Sig­ilon Ther­a­peu­tics said it de­creased ex­ter­nal spend on a rare lyso­so­mal stor­age dis­or­der as the Bob Langer-found­ed biotech fo­cus­es its cell ther­a­py pipeline on type 1 di­a­betes.

The an­nounce­ment comes af­ter the biotech ter­mi­nat­ed the po­si­tion of its chief med­ical of­fi­cer, May Or­fali, last month. Or­fali re­mains as a con­sul­tant to Sig­ilon. In in­ter­views af­ter pub­li­ca­tion, she and CEO Rogério Vi­val­di said the ter­mi­na­tion was due to the port­fo­lio pri­or­i­ti­za­tion.

Flag­ship-found­ed Sig­ilon said it plans to ask the FDA for hu­man study clear­ance in 2024 for the Eli Lil­ly-part­nered pro­gram, with a re­vised de­vel­op­ment strat­e­gy that keeps the lights on in­to 2025. The Cam­bridge, MA biotech pre­vi­ous­ly ex­pect­ed to make it in­to 2024 af­ter lay­ing off 38% of its work­force in De­cem­ber 2021 to fo­cus on mu­copolysac­cha­ri­do­sis type 1, or MPS-1, and di­a­betes. Cash, equiv­a­lents and mar­ketable se­cu­ri­ties stood at $69.6 mil­lion at the end of 2022, at which point the biotech had 62 full-time em­ploy­ees.

The off-the-shelf cell ther­a­py mak­er had a Phase I/II set up to test SIG-005 in MPS-1, but the com­pa­ny nev­er dosed a pa­tient be­cause of is­sues that oc­curred in a sep­a­rate study of an­oth­er can­di­date in he­mo­phil­ia A. The biotech said it op­ti­mized the “shield­ed liv­ing ther­a­peu­tics” plat­form and last No­vem­ber had aimed for IND-en­abling stud­ies of the MPS-1 pro­gram in the sec­ond half of 2023.

No such time­line now ex­ists, per a quar­ter­ly and an­nu­al up­date af­ter the mar­kets closed Tues­day. The biotech said it was still work­ing on “en­gi­neer­ing tech­niques and oth­er cell line strate­gies” to lim­it or avoid pa­tients’ po­ten­tial im­mune re­spons­es to the can­di­dates and en­hance the lyso­so­mal dis­or­der can­di­dates’ abil­i­ty to cross the blood-brain-bar­ri­er.

“Hav­ing lived with di­a­betes for most of my life as well as be­ing a treat­ing physi­cian, I am tru­ly ex­cit­ed about the di­rec­tion of our pro­gram, in­clud­ing our ear­ly pre­clin­i­cal ef­fi­ca­cy and dura­bil­i­ty da­ta,” Sig­ilon pres­i­dent and CEO Vi­val­di said in a state­ment.

Oth­er com­pa­nies work­ing on di­a­betes cell ther­a­pies in­clude Vi­a­Cyte, which linked up with CRISPR Ther­a­peu­tics in 2018 and then was bought by Ver­tex last year. Ver­tex said it re­ceived clear­ance for a sec­ond-gen ap­proach in re­cent weeks.

Orig­i­nal­ly sup­port­ed by Flag­ship Pi­o­neer­ing, Sig­ilon went pub­lic in De­cem­ber 2020 but has seen its share price $SGTX crater to the pen­ny stock range since the De­cem­ber 2021 re­hash­ing. Pri­or to the quar­ter­ly up­date Tues­day, the stock rose near­ly 65%.

Ed­i­tor’s note: This sto­ry was up­dat­ed to cor­rect the sta­tus of May Or­fali’s work with the com­pa­ny and the na­ture of the ter­mi­na­tion of her po­si­tion of chief med­ical of­fi­cer. 

The EMA released updated recommendations today for the use of JAK inhibitors (JAKi) after reviewing data from several clinical trials that showed increased incidents of issues in certain patients who have rheumatoid arthritis and other risk factors.

The EMA noted malignancy, major adverse cardiovascular events (MACE), serious infections, venous thromboembolism (VTE) and mortality in some patients.

Dallas-based biotech Nanoscope Therapeutics unveiled Phase II results on its gene therapy for a rare eye disease Thursday morning.

In the RESTORE trial, 18 patients with retinitis pigmentosa got a gene therapy called MCO-010 while nine got placebo. On a vision test called the MLYMT, the treatment group had a one-point greater change over one year in their score compared to the placebo group, the primary endpoint of the study. However, the 95% confidence interval was 0.0 to 3.0, meaning the result was not statistically significant. The p-value was not provided.

A Massachusetts federal judge on Wednesday dismissed a class action lawsuit filed by investors against Biogen and several of its current and former executives over the company’s failed Alzheimer’s drug, Aduhelm (aducanumab).

The investors argued that Biogen’s contact with the FDA was unlawful and that the company made 25 false and misleading statements, including statements about the rollout and price of the drug.

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.