cal dis­cov­ery, ther­a­peu­tic design, and clin­i­cal devel­op­ment of bet­ter med­i­cines for patients. Found­ed by Flag­ship Pio­neer­ing and head­quar­tered in Lex­ing­ton, MA, Valo also has offices in New York, NY and Tel Aviv, Israel. To learn more, vis­it val​o​health​.com.

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