A Phase 2B, Multicenter, 30-week, Prospective, Cross-over, Double-blind, Randomized, Placebo-controlled Study Followed by a 52-Week Open-label Extension Study to Evaluate the Efficacy and Safety of Basimglurant Adjunctive to Ongoing Anticonvulsive Therapy in Children, Adolescents, and Young Adults with Seizures Associated with Tuberous Sclerosis Complex

Start Date04 May 2023

Sponsor / Collaborator

Noema Pharma AG

[+1]

NCT05059327

/ Active, not recruitingPhase 2

A Phase 2B, Multicenter, 30-week, Prospective, Cross-over, Double-blind, Randomized, Placebo-controlled Study Followed by a 52-Week Open-label Extension Study to Evaluate the Efficacy and Safety of Basimglurant Adjunctive to Ongoing Anticonvulsive Therapy in Children, Adolescents, and Young Adults With Uncontrolled Seizures Associated With Tuberous Sclerosis Complex

The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).

Start Date03 Mar 2022

Sponsor / Collaborator

Noema Pharma AG

NCT05217628

/ RecruitingPhase 2/3

A Phase II/III, Multicentre, 8-week run-in Phase Followed by a 12-week, Prospective, Parallel-group, Double-blind, Randomized Withdrawal, Placebo-controlled Study, With a 52 Week Open Label Extension, to Evaluate the Efficacy and Safety of Daily 1.5 to 3.5 mg Basimglurant in Patients With Pain Associated With Trigeminal Neuralgia With Suboptimal Response to Their Current Anti-pain Therapy.

Trigeminal neuralgia (TN), also called "tic douloureux", is the most common form of craniofacial neuropathic pain and is considered the cause of one of the most painful afflictions known in medical practice.
This study is designed to evaluate the efficacy and safety of 1.5mg - 3.5mg basimglurant in adults with TN.

Start Date11 Jan 2022

Sponsor / Collaborator

Noema Pharma AG

100 Clinical Results associated with Basimglurant Sulfate

100 Translational Medicine associated with Basimglurant Sulfate

100 Patents (Medical) associated with Basimglurant Sulfate

Literatures (Medical) associated with Basimglurant Sulfate

11 Feb 2024·Expert Opinion on Pharmacotherapy

Pharmacological management of fragile X syndrome: a systematic review and narrative summary of the current evidence

Review

Author: Moon, Seungyoun ; Barwell, Julian ; Burrows, Lisa ; Tromans, Samuel ; Shankar, Rohit ; Watkins, Lance V.

INTRODUCTIONFragile X syndrome (FXS) is the most common inherited cause of Intellectual Disability. There is a broad phenotype that includes deficits in cognition and behavioral changes, alongside physical characteristics. Phenotype depends upon the level of mutation in the FMR1 (fragile X messenger ribonucleoprotein 1) gene. The molecular understanding of the impact of the FMR1 gene mutation provides an opportunity to target treatment not only at symptoms but also on a molecular level.METHODSWe conducted a systematic review to provide an up-to-date narrative summary of the current evidence for pharmacological treatment in FXS. The review was restricted to randomized, blinded, placebo-controlled trials.RESULTSThe outcomes from these studies are discussed and the level of evidence assessed against validated criteria. The initial search identified 2377 articles, of which 16 were included in the final analysis.CONCLUSIONBased on this review to date there is limited data to support any specific pharmacological treatments, although the data for cannabinoids are encouraging in those with FXS and in future developments in gene therapy may provide the answer to the search for precision medicine. Treatment must be person-centered and consider the combination of medical, genetic, cognitive, and emotional challenges.

01 Sep 2022·Pharmacology Biochemistry and Behavior

Clinical investigations of compounds targeting metabotropic glutamate receptors

Review

Author: Smith, Jodi L ; Witkin, Jeffrey M ; Pandey, Kamal P

Pharmacological modulation of glutamate has long been considered to be of immense therapeutic utility. The metabotropic glutamate receptors (mGluRs) are potential targets for safely altering glutamate-driven excitation. Data support the potential therapeutic use of mGluR modulators in the treatment of anxiety, depression, schizophrenia, and other psychiatric disorders, pain, epilepsy, as well as neurodegenerative and neurodevelopmental disorders. For each of the three mGluR groups, compounds have been constructed that produce either potentiation or functional blockade. PET ligands for mGlu5Rs have been studied in a range of patient populations and several mGlu5R antagonists have been tested for potential efficacy in patients including mavoglurant, diploglurant, basimglurant, GET 73, and ADX10059. Efficacy with mGlu5R antagonists has been reported in trials with patients with gastroesophageal reflux disease; data from patients with Parkinson's disease or Fragile X syndrome have not been as robust as hoped. Fenobam was approved for use as an anxiolytic prior to its recognition as an mGlu5R antagonist. mGlu2/3R agonists (pomaglumated methionil) and mGlu2R agonists (JNJ-40411813, AZD 8529, and LY2979165) have been studied in patients with schizophrenia with promising but mixed results. Antagonists of mGlu2/3Rs (decoglurant and TS-161) have been studied in depression where TS-161 has advanced into a planned Phase 2 study in treatment-resistant depression. The Group III mGluRs are the least developed of the mGluR receptor targets. The mGlu4R potentiator, foliglurax, did not meet its primary endpoint in patients with Parkinson's disease. Ongoing efforts to develop mGluR-targeted compounds continue to promise these glutamate modulators as medicines for psychiatric and neurological disorders.

01 Aug 2022·Drug Metabolism and Disposition

Application of Empirical Scalars To Enable Early Prediction of Human Hepatic Clearance Using In Vitro-In Vivo Extrapolation in Drug Discovery: An Evaluation of 173 Drugs

Article

Author: Yan, Zhengyin ; Brown, Suzanne ; Liu, Ning ; Chang, Jae H ; Broccatelli, Fabio ; Kenny, Jane R ; Jones, Robert S ; Leung, Christian

The utilization of in vitro data to predict drug pharmacokinetics (PK) in vivo has been a consistent practice in early drug discovery for decades. However, its success is hampered by mispredictions attributed to uncharacterized biological phenomena/experimental artifacts. Predicted drug clearance (CL) from experimental data (i.e. hepatocyte intrinsic clearance: CL_int, fraction unbound in plasma: f_u,p) is often systematically underpredicted using the well-stirred model (WSM). The objective of this study was to evaluate using empirical scalars in the WSM to correct for CL mispredictions. Drugs (N=28) were used to generate numerical scalars on CL_int (α), and f_u,p (β) to minimize the error (AAFE) for CL predictions. These scalars were validated using an additional dataset (N=28 drugs) and applied to a non-redundant AstraZeneca (AZ) dataset available in the literature (N=117 drugs) for a total of 173 compounds. CL predictions using the WSM were improved for most compounds using an α value of 3.66 (~64%<2-fold) compared to no scaling (~46%<2-fold). Similarly, using a β value of 0.55 or combination of α and β scalars (values of 1.74 and 0.66, respectively) resulted in a similar improvement in predictions (~64%<2-fold and ~65%<2-fold, respectively). For highly bound compounds (f_u,p{less than or equal to}0.01), AAFE was substantially reduced across all scaling methods. Using the β scalar alone or a combination of α and β appeared optimal; and produce larger magnitude corrections for highly-bound compounds. Some drugs are still disproportionally mispredicted, however the improvements in prediction error and simplicity of applying these scalars suggests its utility for early-stage CL predictions. Significance Statement In early drug discovery, prediction of human clearance using in vitro experimental data plays an essential role in triaging compounds prior to in vivo studies. These predictions have been systematically underestimated. Here we introduce empirical scalars calibrated on the extent of plasma protein binding that appear to improve clearance prediction across multiple datasets. This approach can be used in early phases of drug discovery prior to the availability of pre-clinical data for early quantitative predictions of human clearance.

News (Medical) associated with Basimglurant Sulfate

11 Dec 2024

·endpts.com

BenevolentAI restructures; Cimeio, Kyowa ink cell therapy deal

Plus, news about NewAmsterdam, Rocket, ImmunityBio, CG Oncology, Spruce Biosciences, Pharming, Veradermics, Noema Pharma, Ambrosia Biosciences, EsoBiotec, Cellectar Biosciences and Hepion: BenevolentAI’s overhaul: The struggling AI biotech said it plans to lay off staff, consider delisting from the Euronext Amsterdam stock exchange, partner its early-stage assets and undergo other additional organizational shifts as co-founder Ken Mulvany returns . Its Amsterdam-listed stock was down 10% on Wednesday. — Kyle LaHucik Cimeio Therapeutics, Kyowa Kirin make a deal: The Japanese drugmaker is paying up to $300 million as part of an agreement to develop new cell therapies. Additional financial terms were not disclosed. — Jaimy Lee At least three public offerings have been disclosed over the past day, including NewAmsterdam ’s bid to raise $300 million; Rocket ’s upsized $165 million offering ; ImmunityBio ’s $100 million financing ; and CG Oncology ’s offering , which has yet to be priced. — Jaimy Lee Spruce’s Phase 2b fail for endocrine disorder drug: The South San Francisco biotech said tildacerfont administered once daily did not meet the primary endpoint of glucocorticoid reduction in a mid-stage study of people with adult congenital adrenal hyperplasia. But the trial’s principal investigator said the data suggest twice daily dosing may be more effective. Its stock $SPRB was down about 25% on Wednesday morning. — Ayisha Sharma Pharming’s rare disease med passes Phase 3 test in kids: The Dutch drugmaker’s Joenja for activated phosphoinositide 3-kinase delta syndrome (APDS) met the primary endpoints in a late-stage trial of patients aged four to 11 years old. Joenja won FDA approval for APDS patients aged 12 and older in March 2023. Pharming plans to submit global regulatory filings in children starting in 2025. — Ayisha Sharma Veradermics’ $75M Series B: The Connecticut-based dermatology and aesthetics biotech raised the financing as it begins a Phase 2/3 study in hair loss. — Kyle LaHucik Noema adds $35M from EQT Life Sciences: A second close of a Series B first announced in March of last year brings the total for the round to CHF 130 million, which is about $147 million. The cash will fund Noema’s four ongoing Phase 2 trials: mGluR5 negative allosteric modulator basimglurant in trigeminal neuralgia and seizures in tuberous sclerosis complex; gemlapodect, a PDE10a inhibitor, in Tourette syndrome; and NOE-115, a monoamine modulator, in menopause symptoms. — Elizabeth Cairns Merck backs Ambrosia: The New Jersey pharma giant invested in the biotech’s Series A, which now totals $25 million. Ambrosia previously announced plans to take over a former Pfizer site in Boulder as it builds out an oral small-molecule pipeline for obesity and metabolic disorders. — Kyle LaHucik EsoBiotec scores €22M: The Belgian cell therapy specialist raised the equivalent of $23 million from backers that include Thuja Capital, UCB Ventures and Invivo Partners. The company’s immune-shielded lentiviral vector, which is called ESO-T01, is designed to reprogram T lymphocytes into BCMA CAR-T cells in vivo . It will enter an investigator-initiated trial in multiple myeloma in China, its first in humans, and data are expected in the second half of 2025, which could allow expansion to indications including autoimmune diseases. — Elizabeth Cairns Cellectar lays off 60% of its workers: The biotech is prioritizing its pipeline and letting go of staff. Cellectar had 20 employees at the end of 2023. Its stock price $CLRB sank 62% in pre-market trading on Wednesday. — Kyle LaHucik Hepion Pharmaceuticals ends merger plans with Pharma Two B. — Jaimy Lee

Cell TherapyPhase 3Phase 2Drug ApprovalClinical Result

11 Dec 2024

·www.prnewswire.com

EQT Life Sciences Leads Series B Extension in Noema Pharma, Raising Total Series B Financing to CHF 130 million

Proceeds will be used to enable key late-stage clinical trial readouts for assets across several underserved neurological diseases with high unmet medical need. EQT Life Sciences has led the extension, and joins a consortium of prominent global biotechnology investors. The investment builds on EQT Life Sciences' previous successful partnership with neurology-focused repeat entrepreneur and Noema Pharma CEO Ilise Lombardo, MD STOCKHOLM, Dec. 11, 2024 /PRNewswire/ -- EQT Life Sciences is pleased to announce that the LSP 7 fund has invested in Noema Pharma ("the Company"). The clinical-stage biotech company headquartered in Basel, Switzerland is developing a pipeline of first-in-disease therapeutics that address central nervous system (CNS) disorders. The Series B extension brings the total capital raised in the round to CHF 130 million, including prior investments from Forbion, Jeito Capital, Sofinnova Partners, Gilde Healthcare, Polaris Partners, Invus and UPMC Enterprises. CNS disorders are a significant area of unmet medical need, affecting hundreds of millions of people worldwide who often face inadequate treatment options with limited effectiveness and significant side effects. Many of the conditions targeted by Noema Pharma have historically been difficult to treat due to their complex nature and the lack of effective therapies. By leveraging its diverse portfolio of neurological assets and innovative clinical pipeline, Noema Pharma is strongly positioned to address these debilitating disorders and provide much-needed hope and transformative solutions to patients living with these life-altering challenges. The Company's portfolio includes four active Phase 2 trials, with key readouts from all studies anticipated in 2025. Proceeds from the financing will drive the continued development of Noema Pharma's clinical-stage assets, including its lead candidate basimglurant (NOE-101), a mGluR5 inhibitor currently in Phase 2 trials for severe pain in trigeminal neuralgia (TN) and seizures in tuberous sclerosis complex (TSC). Additionally, the Company is advancing gemlapodect (NOE-105), a PDE10a inhibitor in Phase 2 for Tourette syndrome and childhood onset fluency disorder (COFD or stuttering), as well as NOE-115, a broad-spectrum monoamine modulator in a Phase 2 trial for vasomotor symptoms and additional symptoms of menopause. Ilise Lombardo, MD, CEO of Noema Pharma, stated: "We are thrilled to welcome EQT Life Sciences as a lead investor and to have Felice join our Board. Their support and expertise will be invaluable as we progress our clinical programs and strive to make a meaningful impact on patients' lives." Felice Verduyn-van Weegen, Partner at EQT who is joining the Company's Board of Directors, commented: "Noema Pharma's innovative approach to CNS disorders aligns very well with our investment strategy and we are excited to support their late-stage clinical pipeline and transformative therapies. Having worked with Ilise Lombardo on a previous successful investment, her exceptional leadership as a repeat entrepreneur further reinforces our confidence in Noema's potential to deliver meaningful impact to patients in need." Contact EQT Press Office, [email protected] This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2

11 Dec 2024

·www.businesswire.com

Noema Pharma extends Series B financing round, closing at CHF 130 Million ($147 Million)

BASEL, Switzerland--( BUSINESS WIRE )-- Noema Pharma , a clinical-stage biotech company targeting debilitating central nervous system (CNS) disorders, announced the successful close of a Series B extension financing round with an investment from EQT Life Sciences, bringing the total capital raised in the round to CHF 130 million (approx. USD 147 million). With its investment in Noema Pharma, EQT Life Sciences joins the syndicate of previous Series B investors including Forbion, Jeito Capital, Sofinnova Partners, Gilde Healthcare, Polaris Partners, Invus and UPMC Enterprises. The new financing will support Noema Pharma’s four active Phase 2 trials, with key data readouts anticipated in 2025. This includes additional development activities for basimglurant (NOE-101), an mGluR5 negative allosteric modulator currently in Phase 2 trials for severe pain in trigeminal neuralgia (TN) and seizures in tuberous sclerosis complex (TSC); gemlapodect (NOE-105), a PDE10a inhibitor in a Phase 2b trial for Tourette syndrome and under development for childhood-onset fluency disorder (COFD or stuttering); and NOE-115, a broad-spectrum monoamine modulator in a Phase 2 trial for vasomotor symptoms and other symptoms of menopause. Ilise Lombardo, MD, CEO of Noema Pharma, stated: " This latest financing underscores the strong support and confidence from investors in Noema’s vision to treat neuroscience-based conditions. We are thrilled to welcome EQT Life Sciences into this strong syndicate of investors and have Felice join our Board. EQT’s support and expertise alongside our syndicate of investors will be invaluable as we progress our clinical programs and strive to make a meaningful impact on patients' lives." Felice Verduyn-van Weegen , Partner at EQT and member of the Board at Noema, commented: " Noema Pharma's innovative approach to CNS disorders aligns very well with our investment strategy and we are excited to support their late-stage clinical pipeline and transformative therapies. Having worked with Ilise Lombardo on a previous successful investment, her exceptional leadership as a repeat entrepreneur reinforces our confidence in Noema's potential to deliver meaningful impact to patients in need.” The Series B extension follows the initial Series B financing of CHF 103 million (USD 112 million) announced on March 2023, and a CHF 54 million (approx. USD 60 million) Series A round in December 2020. Noema Pharma was founded in 2019 with seed investment from Sofinnova Partners. Notes to Editors About Noema Pharma Noema Pharma is a clinical-stage biotech company advancing a portfolio of transformative, first-in-disease therapeutics targeting neuroscience-based conditions with high unmet need. Noema has four programs currently in active Phase 2 clinical trials evaluating seizures in Tuberous Sclerosis Complex, pain in Trigeminal Neuralgia, Tourette syndrome and vasomotor symptoms plus CNS-mediated symptoms of menopause with readouts expected in 2025. Noema was founded by leading venture capital firm Sofinnova Partners and is supported by current investors including Forbion, Gilde Healthcare, Invus, Jeito Capital, Polaris Partners and UPMC Enterprises. Learn more at www.noemapharma.com . About EQT Life Sciences EQT Life Sciences was formed in 2022 following the integration of LSP, a leading European life sciences venture capital firm, into the EQT platform. As LSP, the firm raised over EUR 3.0 billion and supported the growth of more than 150 companies since it started to invest over 30 years ago. With a dedicated team of highly experienced investment professionals coming from backgrounds in medicine, science, business, and finance, EQT Life Sciences backs entrepreneurs who have ideas that could truly make a difference for patients. The team combines deep sector knowledge, analytical skills, and investment experience to provide the added value that entrepreneurs seek. For more information, go to eqtgroup.com/private-capital/life-sciences/

Phase 2

100 Deals associated with Basimglurant Sulfate

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KEGG	Wiki	ATC	Drug Bank
-	Basimglurant Sulfate	-	DB11833

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Pain	Phase 3	Denmark	Noema Pharma AG	11 Jan 2022
Pain	Phase 3	Spain	Noema Pharma AG	11 Jan 2022
Trigeminal Neuralgia	Phase 3	Germany	Noema Pharma AG	11 Jan 2022
Trigeminal Neuralgia	Phase 3	Denmark	Noema Pharma AG	11 Jan 2022
Trigeminal Neuralgia	Phase 3	United Kingdom	Noema Pharma AG	11 Jan 2022
Trigeminal Neuralgia	Phase 3	United States	Noema Pharma AG	11 Jan 2022
Trigeminal Neuralgia	Phase 3	Italy	Noema Pharma AG	11 Jan 2022
Trigeminal Neuralgia	Phase 3	Poland	Noema Pharma AG	11 Jan 2022
Trigeminal Neuralgia	Phase 3	Turkey	Noema Pharma AG	11 Jan 2022
Trigeminal Neuralgia	Phase 3	Spain	Noema Pharma AG	11 Jan 2022

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