APOLLO: A Randomized, Double-Blind, Placebo-Controlled Study of Bitopertin to Evaluate the Efficacy, Safety, and Tolerability in Participants With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older. The main questions it aims to answer are:
* Whether bitopertin increases pain-free sunlight exposure after 6 months of treatment in participants with EPP or XLP.
* How PPIX concentration levels change from before bitopertin treatment to after 6 months of treatment.
Researchers will compare bitopertin to a placebo look-alike substance that contains no drug.
Participants will complete daily questionnaires and attend study visits for assessments.

Start Date04 Apr 2025

Sponsor / Collaborator

Disc Medicine, Inc.

NCT05883748

/ Enrolling by invitationPhase 2/3

An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants with Erythropoietic Protoporphyria (EPP)

This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.

Start Date31 Aug 2023

Sponsor / Collaborator

Disc Medicine, Inc.

NCT05828108

/ RecruitingPhase 1/2IIT

A Phase I/II, Intra-Patient Dose-Escalation Study of the Selective GlyT1 Inhibitor, Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia.

Background:
Diamond-Blackfan anemia (DBA) is an inherited disease that affects the bone marrow. People with DBA have chronic anemia that can be severe. Many must have frequent transfusions of red blood cells. Current treatments for DBA all have risks of serious side effects. Better treatments are needed.
Objective:
To test a new drug (bitopertin) in people with DBA.
Eligibility:
People aged 18 or older with DBA.
Design:
Participants will be screened. They will have a physical exam; they will have blood tests and a test of their heart function. They will have a bone marrow biopsy: An area of their hip will be numbed, and a needle will be inserted to remove a sample of tissue from inside the bone.
Bitopertin is a pill taken by mouth. Participants will take the drug once a day every day for 8 months. They will start with a low dose of the drug; the dosage may increase gradually over time. They will keep a diary to record each dose.
Participants will have blood tests every 4 weeks. This may be done in the clinic. Participants may also have telehealth visits; they can have blood drawn at a local lab and sent to the researchers.
The bone marrow biopsy and other tests will be repeated after 8 months.
Participants who have a positive response to bitopertin will be invited to enter an extended phase of the trial. They may continue to take the drug for 3 more years.
Those who choose not to continue in the extended phase may have a follow-up visit 6 months after they stop taking the drug.

Start Date25 Jul 2023

Sponsor / Collaborator

National Heart, Lung & Blood Institute

100 Clinical Results associated with Bitopertin

100 Translational Medicine associated with Bitopertin

100 Patents (Medical) associated with Bitopertin

Literatures (Medical) associated with Bitopertin

01 Apr 2025·EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY

Novel pharmaceutical treatment approaches for schizophrenia: a systematic literature review

Review

Author: Khdour, Adam ; Awadallah, Heba ; Khdour, Maher ; Jarab, Anan ; Al-Qerem, Walid ; Mimi, Yousef

PURPOSE:

Schizophrenia is a chronic and debilitating neuropsychiatric disorder affecting approximately 1% of the global population. Traditional antipsychotic treatments, while effective for positive symptoms, often have significant side effects and fail to address cognitive and negative symptoms. Novel pharmacological treatments targeting muscarinic receptors, TAAR1 agonists, serotonergic pathways, and glutamate modulation have emerged as promising alternatives.

AIM:

This systematic literature review aims to critically evaluate the efficacy, safety, and mechanisms of action of novel pharmacological agents in the treatment of schizophrenia.

METHODS:

A comprehensive search was conducted across PubMed, Embase, Cochrane Library, Scopus, and Web of Science for randomized controlled trials (RCTs) and clinical trials published between April 2014 and March 2024. Studies evaluating novel treatments targeting muscarinic receptors, TAAR1 agonists, serotonergic agents, and glutamate modulation were included. Primary outcomes focused on symptom reduction and quality of life, while secondary outcomes included cognitive function and adverse events. The Joanna Briggs Institute (JBI) tool was used for quality assessment.

RESULTS:

Eleven studies involving 4614 participants (mean age 37-43 years, predominantly male) were included. Drugs evaluated included xanomeline-trospium (KarXT), pimavanserin, ulotaront, emraclidine, and bitopertin. Significant improvements in PANSS and CGI-S scores were observed, with xanomeline-trospium showing a mean reduction of 17.4 points (p < 0.001). Adverse events were mostly mild and transient, with nausea, constipation, and somnolence being common.

CONCLUSION:

Novel treatments for schizophrenia show promise in managing both positive and negative symptoms, with generally favorable safety profiles. Future studies should focus on large-scale, long-term trials to refine their efficacy, safety, and clinical applicability.

01 Jan 2025·BIOLOGICAL PSYCHIATRY

Finding the Right Dose: NMDA Receptor–Modulating Treatments for Cognitive and Plasticity Deficits in Schizophrenia and the Role of Pharmacodynamic Target Engagement

Review

Author: Sehatpour, Pejman ; Kantrowitz, Joshua T. ; Kantrowitz, Joshua T

Cognitive impairment associated with schizophrenia (CIAS) and related deficits in learning (plasticity) are among the leading causes of disability in schizophrenia. Despite this, there are no Food and Drug Administration-approved treatments for CIAS, and the development of treatments has been limited by numerous phase 2/3 failures of compounds that showed initial promise in small-scale studies. NMDA-type glutamate receptors (NMDARs) have been proposed to play an important role in schizophrenia; moreover, the NMDAR has a well-characterized role in cognition, learning, and neuroplasticity. We review previously published clinical trials in CIAS that focused on NMDAR modulator treatments, focusing on published and recent developments of the use of novel NMDAR-modulating treatments for CIAS both alone and combined with plasticity/learning paradigms to enhance learning. We use this discussion of previous studies to highlight the importance of incorporating pharmacodynamic target engagement biomarkers early in treatment development, which can help predict which compounds will succeed or fail in phase 3. A range of direct and indirect NMDAR modulators are covered, including D-serine, D-cycloserine, memantine, and glycine and first-generation glycine transport inhibitors (e.g., sarcosine and bitopertin), as well as recent positive studies of iclepertin, a novel glycine transport inhibitor, and luvadaxistat, a D-amino acid oxidase inhibitor that increases brain D-serine levels, and indirect noninvasive brain stimulation NMDAR-modulating treatments. Several examples of successful use of pharmacodynamic target engagement biomarkers for dose/drug discovery are emphasized, including the mismatch negativity, auditory steady state, and time-frequency event-related potential approaches.

01 Oct 2024·BIOLOGICAL PSYCHIATRY

Novel Ideas for Drug Discovery in the Treatment of People With Schizophrenia

Article

Author: Chengappa, K N Roy ; Shekhar, Anantha

The dominant focus on dopamine D2 receptor antagonists as the main target of drug development led to market approvals of several antipsychotic drugs that generally improved the pos. symptoms of schizophrenia but had little impact on the rate-limiting steps of functional recovery, i.e., neg. symptoms and cognitive impairment.An excitation/ inhibition imbalance in the cortex is opined to be brought about by lower GABAergic activity, which leads to disinhibition of glutamatergic neurons.In turn, disinhibited glutamatergic neuronal projections to the midbrain activate dopaminergic projections to the striatum, leading to excessive striatal dopamine release and inducing psychoses and other psychopathologies noted in schizophrenia.Therefore, inhibitors of GlyT1 (glycine transporter 1) or DAAO (D-amino acid oxidase) were developed to potentially improve neg. and cognitive symptoms.After initial excitement, bitopertin, a GlyT1 inhibitor, has not shown any benefit as an adjunctive treatment for suboptimally responsive pos. or neg. symptoms in large, controlled trials.Iclepertin, a selective GlyT1 inhibitor, showed pos. results in a phase 2 study for cognitive impairments in schizophrenia, and large multinational trials are underway to confirm the initial findings he mechanistic shift away from dopaminergic D2 receptor antagonists to a broader circuit-level dysfunction in schizophrenia involving GABAergic-glutamatergic projections has opened the door to newer therapeutic opportunities with improved safety profiles and that target symptoms more allied to recovery, i.e., neg. symptoms and cognitive impairments.

News (Medical) associated with Bitopertin

27 Feb 2025

·ir.discmedicine.com

Disc Medicine Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Update

Targeting NDA submission for bitopertin in erythropoietic protoporphyria (EPP) in H2 2025 through accelerated approval pathway; on track to initiate APOLLO post-marketing confirmatory trial by mid-2025 Positive update from Phase 1b trial of DISC-0974 in patients with anemia of myelofibrosis (MF) presented at ASH 2024; initial data from ongoing Phase 2 expected in H2 2025 Positive data from initial cohorts of ongoing Phase 1b study of DISC-0974 in patients with anemia of non-dialysis-dependent chronic kidney disease (NDD-CKD) presented at ASN Kidney Week 2024; initial data from multiple dose portion of this study expected in H2 2025 Presented first-in-human SAD/MAD data from Phase 1 trial of DISC-3405 demonstrating proof-of-mechanism; a Phase 2 study in polycythemia vera (PV) expected to initiate in H1 2025 Strong financial position ending 2024 with $490 million in cash, cash equivalents, and marketable securities, further strengthened by the net proceeds of our public offering in January 2025, expected to fund operations into 2028 WATERTOWN, Mass., Feb. 27, 2025 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today reported financial results for the fourth quarter and full year ended December 31, 2024, and provided a recap of recent program and corporate developments. “2024 was a transformative year for Disc, marked by the achievement of several milestones, most notably opening the door to a potential accelerated approval for bitopertin based on Phase 2 results linking PPIX reduction to improvement on clinical outcomes in EPP patients, and subsequently gaining alignment on the design of the APOLLO trial as a confirmatory study,” said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. “These achievements bring us meaningfully closer to delivering a potentially disease-modifying and life-altering treatment to patients. We expect to initiate the APOLLO trial in mid-2025 followed by an NDA submission in the second half of this year and are diligently preparing for the opportunity to bring bitopertin to market. In addition, we saw momentum across our portfolio, presenting positive clinical data readouts for each of our clinical programs. This included exciting data updates for DISC-0974 in anemias of MF and NDD-CKD, both serious conditions with high unmet patient need and significant opportunity for better treatment options. These latest clinical results strengthen our belief in the potential of our iron homeostasis portfolio assets to address a range of hematological diseases.” Recent Highlights and Anticipated Milestones: Bitopertin: GlyTI Inhibitor (Heme Synthesis Modulator) Presentation of full results from Phase 2 AURORA and BEACON studies at 2024 ASH Annual Meeting demonstrating significant reductions in PPIX are associated with substantial improvements in time spent in sunlight, measures of quality of life, and reduction in phototoxic reactions Positive end-of-phase 2 meeting with FDA providing a path toward potential accelerated approval for bitopertin in EPP with protoporphyrin IX (PPIX) reduction as a surrogate endpoint Positive Type C meeting with FDA to achieve regulatory alignment on APOLLO post-marketing confirmatory trial design; on track to initiate trial by mid-year 2025 Planning to submit NDA under accelerated approval pathway in H2 2025 based on existing clinical data, including results from BEACON and AURORA Phase 2 trials DISC-0974: Anti-Hemojuvelin Antibody (Hepcidin Suppression) Presented positive data from the Phase 1b/2 study of DISC-0974 in anemia of myelofibrosis (MF), demonstrating robust and broad hematologic activity across patient segments Results showed substantial and durable improvements in hemoglobin, reductions in transfusion burden, and improvements in fatigue scores Initiated the Phase 2 portion of the study in December 2024, enrolling a broad range of patients after positive discussions with FDA Presented data from initial cohorts of ongoing Phase 1b study of DISC-0974 in patients with anemia of NDD-CKD, demonstrating hematologic activity following a single dose Presented preclinical data at ASH 2024 demonstrating the potential of DISC-0974 to treat anemia of chronic inflammatory diseases such as IBD The Company expects initial data from the ongoing Phase 2 MF anemia trial and multiple-dose data from the ongoing Phase 1b NDD-CKD trial in H2 2025 Results showed substantial and durable improvements in hemoglobin, reductions in transfusion burden, and improvements in fatigue scores Initiated the Phase 2 portion of the study in December 2024, enrolling a broad range of patients after positive discussions with FDA DISC-3405: Anti-TMPRSS6 Antibody (Hepcidin Induction) Presented complete data from the Phase 1 SAD/MAD trial in healthy volunteers, demonstrating proof-of-mechanism with substantial, dose-dependent increases in hepcidin and reductions in serum iron supportive of a once-monthly dosing regimen Presented positive preclinical data in sickle cell disease highlighting the potential for DISC-3405 to provide therapeutic benefit in SCD by restricting iron The Company plans to initiate a Phase 2 study in PV in H1 2025 Corporate: In January 2025, the Company completed an underwritten public offering of 4,533,182 shares of its common stock at $55.00 per share and 181,818 pre-funded warrants at $54.9999 per pre-funded warrant. The offering included 615,000 shares which were issued upon the exercise in full by the underwriters of their option to purchase additional shares of common stock. The gross proceeds to the Company from the offering were approximately $259 million, before deducting underwriting discounts, commissions and offering expenses. In the fourth quarter of 2024, the Company closed a $200 million non-dilutive debt financing with Hercules Capital. An initial $30 million was drawn at closing. An additional $80 million is available to be drawn through the second half of 2026 at the Company's discretion. An additional $65 million is available subject to the Company’s achievement of certain performance milestones. The final $25 million is available subject to Hercules’ consent during the interest-only period, which lasts for a minimum of 48 months from closing. The gross proceeds to the Company from the offering were approximately $259 million, before deducting underwriting discounts, commissions and offering expenses. Full Year 2024 Financial Results: Cash Position: Cash, cash equivalents, and marketable securities were $489.9 million as of December 31, 2024, compared to $360.4 million as of December 31, 2023. The increase was largely due to net proceeds of $172.5 million and $27.6 million from the follow-on offering in June 2024 and the debt financing in November 2024, respectively. We expect that our existing cash, cash equivalents, and marketable securities as of December 31, 2024, together with the estimated net proceeds of $243.3 million from our underwritten public offering completed in January 2025, will be sufficient to fund operational plans into 2028. Research and Development Expenses: R&D expenses were $96.7 million for the full year ended December 31, 2024, as compared to $69.3 million for the full year ended December 31, 2023. The increase in R&D expenses was primarily driven by the progression of Disc’s portfolio, including bitopertin’s clinical studies and drug manufacturing, advancement of DISC-0974 and DISC-3405 programs deeper into development, and increased headcount. Selling, General and Administrative Expenses: SG&A expenses were $33.0 million for the full year ended December 31, 2024, as compared to $21.9 million for the full year ended December 31, 2023. The increase in SG&A expenses was primarily due to increased headcount including establishing infrastructure to support potential commercialization. Net Loss: Net loss was $109.4 million for the full year ended December 31, 2024, as compared to $76.4 million for the full year ended December 31, 2023. The increase was primarily due to higher operating costs in the current period to support the continued advancement of our pipeline. About Disc Medicine Disc Medicine (NASDAQ:IRON) is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit www.discmedicine.com. Available Information Disc announces material information to the public about the Company, its products and services, and other matters through a variety of means, including filings with the U.S. Securities and Exchange Commission (SEC), press releases, public conference calls, webcasts and the investor relations section of the Company website at ir.discmedicine.com in order to achieve broad, non-exclusionary distribution of information to the public and for complying with its disclosure obligations under Regulation FD. Disc Cautionary Statement Regarding Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding: expectations with respect to the next stages of its development programs for bitopertin, DISC-0974 and DISC-3405, including projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data, and other clinical activities; the registrational pathway for bitopertin, including the potential for accelerated approval; the potential of its development programs in new indications; and the strength of its financial position and its anticipated cash runway. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” “opportunity,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the adequacy of Disc’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc’s product candidates; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc’s planned preclinical studies and clinical trials; the timing of the availability of data from Disc’s clinical trials; Disc’s ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; and the other risks and uncertainties described in Disc’s filings with the SEC, including in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2024. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. Media Contact Peg Rusconi Deerfield Group peg.rusconi@deerfieldgroup.com Investor Relations Contact Christina Tartaglia Precision AQ Christina.tartaglia@precisionaq.com

Clinical ResultPhase 1Financial StatementPhase 2

23 Jan 2025

·www.pharmaceutical-technology.com

Disc raises $225.5m as it eyes approval of rare skin disorder drug

EPP is a rare genetic disorder that causes severe burn injuries on the skin following exposure to visible light. Credit: Cavan Images via Getty Images. Disc Medicine has raised $225.5m through an upsized public offering to support the company’s pipeline, including its lead investigational drug, bitopertin. The therapy targets erythropoietic protoporphyria (EPP), a rare genetic disorder that causes extreme light sensitivity. Disc is preparing to file a new drug application (NDA) for the therapy this year, seeking accelerated approval from the US Food and Drug Administration (FDA). The drug candidate is designed to reduce levels of protoporphyrin IX (PPIX), a phototoxic compound that accumulates in the blood of EPP and X-linked protoporphyria (XLP) patients, causing severe pain and burns from even brief sun exposure. Earlier this week, Disc announced feedback from a Type C meeting with the FDA, where the agency finalised its agreement with the design of the upcoming Phase III APOLLO trial, which will evaluate the safety and efficacy of bitopertin. The meeting resulted in the addition of a co-primary endpoint to the trial, which will now assess both changes in PPIX levels and the average time patients can spend in sunlight without pain at the end of six months of treatment. These endpoints are designed to demonstrate bitopertin’s ability to improve a patient’s quality of life by reducing sensitivity to sunlight. This follows an earlier end-of-Phase II meeting with the FDA in which Disc secured support for an accelerated approval pathway based on existing clinical data. At that meeting, the regulator agreed that PPIX reduction could serve as a surrogate endpoint for efficacy. The agency also approved key elements of the Phase III trial design, including its randomised, double-blind, placebo-controlled structure, a six-month treatment duration, and a primary endpoint focused on pain-free sunlight exposure during the final month of treatment. Disc’s prior Phase II study showed that bitopertin was well-tolerated at 20mg and 60mg daily doses over 24 weeks in 22 patients, with no serious adverse events reported. Patients who opted to continue into an open-label extension study for another 24 weeks demonstrated further positive outcomes. These data supported Disc’s decision to pursue accelerated approval and informed the design of the Phase III APOLLO trial, which is set to begin in mid-2025. The APOLLO study will enrol patients aged 12 years and older with EPP or XLP and will include additional secondary endpoints such as cumulative time in sunlight without pain, reductions in phototoxic reactions, and patient-reported global impressions of change. If accelerated approval is granted, data from APOLLO would serve as a confirmatory trial to convert the conditional approval into a full approval. Bitopertin could become the second FDA-approved treatment for EPP, following Clinuvel Pharmaceuticals ’ Scenesse (afamelanotide). Scenesse increases skin pigmentation and protects against light-induced skin damage, allowing patients to spend an additional 20 minutes in sunlight daily without pain as demonstrated in a pivotal trial (NCT01605136). A patient with EPP highlighted the ethical challenges in EPP trials at the Outsourcing in Clinical Trials (OCT) DACH 2024 conference, in Zurich, Switzerland, between 29 and 30 October. EPP trials have to reckon with the potential for phototoxic burns in participants randomised to control groups, and the risk of a bias in patient selection, as more severely affected patients may hesitate to participate. However, this risk is met with the drive for new treatments and the unmet need for EPP patients.

Phase 3Phase 2

22 Jan 2025

·endpts.com

Shanghai Bao, Sichuan Biokin file IPOs in Hong Kong; Disc's $200M offering

Plus, news about Rivus and Crown Labs: Two Chinese biotechs file for stock listings in Hong Kong: Shanghai Bao Pharmaceuticals and Sichuan Biokin Pharmaceutical join a queue of Chinese drug developers looking to float their shares in Hong Kong. Bao, which was founded in 2019, has five clinical-stage drugs in development and seven preclinical assets. Bao’s pipeline includes candidates for bacterial infections, solid tumors, autoimmune diseases and other areas. Biokin, meanwhile, was founded in 1996 as Baili Pharmaceutical; it has multiple programs across generics, traditional Chinese medicine, and ADCs. — Kyle LaHucik Disc Medicine’s $200M offering: The company is raising the funding to support plans to commercialize bitopertin for patients with erythropoietic protoporphyria and X-linked protoporphyria. It previously said it planned to seek accelerated approval. — Jaimy Lee Rivus Pharmaceuticals adds $57M to Series B: The cardiometabolic biotech reeled in the additional capital this month, a spokesperson told Endpoints News . The biotech originally disclosed a $132 million Series B in 2022. CEO Jayson Dallas told Endpoints in September that the company planned to raise more money in 2024. Rivus’ lead candidate, dubbed HU6, has been in three Phase 2 studies, and the latest trial, in MASH, is expected to read out in the second quarter of this year. Rivus was considering an IPO in October, people familiar with the matter told Bloomberg News . — Kyle LaHucik Crown Laboratories raises bid for Revance Therapeutics: After facing competition from Teoxane, Crown has now offered to buy Revance for $3.65 per share — 55 cents more than its most recent offer and 5 cents more than Teoxane’s competing bid. Revance, which produces a Botox competitor called Daxxify, said Tuesday that Crown’s proposal is “the only fully financed offer currently available.” The offer expires early next month. — Nicole DeFeudis

IPODrug ApprovalPhase 2Acquisition

100 Deals associated with Bitopertin

External Link

KEGG	Wiki	ATC	Drug Bank
D10186	Bitopertin	-	DB12426

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Protoporphyria, Erythropoietic, X-Linked Dominant	Phase 3	United States	Disc Medicine, Inc.	04 Apr 2025
Protoporphyria, Erythropoietic	Phase 3	Australia	Disc Medicine, Inc.	31 Aug 2023
Paranoid Schizophrenia	Phase 3	United States	Hoffmann-La Roche, Inc.	11 Dec 2010
Paranoid Schizophrenia	Phase 3	Argentina	Hoffmann-La Roche, Inc.	11 Dec 2010
Paranoid Schizophrenia	Phase 3	Australia	Hoffmann-La Roche, Inc.	11 Dec 2010
Paranoid Schizophrenia	Phase 3	Colombia	Hoffmann-La Roche, Inc.	11 Dec 2010
Paranoid Schizophrenia	Phase 3	Finland	Hoffmann-La Roche, Inc.	11 Dec 2010
Paranoid Schizophrenia	Phase 3	France	Hoffmann-La Roche, Inc.	11 Dec 2010
Paranoid Schizophrenia	Phase 3	Hungary	Hoffmann-La Roche, Inc.	11 Dec 2010
Paranoid Schizophrenia	Phase 3	India	Hoffmann-La Roche, Inc.	11 Dec 2010

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05308472 (AURORA, ASH2024) Manual	Phase 2	Protoporphyria, Erythropoietic	75	Bitopertin 20mg	rqzdoowatg(dvzeyaakbj) = nearly all treatment-emergent adverse events were mild to moderate in intensity thmrwxzmis (brpmagvxrx ) View more	Positive	07 Dec 2024
				Bitopertin 60mg
ACTRN12622000799752 (BEACON, ASH2024) Manual	Phase 2	Protoporphyria, Erythropoietic	22	Bitopertin 20 mg	aofftnmgkc(jbxskojmjk) = oxuxcoxqxa ejgzvuhrfy (tozqyktayb, 7.1) View more	Positive	07 Dec 2024
				Bitopertin 60 mg	aofftnmgkc(jbxskojmjk) = wvpgiwmjtt ejgzvuhrfy (tozqyktayb, 7.0) View more
ACTRN12622000799752 (BEACON, EHA2024) Manual	Phase 2	Protoporphyria, Erythropoietic PPIX	22	Bitopertin 20 mg	gvlwbztmiz(moleytlevu) = fvmguwcekm mphbfjrksj (doylvhsxpu ) View more	Positive	14 May 2024
NCT05308472 (AURORA, GlobeNewswire) Manual	Phase 2	Protoporphyria, Erythropoietic	75	bitopertin 20 mg	kwrulzoojw(zgjnrtcvxn) = xflxgcltyb lbpvyrxqkh (fyhsovvxae ) Met View more	Positive	01 Apr 2024
				bitopertin 60 mg	kwrulzoojw(zgjnrtcvxn) = kybaivvtht lbpvyrxqkh (fyhsovvxae ) Met View more
ACTRN12622000799752 (Beacon, ASH2023) Manual	Phase 2	Protoporphyria, Erythropoietic	17	Bitopertin	msriotitca(olgdtplrjr) = khwqityaek kclhfjjxlw (rfwjdabxry, 17) View more	Positive	11 Dec 2023
NCT01116830 (Pubmed \| J Clin Psychopharmacol) Manual	Phase 1	Schizophrenia	29	bitopertin	zleuuvvemt(kivkwhiwsc) = lrdnumjwer eovilobjdz (yjcvkkxjnj, 7.4) View more	Negative	01 Aug 2017
				Placebo	zleuuvvemt(kivkwhiwsc) = tyhrydnjro eovilobjdz (yjcvkkxjnj, 11.5) View more
NCT01613040 (Pubmed \| Clin Ther)	Phase 3	-	169	Bitopertin 30 mg	lfxrvemhpg(mtunajfgtu) = ziiffenysf uzedkrhcvl (jqohhusxxe )	-	01 Oct 2012
				Bitopertin 175 mg	lfxrvemhpg(mtunajfgtu) = tyhkjmfbhi uzedkrhcvl (jqohhusxxe )

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis