SP-101 / Sumitomo Pharma - LARVOL DELTA

Spirovant Sciences Doses First Patient in the SAAVe Phase 1/2 Clinical Trial of Its Investigational, Aerosol-Delivered Genetic Medicine for the Treatment of Cystic Fibrosis (PRNewswire) - "The SAAVe Phase 1/2 clinical trial is a multicenter, open-label, dose escalation and dose expansion trial of SP-101 + Augmenter in patients with cystic fibrosis who are not eligible for CFTR modulator therapy or for whom the CFTR modulators are insufficient. The primary endpoint of the study is safety and tolerability."

Trial status • Cystic Fibrosis

Intratracheal administration of AAV2.5T-SP183-fCFTRΔR in combination with doxorubicin is able to correct the mucociliary clearance defect in cystic fibrosis model ferrets (NACFC 2024) - "These findings indicate that mRNA levels are a good predictor of potential functional benefit and that AAV2.5T-based vectors, such as SP-101, are likely to require doxorubicin to realize a functional benefit in people with CF."

Combination therapy • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Respiratory Diseases

SP-101, a novel adeno-associated virus gene therapy for the treatment of cystic fibrosis, mediates functional correction of primary human airway epithelia from donors with cystic fibrosis. (PubMed, Hum Gene Ther) - "Tropism studies using a fluorescent reporter vector and single-cell mRNA sequencing of SP-101-mediated hCFTRΔR mRNA demonstrated broad expression in all cell types after apical transduction, including secretory, ciliated, and basal cells. In summary, SP-101, particularly in combination with doxorubicin, shows promise for a novel CF treatment strategy, and strongly supports continued development."

Gene therapy • Journal • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Infectious Disease • Pulmonary Disease • Respiratory Diseases

Inhalation of SP-101 followed by inhaled doxorubicin results in robust and durable hCFTRΔR transgene expression in the airways of wild-type and cystic fibrosis ferrets. (PubMed, Hum Gene Ther) - "Only a portion of ferrets exhibited a weak T-cell response to AAV2.5T and no T-cell response was seen against hCFTRΔR. These data strongly support the continued development of inhaled SP-101, followed by inhaled doxorubicin, for the treatment of cystic fibrosis."

Journal • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases • CFTR

SAAVe: A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF) (clinicaltrials.gov) - P1/2 | N=15 | Recruiting | Sponsor: Spirovant Sciences, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

SAAVe: A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF) (clinicaltrials.gov) - P1/2 | N=15 | Not yet recruiting | Sponsor: Spirovant Sciences, Inc.

New P1/2 trial • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Administration of SP-101 and Doxorubicin Results in Robust and Durable hCFTRΔR Transgene Expression in the Airways of Ferrets and Corrects Human CF Airway Epithelia In Vitro (NACFC 2022) - "These data provide a strong rationale for co-development ofinhaled doxorubicin to realize the full potential of SP-101 gene therapy forindividuals with CF who do not benefit from treatment with small-molecule modulators.622 Development of in vitro transcribed messenger ribonucleic acidtherapeutics for cystic fibrosis"

Preclinical • Cystic Fibrosis • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Inhaled Administration of SP-101 Followed by Doxorubicin Leads to Durable hCFTRΔR Transgene Expression in the Airways of Wild-Type and CF Ferrets (ASGCT 2022) - "Similar hCFTRΔR mRNA expression was evident in the respiratory tract of both wild-type and CF ferrets, indicating successful airway transduction regardless of preexisting mucus accumulation in the respiratory system of CF ferrets. In summary, these data strongly support continued development of SP-101 for the treatment of CF."

Cystic Fibrosis • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Infectious Disease • Pulmonary Disease • Respiratory Diseases • CFTR

SP-101, a Novel Adeno-Associated Virus Vector, Functionally Corrects Human Airway Epithelia from Donors with Cystic Fibrosis at Low Multiplicity of Infection in the Presence of Doxorubicin (ASGCT 2022) - "Despite a significant impact on hCFTRΔR mRNA and chloride conductance, doxorubicin did not alter VCN of SP-101 in a dose-dependent manner. In summary, the apical tropism of the AAV2.5T capsid for human airway, combined with the enhanced mRNA expression afforded by doxorubicin, show promise for a novel CF treatment strategy and strongly support continued development."

Cystic Fibrosis • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Infectious Disease • Inflammation • Pulmonary Disease • Respiratory Diseases

"#Spirovant presented ferret studies of SP-101, an inhalation CF #genetherapy, showing it effectively and durably increased #CFTR gene activity. https://t.co/VS6Il1BXPG #cysticfibrosis #CF #livingwithCF #CFpatients #CFtreatment #NACFC2021 #CFTRgeneactivity #genetherapy #Spirovant" (@CFNewsToday)

Clinical • Cystic Fibrosis • Fibrosis • Gene Therapies • Genetic Disorders • Pulmonary Disease • Respiratory Diseases • CFTR

Spirovant to Present SP-101 Data at the 2021 North American Cystic Fibrosis Conference (PRNewswire) - "Spirovant Sciences...announced that it will be delivering oral and poster presentations highlighting pre-clinical data for its compound, SP-101, at the 2021 North American Cystic Fibrosis Conference (NACFC 2021), November 2-5. SP-101."

Preclinical • Cystic Fibrosis

First report of Cnidium officinale as a natural host plant of Apple stem grooving virus in South Korea. (PubMed, Plant Dis) - "Phylogenetic analysis with the Maximum Likelihood method using the MEGA X program (Kumar et al., 2018) showed that ASGV-CO-kr1 grouped with isolates Cuiguan (KR185346), BH (LC480456), and YY (LC480457) based on the CP aa sequences, while it grouped with isolates SG (LC475148) and TL101 (MH108976) based on the MP aa sequences...officinale plants are propagated by root division, so they are susceptible to infection with viruses. The result of this study is important for generating virus-free seedlings to produce C. officinale."

Journal • Infectious Disease • PCR

FDA Grants Spirovant Sciences Orphan Drug and Rare Pediatric Disease Designations for SPIRO-2101 for Treatment of Cystic Fibrosis (PRNewswire) - "Spirovant Sciences...announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease Designations to its lead product candidate, SPIRO-2101, for the treatment of cystic fibrosis."

Orphan drug • Cystic Fibrosis • Genetic Disorders

Talee Bio announces $4.5 million award from the cystic fibrosis foundation to advance development of two gene therapy product candidates (Businesswire) - “Talee Bio…announced agreements with the Cystic Fibrosis Foundation (CF Foundation) to fund the preclinical development of two mutation-agnostic gene therapy product candidates, TL-101 and TL-102.”

Financing

Newly added product (Businesswire) - Preclinical, Cystic Fibrosis

Pipeline update