A Randomized, Double-blind, Placebo-controlled Study of the Safety, Tolerability, and Pharmacokinetics of Single and Multiple Ascending Oral Doses of CFTX-1554 in Healthy Subjects, With Comparison of Intake of CFTX-1554 as Liquid Formulation and as Capsule, and After a High-fat Breakfast Versus Fasted

The study will consist of 2 parts, i.e. a single ascending dose part with integrated food effect assessment and assessment of relative bioavailability (Part A), and a multiple ascending dose part (Part B).
Part A will have a randomized, double-blind, placebo-controlled design. Subjects will receive single ascending doses of CFTX-1554 or placebo (as liquid formulation under fasted condition) in 7 subsequent cohorts. Drug intake under fed conditions, and as capsule under fasted conditions and under fed conditions (Periods 2 to 4), compared to liquid formulation under fasted conditions (Period 1) (1 single dose level only) will be assessed.
Part B will have a randomized, double-blind, placebo-controlled design, assessing multiple ascending oral doses of CFTX-1554 or placebo in 4 subsequent cohorts.

Start Date09 Feb 2022

Sponsor / Collaborator

Confo Therapeutics NVStartup

NL-OMON51471 / CompletedNot Applicable

A randomized, double-blind, placebo-controlled study of the safety, tolerability, and pharmacokinetics of single and multiple ascending oral doses of CFTX-1554 in healthy subjects, with comparison of intake of CFTX-1554 as liquid formulation and as capsule, and after a high-fat breakfast versus fasted. - SAD / MAD/ FE HV

Start Date09 Feb 2022

Sponsor / Collaborator

Confo Therapeutics NVStartup

100 Clinical Results associated with CFTX-1554

100 Translational Medicine associated with CFTX-1554

100 Patents (Medical) associated with CFTX-1554

Literatures (Medical) associated with CFTX-1554

01 Sep 2014·EndocrinologyQ2 · MEDICINE

Angiotensin II Type 2 Receptor Stimulation Improves Fatty Acid Ovarian Uptake and Hyperandrogenemia in an Obese Rat Model of Polycystic Ovary Syndrome

Q2 · MEDICINE

Article

Author: Vine, Donna F. ; Noll, Christophe ; Hallberg, Anders ; Gallo-Payet, Nicole ; Carpentier, André C. ; Battista, Marie-Claude ; Baillargeon, Jean-Patrice ; Leblanc, Samuel

Abstract:

Polycystic ovary syndrome (PCOS) is mainly defined by hyperandrogenism but is also characterized by insulin resistance (IR). Studies showed that overexposure of nonadipose tissues to nonesterified fatty acids (NEFA) may explain both IR and hyperandrogenism. Recent studies indicate that treatment with an angiotensin II type 2 receptor (AT2R)-selective agonist improves diet-induced IR. We thus hypothesized that PCOS hyperandrogenism is triggered by ovarian NEFA overexposure and is improved after treatment with an AT2R agonist. Experiments were conducted in 12-week-old female JCR:LA-cp/cp rats, which are characterized by visceral obesity, IR, hyperandrogenism, and polycystic ovaries. Control JCR:LA +/? rats have a normal phenotype. Rats were treated for 8 days with saline or the selective AT2R agonist C21/M24 and then assessed for: 1) fasting testosterone, NEFA, and insulin levels; and 2) an iv 14(R,S)-[18F]fluoro-6-thia-heptadecanoic acid test to determine NEFA ovarian tissue uptake (Km). Compared with controls, saline-treated PCOS/cp rats displayed higher insulin (100 vs 5.6 μU/mL), testosterone (0.12 vs 0.04 nmol/L), NEFA (0.98 vs 0.48 mmol/L), and Km (20.7 vs 12.9 nmol/g·min) (all P < .0001). In PCOS/cp rats, C21/M24 did not significantly improve insulin or NEFA but normalized testosterone (P = .004) and Km (P = .009), which were strongly correlated together in all PCOS/cp rats (ρ = 0.74, P = .009). In conclusion, in an obese PCOS rat model, ovarian NEFA uptake and testosterone levels are strongly associated and are both significantly reduced after short-term C21/M24 therapy. These findings provide new information on the role of NEFA in PCOS hyperandrogenemia and suggest a potential role for AT2R agonists in the treatment of PCOS.

01 Jun 2014·NeuropharmacologyQ2 · MEDICINE

The angiotensin type 2 receptor agonist Compound 21 elicits cerebroprotection in endothelin-1 induced ischemic stroke

Q2 · MEDICINE

Article

Author: Regenhardt, Robert W ; Unger, Thomas ; Bennion, Douglas M ; Katovich, Michael J ; Pioquinto, David J ; Steckelings, U Muscha ; Joseph, Jason P ; Desland, Fiona ; Mecca, Adam P ; Patel, Neal A ; Sumners, Colin ; Rodríguez, Vermali

Evidence indicates that angiotensin II type 2 receptors (AT2R) exert cerebroprotective actions during stroke. A selective non-peptide AT2R agonist, Compound 21 (C21), has been shown to exert beneficial effects in models of cardiac and renal disease, as well as hemorrhagic stroke. Here, we hypothesize that C21 may exert beneficial effects against cerebral damage and neurological deficits produced by ischemic stroke. We determined the effects of central and peripheral administration of C21 on the cerebral damage and neurological deficits in rats elicited by endothelin-1 induced middle cerebral artery occlusion (MCAO), a model of cerebral ischemia. Rats infused centrally (intracerebroventricular) with C21 before endothelin-1 induced MCAO exhibited significant reductions in cerebral infarct size and the neurological deficits produced by cerebral ischemia. Similar cerebroprotection was obtained in rats injected systemically (intraperitoneal) with C21 either before or after endothelin-1 induced MCAO. The protective effects of C21 were reversed by central administration of an AT2R inhibitor, PD123319. While C21 did not alter cerebral blood flow at the doses used here, peripheral post-stroke administration of this agent significantly attenuated the MCAO-induced increases in inducible nitric oxide synthase, chemokine (C-C) motif ligand 2 and C-C chemokine receptor type 2 mRNAs in the cerebral cortex, indicating that the cerebroprotective action is associated with an anti-inflammatory effect. These results strengthen the view that AT2R agonists may have potential therapeutic value in ischemic stroke, and provide the first evidence of cerebroprotection induced by systemic post stroke administration of a selective AT2R agonist.

CURRENT ISSUES IN MOLECULAR BIOLOGY

Stimulation of Angiotensin II Receptor Subtype 2 Reduces Preeclampsia-like Symptoms in a Mouse Model of Preeclampsia

Article

Author: Matsubara, Keiichi ; Sugiyama, Takashi ; Uchikura, Yuka ; Matsubara, Yuko

Angiotensin II (AngII) receptor subtype 1 (AT1R) is involved in the pathogenesis of preeclampsia (PE). Angiotensin II receptor subtype 2 (AT2R) can antagonize the effects of AT1R, but its effects during pregnancy are not known. We investigated the effect of AT2R on the pathogenesis of PE using a mouse model and recently developed AT2R agonist (compound 21 [C21]). Blastocysts collected from pregnant imprinting control region (ICR) mice were incubated with adenovirus containing the CD40L gene and transferred into the uterine horns of pseudo-pregnant ICR mice to express PE-like features. Osmotic pumps were placed subcutaneously on the dorsal side with C21 or saline. C21 reduced the plasma soluble fms-like tyrosine kinase 1 (sFlt-1) concentration, ameliorating hypertension. The splenic T and B cell profiles in model mice were analyzed by flow cytometry. The gated percentage of IFN-γ-positive Th cells was significantly increased and the percentage of plasma cells in B cells was significantly decreased; however, the percentages were not altered by C21. sFlt-1 and soluble endoglin concentrations in plasma were measured with an enzyme-linked immunosorbent assay, and sFlt-1 was reduced. C21 could become a candidate PE drug as it ameliorated the pathophysiology of PE as a result of decreased production of sFlt-1.

News (Medical) associated with CFTX-1554

29 Aug 2023

·www.biospace.com

Confo Therapeutics Appoints Dieter Weinand, Former CEO of Bayer Pharmaceuticals, as Independent Chairman

GHENT, Belgium--(BUSINESS WIRE)-- Confo Therapeutics, a leader in the discovery of medicines targeting G-protein coupled receptors (GPCRs), today announced the appointment of Dieter Weinand as Independent Chairman of its Board of Directors. Mr. Weinand brings with him a wealth of experience with more than 35 years in the pharmaceutical industry and over 25 years as the leader of geographic and strategic business units and drug commercialization efforts across global organizations, including several years as the President, CEO and Chairman of the Board of Bayer Pharmaceuticals AG. He joins Confo as the company advances its pipeline and continues to leverage its proprietary GPCR drug discovery engine. Mr. Weinand succeeds John Berriman, who is retiring from his current position as Chairman of Confo's Board of Directors. “Dieter is an exceptional leader in the pharmaceutical industry with a proven track record across the spectrum of research and development to commercialization, bringing impactful medications to patients. A chairman of his caliber will be invaluable as we continue progressing as an organization with a truly innovative GPCR-targeting technology and a growing pipeline of novel drug candidates,” said Cedric Ververken, CEO of Confo Therapeutics. “On behalf of the Confo team, I sincerely thank John Berriman for his guidance over the past seven years and his contributions to Confo’s growth into a clinical-stage drug development company.” “Confo is moving on an exciting trajectory, harnessing the momentum of a pivotal agreement with Eli Lilly for its first clinical program, CFTX-1554, and building out a robust pipeline of large and small molecules based on its unique platform,” added Dieter Weinand, Independent Chairman. “I look forward to working together with the Confo team to advance the company through its next stages of growth and to achieve its future strategic business and pipeline objectives.” Mr. Weinand has had a highly successful career across all areas of pharmaceutical development, commercialization, and international organizational leadership. Most recently, he was Head of Global Primary Care Business at Sanofi where he led the optimization of the company’s portfolios. Before this, he was President, CEO, and Chairman of the Board of Bayer Pharmaceuticals AG, where he was responsible for integrating the R&D, manufacturing, and all commercial and support functions of the healthcare business of the Bayer AG corporation. Mr. Weinand held various senior management positions across international corporations including at Bristol-Meyers Squibb and Pfizer. He has been instrumental in the launch and marketing of some of the industry’s pivotal products including Lipitor®, Neurontin®, Abilify® and Cipro®. Mr. Weinand holds a B.A. in Biology from Concordia College, New York, and an M.S. in Pharmacology and Toxicology from Long Island University, New York. Mr. Weinand is Chairman of the Board of Nasdaq-listed Replimune (REPL), FORE Biotherapeutics, and ZielBio, among others. About Confo Therapeutics Confo Therapeutics is the only GPCR company with a proprietary discovery engine that precisely targets desired GPCR conformations. This unique capability allows us to unlock a vast untapped potential for the discovery and development of breakthrough medicines. We are advancing a robust pipeline of large and small molecules focused on validated targets in endocrine and metabolic diseases, as well as addressing a broader array of critical unmet medical needs in collaboration with our partners. Our team of accomplished experts is dedicated to advancing our patent protected technology, expanding our capabilities, and building out our pipeline in order to achieve the best possible therapeutic outcomes for patients. For more information, visit View source version on businesswire.com: Contacts Confo Therapeutics Dr. Cedric Ververken, CEO +32 (0) 9 396 74 00 Email: info@confotherapeutics.com Trophic Communications Valeria Fisher or Desmond James + 49 (0) 175 8041816 or +49 (0) 1516 7859086 Email: confo@trophic.eu Source: Confo Therapeutics View this news release online at:

Executive Change

30 Mar 2023

·www.fiercebiotech.com

Daiichi finds line of attack on once-undruggable CNS target by penning $182M Confo collaboration

A Daiichi Sankyo facility Confo Therapeutics’ business development team is on a roll. Weeks after licensing its lead candidate to Eli Lilly, the Belgian biotech has landed a 168 million euro ($182 million) deal with Daiichi Sankyo to discover small molecule agonists of a central nervous system (CNS) target. Daiichi has committed the cash, in an undisclosed mix of upfront and milestone payments, to get Confo to apply its platform to “a previously undruggable G-protein coupled receptor (GPCR) target in an area of high unmet medical need.” The project is expected to generate a series of small-molecule compounds that Daiichi will have an exclusive option to license globally and take into the clinic. Confo, part of the Fierce 15 class of 2019, landed the deal on the strength of technology for stabilizing GPCRs. The membrane receptors are flexible proteins that function through conformational changes to transmit a signal inside the cell. The shape shifting makes GPCRs hard to isolate and find hits for using biophysical methods. While a significant minority of drugs on the market target GPCRs, most of the membrane proteins are yet to be drugged. The upshot is that the industry is yet to develop drugs against targets that are thought or known to play a role in diseases with major unmet needs. Brute force can help overcome the challenges, with companies sometimes able to find hits by exposing GPCRs to large compound libraries during high-throughput screening, but Confo has tried to develop a more reliable method that is less prone to delivering false positives. Using VHH fragments, the technology that turned Ablynx into a 3.9 billion euro biotech, Confo stabilizes GPCRs in their active state. Once the proteins are stabilized, Confo uses biophysical, competition and biosensor-type screening assays to generate hits. The model allows the biotech to find hits using a library of small-molecule fragments that is far smaller than those used in alternative approaches to GPCRs. Attracted by the platform, Regeneron signed up to collaborate with Confo in 2021. The Belgian biotech took its lead candidate, neuropathic pain prospect CFTX-1554, into the clinic the following year. At the start of March, Lilly struck a deal to license CFTX-1554 and associated backup compounds for $40 million upfront and up to $590 million in milestones. Confo last publicly disclosed a financing round in 2019.

License out/inPROTACs

06 Mar 2023

·www.pharmaceutical-technology.com

US FDA approves expanded indication for Lilly’s breast cancer therapy

Lilly’s Verzenio has been approved to treat HR+, HER2-, node-positive, high risk early breast cancer. Credit: National Cancer Institute / Unsplash. The US Food and Drug Administration (FDA) has granted approval for the expanded indication of Eli Lilly and Company ’s (Lilly) Verzenio (abemaciclib) to treat breast cancer patients. Verzenio along with endocrine therapy (ET) has received approval as an adjuvant treatment for hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-), node-positive, early breast cancer (EBC) adult patients who are at a high recurrence risk. Patients at high risk can be identified solely based on tumour size, nodal status, and tumour grade to receive treatment with Verzenio. The regulatory approval was supported by the data obtained from the randomised, open-label, two-cohort, multicentre, global Phase III monarchE clinical trial, which was conducted in 5,637 HR+, HER2-, node-positive EBC adult patients who are at high risk of recurrence. The findings demonstrated a deepened benefit in invasive disease-free survival (IDFS) beyond the treatment for two years with adjuvant Verzenio. In the trial, Verzenio along with ET reduced disease recurrence risk by 35% compared to ET alone. Loxo@Lilly CEO Jacob Van Naarden said: “This expanded approval will allow us to bring Verzenio to many more women and men with HR+, HER2-, high-risk early breast cancer in the curative setting – before patients experience recurrence, potentially to incurable metastatic disease. “The initial adjuvant approval for Verzenio changed the treatment paradigm, and the strength of the monarchE results supporting this approval underscores the role this differentiated CDK4/6 inhibitor can play in reducing the risk of recurrence in early breast cancer.” According to Lilly, over 300,000 people are anticipated to be diagnosed with breast cancer this year in the US. Last week, the company and Confo Therapeutics signed a global licence deal for CFTX-1554, the latter’s clinical-stage peripheral pain candidate.

Phase 3Drug ApprovalLicense out/inClinical Result

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Indication	Highest Phase	Country/Location	Organization	Date
Neuralgia	Phase 1	NL	Confo Therapeutics NVStartup	10 Mar 2022

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