R 333 / Rigel - LARVOL DELTA

Usefulness of 18F-FDG PET/CT, LDH and tumor markers in the prognosis and evaluation of tumor response after RMB therapy in advanced, metastatic colorectal cancer (mCRC) (ESMO-GI 2017) - P2; "Introduction: RENCA macrobeads (RMBs) are a form of biological-systems based therapy for mCRC [USA FDA BB-IND 10091; NCT01053013]...The R and NR groups were also statistically different with respect to their mean LDH levels at Day 60 (R, 333.88+/-445.89 vs. NR, 1278.50+/- 761.9; p < 0.0001)... The data here demonstrate that LDH levels and PET-CT SUVmax changes, along with CEA and CA19-9 levels were useful in predicting and determining positive therapeutic response to RMBs in late-stage mCRC. Increasing LDH levels as a poor prognostic indicator have been reported to be useful by others, for example see Br J Cancer 116(3),318-323.2017. The correlation of PET-CT and tumor marker data with LDH levels offer further support for the clinical anti-tumor effects of the RMBs."

HEOR • Colorectal Cancer • Gastrointestinal Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor

"Guys, this rōcks! Seriously." (@darioT_)

RMB (RENCA Macrobead) therapy in advanced mCRC: Phase IIb preliminary multi-site survival findings; correlation & combination with Phase I and IIa data including imaging and lab profiles [U.S.FDA BB-IND 10091] (ESMO-GI 2018) - "...The P I/IIA trials differed from IIb in that none of their patients had been treated with regorafenib or trifluridine/tipiracil.; Mean survival for 84/89 patients (all three trials) was 41 weeks [median survival=33 weeks; SD +/-37.5 wks]...For the combined PI/IIa data, two groups of patients: R (n=25) and NR (n=9) were defined by their LDH values at days 30 and 60 after first implantation (D30, mean R value 305.92+/-284.76 vs. NR, 649.33+/- 363.60; p<0.0070), D60 (R, 333.88+/-445.89 vs. NR, 1278.50+/- 761.9; p<0.0001)... Taken together, the PI/IIa/IIb trial data are encouraging in indicating improved survival of late-stage mCRC patients with RMB treatment. The laboratory data (LDH levels, CEA and CA19-9) decreases, as well as PET-CT imaging (decreased SUVmax) support this. The data to date merit development of a Phase III randomized trial of RMB vs."

P2b data • Colorectal Cancer • Renal Cell Carcinoma

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Review

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HEOR

Generation of an induced pluripotent stem cell line from a patient with surfactant metabolism dysfunction carrying ABCA3 mutations. (PubMed, Stem Cell Res) - "In this study, we established an induced pluripotent stem cell line (SMCPGHi001-A) from the peripheral blood cells of a 49-day-old male infant, carrying compound heterozygous variations of the ABCA3 gene (c.3997_3998del, p.R1333fs, and c.3137C > T, p.A1046V). This iPSC line would be a useful tool to study the pathogenesis, disease development, and treatment of pulmonary surfactant dysfunction."

Journal • Preclinical • Metabolic Disorders • Respiratory Diseases

[VIRTUAL] Association between serum prolactin as well as sex hormone levels and sexual dysfunction (EAU-I 2020) - "It was also significantly correlated with EF subscores (r=-333, p<0.001) (Figure1a)... Our study shows that patients with serum prolactin levels of higher than 12.3 ng/ml are more prone to ED. Also, TT levels are positively correlated with sexual desire."

Erectile Dysfunction • Sexual Disorders

[VIRTUAL] The Utility of Automated Vocalisation Indices Collected in a Clinical Setting for Preschool Children on the Autism Spectrum. (INSAR 2020) - "Moderate positive correlations were found between average RCTs and age equivalence scores in receptive, and relationship subscales of the VABS (r=.348, p=.012; r=.333, p=.016), with a moderate negative association with language scores on the ADOS (rs=-.395, p=.004). The findings provide preliminary evidence that novel LENA AVIs collected during brief individual play-based interactions with a clinician may be a valid and useful measure of childrens naturalistic language abilities that may not be captured by standardised assessments such as the MSEL. Additional data on a larger cohort of children will be available by INSAR 2020."

Clinical • Autism Spectrum Disorder • Genetic Disorders

Safety and efficacy of topical R333 in patients with discoid lupus erythematosus (DLE) and systemic lupus erythematosus (SLE) lesions (SKINDLE) (clinicaltrials.gov) - P2, N=54; Not yet recruiting -> Recruiting

Enrollment open • Immunology • Lupus

Rigel to focus on ITP, DLE and dry eye (Rigel Press Release) - P2, N=54; Sponsor: Rigel; SKINDLE (NCT01597050); P2, N=210; Sponsor: Rigel; DROPS (NCT01900249); "Rigel's other two lead programs, R333, a topical dermatological JAK/SYK inhibitor for discoid lupus erythematosus, and R348, a topical ophthalmic JAK/SYK inhibitor for dry eye, are presently in Phase 2 studies, with results expected in Q4 2013 and Q2 2014, respectively. Rigel expects to advance one of these two molecules into a Phase 3 clinical program by 2014/15."

Anticipated new P3 trial • Anticipated P2 data • Immunology • Lupus

Q2 2012 Results (Rigel) - Anticipated initiation of P2 trial for discoid lupus this month

Anticipated new P2 trial • Lupus

Rigel initiates phase 2 clinical studies with R343 for asthma and R333 for discoid lupus (Rigel) - P2, N=54; NCT01597050; Rigel announced commencement of P2 clinical studies with R333 for discoid lupus erythematosus; Primary endpoints will be measurement of each person's decrease in total combined Erythema & Scaling Score of all treated lesions from baseline to Day 28; Rigel anticipates completion of the study in 2013

Trial completion date • Trial initiation date • Immunology • Lupus

Rigel provides pipeline update (Rigel Press Release) - "Rigel announced that R333, which was being evaluated as a potential therapeutic for active skin lesions in patients with discoid lupus erythematosus (DLE), did not meet the primary endpoint in a recently completed Phase 2 clinical study. The primary endpoint was the proportion of patients who achieved at least a 50% decrease from baseline in the total combined Erythema and Scaling score of all treated lesions at Week 4. R333 was shown to be relatively safe and well tolerated. In light of these overall findings, Rigel has decided not to pursue this indication further with R333."

Discontinued • Pipeline update • Immunology • Lupus

Real time polymerase chain reaction versus conventional culture method to detect respiratory pathogens in subjects with non-cystic fibrosis bronchiectasis. (ERS 2019) - "Forty patients were enrolled.Sixty percent were males, the median age was 10 years.For PCR findings, 79% had a positive result and 21% had normal flora, this is in contrast to CCM which yielded 66% positive results and 34% normal flora.This indicates that PCR yielded more pathogens and the McNemar's test for symmetry was significant(p=0.035), with PCR detecting more positive results when CCM detects normal flora than the reverse.The median turn-around time for PCR was significantly shorter than culture (McNemar's test p<0.001, 2 vs 4 days).The actual cost of PCR is higher than CCM(R1333 vs R247), however faster turn-around times are generally associated with reduced hospital stay and reduction in overall cost."

Polymerase Chain Reaction