A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability tofersen and to evaluate the effect of tofersen on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).

Start Date17 May 2021

Sponsor / Collaborator

Biogen, Inc.

NCT03764488 / CompletedPhase 1

A Phase 1, Safety, Tolerability, and Distribution Study of a Microdose of Radiolabeled BIIB067 Co-administered With BIIB067 to Healthy Adults

The primary objective of the study is to evaluate the distribution in the central nervous system (CNS) of a microdose 99mTc-MAG3-BIIB067 co-administered with unlabeled BIIB067 (Tofersen). The secondary objective of the study is to assess the safety and tolerability of unlabeled BIIB067 co-administered with a microdose of 99mTc-MAG3-BIIB067 to healthy participants.

Start Date20 Dec 2018

Sponsor / Collaborator

Biogen, Inc.

NCT03070119 / CompletedPhase 3

An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults With Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation

The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 (tofersen) in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK), pharmacodynamic (PD), biomarker effects, and efficacy of BIIB067 administered to participants with ALS and a confirmed SOD1 mutation.

Start Date08 Mar 2017

Sponsor / Collaborator

Biogen, Inc.

[+1]

100 Clinical Results associated with Tofersen

100 Translational Medicine associated with Tofersen

100 Patents (Medical) associated with Tofersen

Literatures (Medical) associated with Tofersen

12 Dec 2024·BRITISH MEDICAL BULLETIN

New developments in the diagnosis and management of motor neuron disease

Review

Author: Shaw, Pamela J ; O’Brien, David

Abstract:

Introduction:

Motor neuron disease (MND) is a devastating neurodegenerative disease characterized by progressive muscle weakness.

Sources of data:

PubMed, MEDLINE, and Cochrane databases were searched for articles to March 2024. Searches involved the terms ‘motor neuron disease’ or ‘amyotrophic lateral sclerosis’ and ‘epidemiology’, ‘diagnosis’, ‘clinical’, ‘genetic’, ‘management’, ‘treatment’, or ‘trial’.

Areas of agreement:

Evidence-based management involves riluzole, multidisciplinary care, provision of noninvasive ventilation and gastrostomy, and symptomatic treatments. Tofersen should be offered to treat SOD1-MND.

Areas of controversy:

Edaravone and Relyvrio are approved treatments in the USA, but insufficient evidence was found to support approval in the UK and Europe.

Growing points:

The discovery of neurofilaments as MND biomarkers, growth of platform trials and development of novel therapies provide optimism for more powerful neuroprotective therapies.

Areas timely for developing research:

Further work should focus on the elucidation of environmental causes of MND, gene–environment interactions, and advanced cellular models of disease.

01 Dec 2024·Annals of Clinical and Translational Neurology

Rethinking antisense oligonucleotide therapeutics for amyotrophic lateral sclerosis

Review

Author: Ito, Daisuke ; Okada, Kensuke

Abstract:

Antisense oligonucleotides, which are used to silence target genes, are gaining attention as a novel drug discovery modality for proteinopathies. However, while clinical trials for neurodegenerative diseases like amyotrophic lateral sclerosis have been conducted in recent years, the results have not always been favorable. The results from a Phase III trial of the antisense oligonucleotide, that is, tofersen, which targets SOD1 mRNA, showed decreased levels of cerebrospinal fluid SOD1 and plasma neurofilament light chain but no improvements in primary clinical endpoint. Moreover, case reports pertaining to patients with amyotrophic lateral sclerosis carrying FUS and C9orf72 mutations who received antisense oligonucleotide‐based treatments have demonstrated a notable reduction in the targeted protein (thus providing the proof of mechanism) but with no discernible clinical benefits. There are several possible reasons why antisense oligonucleotides knockdown fails to achieve proof of concept, which need to be addressed: on‐target adverse effects resulting from the loss of function of target gene and irreversible neuronal death cascade due to toxic protein accumulation, among other factors. This review provides an overview of the current status and discusses the prospects of antisense oligonucleotides treatment for amyotrophic lateral sclerosis.

01 Dec 2024·JAMA Neurology

Article

Author: Thonhoff, Jason R. ; Appel, Stanley H.

This Viewpoint advocates for the accelerated adoption of tofersen therapy to treat patients with variant SOD1-mediated amyotrophic lateral sclerosis (ALS) and describes the current barriers to treatment.

163

News (Medical) associated with Tofersen

11 Dec 2024

·www.prnewswire.com

Groundbreaking Directive Ensures ALS Patients on Medicare Advantage Gain Access to Qalsody

Plans Required to Cover Treatment for SOD1-ALS ARLINGTON, Va., Dec. 11, 2024 /PRNewswire/ -- The Centers for Medicare & Medicaid Services (CMS) has announced a first-of-its-kind directive requiring Medicare Advantage plans to approve coverage for Qalsody, a treatment for people living with genetic SOD1-ALS. This decision reflects the ALS Association's commitment to ensuring that people living with ALS have access to promising ALS treatments without delay. "This is a victory for the entire ALS community," said Calaneet Balas, president and CEO of the ALS Association. "It's critical that FDA-approved treatments are made accessible rather than being labeled as 'experimental.' We are grateful to everyone in our community who helped make this day happen." Balas added: "We hope this sends a message to the entire rare disease community and pharmaceutical industry that new treatments can be developed and made accessible to those who will benefit from them. We need to ensure more ALS treatments are developed and approved that will help everyone living with ALS." The ALS Association played a key role in the historic CMS directive by participating and advocating for the research, approval, and accessibility of Qalsody for people living with ALS. In 2004, the ALS Association was the first to fund research into ALS-specific antisense oligonucleotide (ASO) technology, investing over $1.3 million in groundbreaking studies that laid the foundation for the development of Qalsody. This early commitment supported preclinical studies, safety trials, and the first-in-human phase 1 trial, which established SOD1 as a viable therapeutic target. In 2023, Qalsody became the first gene-based therapy approved for ALS, thanks in part to the ALS Association's efforts, including a united push from the ALS community for FDA accelerated approval. This new CMS directive provides precedent for state regulators to issue similar actions for other private insurers. According to the Valor study published in 2022, Qalsody, which was developed specifically to target the RNA produced by mutated SOD1 genes, has demonstrated significant promise in clinical trials and real-world studies. In the phase 3 VALOR trial, Qalsody reduced levels of mutated SOD1 proteins in cerebral spinal fluid by 35% within eight weeks and decreased bloodstream levels of neurofilament light chain (NfL)—a key biomarker of neurodegeneration—by 50% within 12-16 weeks. These biological changes translated into measurable clinical benefits, including slower disease progression, improved respiratory function, and enhanced quality of life after 52 weeks of treatment. Real-world data has since validated these findings, with some patients stabilizing or even experiencing improvements in motor function and quality of life. When insurance companies began denying access to Qalsody, labeling it as "experimental," the ALS Association took decisive action, working directly with CMS to launch an investigation into these unjust denials. By presenting evidence of widespread insurance rejections and demonstrating the critical need for immediate intervention. In addition to working with CMS, the ALS Association actively engaged policymakers, held strategic meetings with state and federal legislators, and served as a staunch advocate for patients, ensuring their voices were heard and their rights to life-changing treatment were upheld. The ALS Association urges anyone previously denied Qalsody by their Medicare Advantage plan to contact their ALS specialist immediately to begin the process of securing access to this critical treatment. For more information on the CMS directive visit als.org About the ALS Association The ALS Association is the largest ALS organization in the world. The ALS Association funds global research collaborations, assists people with ALS and their families through its nationwide network of care and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association is working to make ALS a livable disease while urgently searching for new treatments and a cure. For more information about the ALS Association, visit our website at . About ALS Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. Over the course of the disease, people lose the ability to move, to speak, and eventually, to breathe. The disease is always fatal, usually within five years of diagnosis. Few treatment options exist, resulting in a high unmet need for new therapies to address functional deficits and disease progression. SOURCE The ALS Association WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3OligonucleotideClinical ResultDrug Approval

15 Nov 2024

·www.biopharmadive.com

How a Biogen drug set the stage for a new biotech targeting ALS

Trace Neuroscience, a freshly launched startup, is developing a type of genetic medicine it believes could help most, if not all, people with ALS.Its a bold idea, considering nearly every attempt to combat the nerve-destroying disease has ended in failure. Researchers still arent sure what causes 90% of cases. And in the U.S., the short list of drugs approved to treat ALS, or amyotrophic lateral sclerosis, got shorter still this year, as a once-promising medication hit a major setback that ultimately got it pulled from the market.Yet Trace was still able to fundraise $101 million from a group of prominent biotechnology investment firms that includes Third Rock Ventures, Atlas Venture, RA Capital Management and Alphabets venture capital arm, GV. Jeffrey Tong, a Third Rock partner whos now on Traces board of directors, says there are a few reasons why the startup earned his firm's support.One was the companys underlying science. It was built on recent discoveries from the labs of Aaron Gitler, a Stanford University professor; Pietro Fratta, a professor of cellular and molecular neuroscience at University College London; and Michael Ward, a senior investigator in the National Institutes of Healths neurological disorders division. Almost at the same time, these labs identified connections between an important RNA-processing molecule and a protein thats often impaired in people with ALS and other neurodegenerative disorders.Another selling point, according to Tong, was the precedent set by Biogens Qalsody.Like Traces medicine, Qalsody is whats known as an antisense oligonucleotide, meaning it regulates protein production by binding to RNA. Qalsody flunked the main trial designed to show it can slow the functional decline associated with ALS. The drug was still approved last year, though, due to its apparent effects on neurofilament light chain, a protein tied to nerve cell damage.Traces CEO, Eric Green, plans to incorporate those learnings into the development plans for his companys drug, which is on track to enter human testing in early 2026.Green has helped set up multiple Third Rock portfolio companies. He co-founded the precision medicine developer Maze Therapeutics and served as head of translational research at heart drug startup MyoKardia. In an interview with BioPharma Dive, Green explained why his new, big-swing endeavor resonated with investors.The following conversation has been edited and condensed for clarity.BIOPHARMA DIVE: Was there a concerted effort to go to these different parties and ask them to come together and build a single company, rather than three separate ones?ERIC GREEN: Absolutely. This, to me, was just a central part of how I thought about this. I wanted us to join forces and not dilute our efforts across three companies, because everybody here brings different expertise. It's a really nice complementary team.This is my third go around doing this with Third Rock. One of the things I have learned over the years is that the more you can bring together a group of diverse experts who understand an area of biology, the greater chance you're going to have to be successful in translating that into a medicine.We have an academic world that I think is very much oriented toward independent discoveries and independent credit. That makes perfect sense from an academic standpoint, but as we think about trying to make this into a medicine, we need to join forces.There really is that shared goal and shared mission here, recognizing that we're stronger together.Atlas, RA, GV what about those firms made you want them to be part of your investor syndicate?GREEN: The first thing I had in mind was to allow us to raise a sum of money that would allow us to get through what we see as the key clinical data points where we're going to be able to learn about how this medicine is working in people with ALS.But [another piece was] to bring together a diverse set of voices. Atlas, for example, built a number of different RNA therapy and oligonucleotide companies. That was something really important to me, thinking about how we can build companies. [We wanted] a syndicate that has different perspectives, both really early company creation in the case of Third Rock and Atlas but then also people who think about company growth and some of the longer-term horizons. This was where having a group like RA has been really helpful to us. And similarly, with Google Ventures, a group that really sees companies across the life cycle, from very early discovery all the way through to clinical development and commercialization.One of the things that, to me, is a little unusual, maybe particularly exciting, about this opportunity is that we're very early. We're not far from the original discovery. But there's a potential here to move really quickly, and to go from being a discovery company to a company that's generating really meaningful clinical data. And in this indication, meaningful clinical data can have a very direct path towards getting a drug on the market.You raised $101 million. Why stop there, or set that as the goal, in terms of fundraising?GREEN: We want to build an R&D plan that we think is the right one, and then finance that. Not, Lets test the waters and see how much money we could raise. Yes, we could have certainly gotten $1 more than [our goal] and so, by definition, we were oversubscribed. But I have a pet peeve about that myself. Our goal was to set out to raise the amount of money we needed to support the R&D plan, and that's what we did. In general, we're really happy with the round we put together. And we'll have a lot of options with this syndicate to take the next steps.Your company is at the intersection of genetic medicine and neuroscience, two research areas that could be seen as high-risk. What were the biggest concerns among these investors? Do you think youve alleviated them?GREEN: ALS, and neurodegenerative diseases more broadly, are an area where there's been a lot of disappointment.It was really important for me to be able to convey what we thought was different about this opportunity. I can summarize it with three components. Here is this focus on robust human genetics as our guide to give us confidence we're going after a mechanism relevant in people with ALS, rather than in some cell or animal model of the disease. The quality of the human genetics evidence here is exceptionally high, both because it's been reproduced across so many different cohorts, but also because of this relationship with disease progression and survival. And that's unusual, as opposed to having a relationship with just the risk of disease. It really gives us confidence there's a window to intervene on this mechanism after people are diagnosed.But genetics is not enough. You need to understand what the mechanism is by which the gene is working. And this is where that set of discoveries I described from Aaron and Pietro and Michael was so critical of saying we really understand how this gene is working. This has been, even with some of the previous genetic targets, one of the limitations. We don't exactly know what it is that's going on, and therefore we don't exactly know what we want to do therapeutically.Then the third piece was, this is a mechanism that really is amenable to a particular modality, in this case, antisense oligonucleotides.Where we are today, we have a lot more confidence we actually have a particular molecule that has all those properties we're looking for that can potently correct splicing, both in cells and animal models of disease, and that can do that in a way that is sufficiently well tolerated so we'll be able to have some flexibility in how we dose in the clinic.Trace remains a single-asset company. Is that a harder sell right now, given that biotech investors appear at least somewhat more risk averse?GREEN: This was something I spent a lot of time thinking about, because my prior experience at MyoKardia, Maze, other companies, has been in broader, platform-oriented companies. So this was a big part of the thought process I had as we went from this discovery to: What's the right company?A big part of what made the single asset make sense is it allows us to really get to those important clinical inflection points on a Series A raise, as opposed to saying we're going to explore a bunch of different things, but not be able to get any of them to the point where we get the meaningful clinical data. That was also something that really resonated with investors, because I think there is a real focus on being able to get that meaningful clinical data. That was, in some ways, a tailwind for us.There are some aspects of increased risk when you think about a single asset company. So for us, it was being able to have things far enough along by the time that we launched, that we felt like we had mitigated a lot of those risks. We felt strongly about the genetics, we felt strongly about the mechanism, and we felt strongly enough about being able to find a molecule that could meet our criteria. The risk of not getting to the point where we can test this in patients is relatively mitigated. This wouldn't have made sense when we were talking in 2020 and said, Oh, here's an interesting idea, and here's an interesting mechanism, but we don't know whether it's going to be possible to find a molecule.In some ways, it became an advantage. Now we can really focus the organization on being able to get that critical data. And then after that think about how we might broaden the company, whether it be to other indications or other ways of attacking this target.Whats the current development timeline investors envision for your asset? What data represent a go-no go decision for Trace and its investors?GREEN: We're on track with our lead molecule to be in the clinic by early 2026. We'll be able to, very quickly, do these trials in patients, so that the first studies have the opportunity to show us meaningful clinical data.This is one of the places were able to benefit from some of the work that was done with [Qalsody], even though the focus there is on a very rare population of people with ALS. A lot of the things they learned with respect to biomarkers, in particular, are very relevant for how we think about our clinical development. [In particular] they were able to take advantage of neurofilament as a biomarker of disease progression that really correlated well with the ultimate changes we all hope to see, which is improvements in how people feel, function and survive with ALS.I think that gives us a much stronger toolkit for evaluating our molecules than we had before. So we're obviously going to be looking for those clinical signs, but having some strong biomarkers will help accelerate and give us more tools to see proof-of-concept in the clinic earlier. '

Oligonucleotide

06 Nov 2024

·www.prnewswire.com

Ionis Announces Pivotal Phase 3 Trial Design for ION582 in Angelman Syndrome

Positive End of Phase 2 discussion with FDA, including alignment on Phase 3 design Bayley-4 expressive communication selected as Phase 3 study primary endpoint Initiation of ION582 Phase 3 study planned for H1 2025 Ionis to share ION582 program update at the FAST Global Science Summit in November CARLSBAD, Calif., Nov. 6, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the pivotal Phase 3 study design following successful alignment with the U.S. Food and Drug Administration (FDA) on ION582, an investigational medicine for the treatment of people living with Angelman syndrome (AS). AS typically presents in infancy and is characterized by profound intellectual disability, impaired verbal abilities and severe motor impairment. "Following positive results for ION582 in the Phase 2 HALOS trial, we are pleased to have alignment with the FDA on the design of our Phase 3 REVEAL trial, which will address clinical endpoints that reflect the most pressing and meaningful outcomes for people living with AS and their caregivers," said Brett Monia, Ph.D., chief executive officer of Ionis. "We will enroll a broad group of individuals living with AS in the global pivotal Phase 3 trial, planned to begin in the first half of 2025. We look forward to working with the community to advance a potential new treatment targeting the underlying cause of disease in this debilitating neurological condition that has no approved medicines." The planned global, randomized, placebo-controlled Phase 3 study will enroll approximately 200 children and adults with AS that have a maternal UBE3A gene deletion or mutation. The primary analysis will occur after approximately one year of treatment, followed by all patients transitioning into an open-label long-term extension (LTE) phase of the study. Patients will be randomized 2:1 to active therapy or placebo, and ION582 will be evaluated at two dose levels which will be dosed quarterly without a loading regimen. The primary endpoint will be improvement in expressive communication as assessed by the Bayley Scales for Infant and Toddler Development-4 (Bayley-4), an objective and direct clinician-administered assessment of clinical functioning. Deficits in expressive communication are reported to be the symptoms most challenging to caregivers of people with AS. The study will evaluate several secondary endpoints including overall disease severity, cognition, communication, sleep, motor functioning and daily living skills, in addition to other exploratory endpoints. The End of Phase 2 meeting was supported by data from the Phase 2 open-label HALOS study. In the recently completed multiple ascending dose (MAD) portion of the study, ION582 treatment provided strong evidence of clinically meaningful improvement on all functional domains including communication, cognition and motor function. Overall, 97% of people in the medium and high dose groups assessed in the study saw an improvement in overall AS symptoms as measured by the Angelman Syndrome Clinical Global Improvement Change (SAS-CGI-C) scale, which evaluates clinicians' impressions. ION582 showed favorable safety and tolerability at all dose levels in the study. At the FAST Global Science Summit this weekend (November 8-9), Ionis will provide an update to the community on the Phase 3 program and review data from the MAD portion of the HALOS trial. About ION582 ION582 is an investigational antisense medicine designed to inhibit the expression of the UBE3A antisense transcript (UBE3A-ATS) and increase production of UBE3A protein, for the potential treatment of Angelman syndrome (AS). In 2022, the U.S. Food and Drug Administration (FDA) granted ION582 Orphan Drug designation and Rare Pediatric designation. About Angelman Syndrome (AS) AS is a rare, genetic neurological disease caused by the loss of function of the maternally inherited UBE3A gene. AS typically presents in infancy and is characterized by profound intellectual disability, balance issues, motor impairment and debilitating seizures. Most patients are unable to speak. Individuals with AS have a normal lifespan but require complete care from a caregiver. Some symptoms can be managed with existing medicines; however, there are no approved disease modifying therapies. About Ionis' Neurology Franchise Ionis has been at the forefront of discovering and developing leading neurological disease medicines, including SPINRAZA® (nusinersen), the first approved treatment for spinal muscular atrophy, WAINUA™ (eplontersen), a medicine to treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), and QALSODY® (tofersen) for SOD1-ALS. The clinical-stage portfolio includes 11 therapies, of which five are wholly owned by Ionis. Ionis' investigational portfolio includes medicines for which there are few or no disease modifying treatments, such as rare diseases including Prion disease and Alexander disease and more common conditions such as Alzheimer's and Parkinson's disease. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram. Ionis Forward-looking Statements This press release includes forward-looking statements regarding Ionis' business, and the therapeutic and commercial potential of Ionis' commercial medicines, ION582 and Ionis' additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions, or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended Dec. 31, 2023, and most recent Form 10-Q, which are on file with the SEC. Copies of these and other documents are available at . In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. ION582 is an investigational medicine that has not been approved for the treatment of any disease by regulatory authorities. Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph D. - [email protected] - 760-603-2331 Ionis Pharmaceuticals Media Contact: Hayley Soffer - [email protected] - 760-603-4679 SOURCE Ionis Pharmaceuticals, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultPhase 2Phase 3Orphan DrugDrug Approval

100 Deals associated with Tofersen

External Link

KEGG	Wiki	ATC	Drug Bank
D11811	-	-	DB14782

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Amyotrophic Lateral Sclerosis	EU	Biogen, Inc.	30 May 2024
Amyotrophic Lateral Sclerosis	IS	Biogen, Inc.	30 May 2024
Amyotrophic Lateral Sclerosis	LI	Biogen, Inc.	30 May 2024
Amyotrophic Lateral Sclerosis	NO	Biogen, Inc.	30 May 2024
Amyotrophic Lateral Sclerosis 1	US	Biogen MA, Inc.	25 Apr 2023

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02623699 (FDA) Manual	Phase 3	Amyotrophic Lateral Sclerosis 1 SOD1 Mutation	108	QALSODY 100 mg (ITT population)	ydaeheyvbd(xpicbbvvpw) = lkpajovlnn hwzjyxpetq (fxubcizupx ) View more	Positive	25 Apr 2023
				Placebo (ITT population)	ydaeheyvbd(xpicbbvvpw) = cuwobhexmz hwzjyxpetq (fxubcizupx ) View more
NCT02623699 (VALOR, Pubmed \| Br Dent J \| N Engl J Med) Manual	Phase 3	Amyotrophic Lateral Sclerosis 1 SOD1 Mutation	108	Tofersen	jqhybazjmi(pozwhdkizv) = olxwkeirlr tuqoxmdrrr (lablvkobyk )	Negative	22 Sep 2022
				placebo	jqhybazjmi(pozwhdkizv) = ypyfclwahg tuqoxmdrrr (lablvkobyk )
NCT02623699 (VALOR, Corporate Publications) Manual	Phase 3	Amyotrophic Lateral Sclerosis 1 SOD1	108	Tofersen	gyezpgrdwn(ncqxnwxhyv) = beqawrqkgi ekyiizfylp (bictqfurua )	Positive	03 Jun 2022
				Placebo	gyezpgrdwn(ncqxnwxhyv) = ipudsfdmuw ekyiizfylp (bictqfurua )
NCT02623699 (VALOR, Corporate Publications) Manual	Phase 3	Amyotrophic Lateral Sclerosis 1 SOD1 mutation	108	tofersen	vvjmypkhwj(albvywlrfz) = ophtvjytvq kybtsyffqy (yyquhdwwgd ) View more	Positive	17 Oct 2021
				Placebo	vvjmypkhwj(albvywlrfz) = mmnavmippc kybtsyffqy (yyquhdwwgd ) View more
NCT02623699 (VALOR, Pubmed \| Br Dent J \| N Engl J Med) Manual	Phase 1/2	Amyotrophic Lateral Sclerosis 1 SOD1 Mutation	50	Tofersen	jntsokgdxi(vmfsmmqafs) = xxhztnqfki xgoruurxld (necuoibjbv ) View more	Positive	09 Jul 2020
				Placebo	fbddqhynxk(nlenkabgyc) = hkxkpaifiu tatwcxppai (zjdoldevkn )
NCT01041222 (Pubmed) Manual	Phase 1	Amyotrophic Lateral Sclerosis 1 SOD1 Mutation	32	ISIS 333611	erxnnjopzn(kenmlnmirr) = lrbpkwiven qpcctqkzrt (ukbgipgmtv ) View more	Positive	01 May 2013
				Placebo	-

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