daniel-gordon/biopharma-dive huggingface dataset:
One called navacaprant is being studied in patients with moderate-to-severe major depressive disorder
Its primary focus Harasaki told BioPharma Dive is on helping biotech startups speed up the drug discovery and development process
According to the documents FDA staff found BrainStorms approval application filed last September to be scientifically incomplete.
Two were in April three in May recovered either back to where it started or almost to baseline
About SRP9001102 Study SRP9001102 Study 102 is a doubleblind 11 randomized placebocontrolled clinical trial of SRP9001 in 41 participants with Duchenne muscular dystrophy between the ages of 47
If successful the PPMO offers the potential for improved efficacy and less frequent dosing for patients
Without an approval there the commercial opportunity for the drug is much smaller
Still there have been some signs of a turnaround before Friday. Apogee Therapeutics and Sagimet Biosciences which both went public in July upsized their IPOs and offered more shares than originally planned. And on Wednesday British chip designer Arm raised nearly 49 billion in the largest IPO to list on Nasdaq this year;  an event The New York Times likened to Wall Streets Groundhog Day Arms doubledigit stock gains on Thursday along with the news from RayzeBio and Neumora might indicate sunnier days ahead
Research indicates that people with this disease have a life expectancy two decades shorter than the general population. Vertex and CRISPR Therapeutics hope to provide a potentially onetime therapy in exacel which Vertex licensed in 2017 after previously partnering with the smaller biotech. Vertex says its drug development strategy revolves around picking targets where the need for new treatments
TSHA102 utilizes a novel miRARE platform designed to mediate levels of MECP2 in the CNS on a cellbycell basis without risk of overexpression
Shares in Acelyrin fell by more than half the morning aer the company disclosed clinical trial results
Serum cystatin C urine dipstick and urine proteintocreatinine ratio should be measured before starting VYONDYS 53
Two more clinicalstage biotechs Adlai Nortye and Jyong Biotech are also waiting in the queue
Prior to treatment the patient was in a constant state of hypertonia had limited body movement required constant back support and had lost fine and gross motor function early in childhood
Contacts Investors Media Traci McCarty Debra Charlesworth BioMarin Pharmaceutical IncBioMarin Pharmaceutical Inc 415 4557558 415 4557451 SOURCE BioMarin Pharmaceutical Inc102023 651 PM BioMarin Receives Complete Response Letter CRL from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A  Au httpsinvestorsbiomarincom20200819BioMarinReceivesCompleteResponseLetterCRLfromFDAforValoctocogeneRoxaparvovecGeneTherapyforSe 66Navigate Our Motivation Our Treatments Our Company Investors  MediaAbout Our History Our Products Our Pipeline Leadership Team Board of Directors Diversity Equity  Inclusion Contact Global Contact Information Our Locations Report a Product Complaint Report Adverse Event Publication Data Request Funding  SupportConnect Careers LinkedIn Facebook Instagram YouTube Privacy Policy Terms of Use Site Map Supplier Information Slavery Act Statement Glossary Contact Us  2023 BioMarin
Roche and Ionis have worked together for years. The two are currently collaborating on tominersen a type of drug called an antisense oligonucleotide that blocks the expression of a protein mutated in Huntingtons disease. That drug is in a Phase 2 trial measuring biologic signs of disease as well as patient function
Sage took home more than 15 billion from the deal to start and is eligible for as much as 16 billion in further payments provided it hits certain milestones
Sanofis interest in MeiraGTx is outside the eye however. Specifically the pharma is interested in MeiraGTxs Riboswitch technology for regulating targets related to immune and central nervous system conditions as well as gut hormones like GLP1
In the 88 patients who received 30 mgkgweek of EXONDYS 51 for up to 208 weeks in clinical studies the following events were reported in 10 of patients and occurred more frequently than on the same dose in Study 1 vomiting contusion excoriation arthralgia rash catheter site pain and upper respiratory tract infection
These were difficult but necessary decisions. The job cuts make Apellis one of more than 100 drugmakers to lay off employees this year according to data compiled BioPharma Dive
This is an extraordinarily important moment for our program and for the many families living with Duchenne
Known risk factors include the following the FDA may not approve the BLA for SRP9001 by the application PDUFA date or at all we may not be able to comply with all FDA requests including with respect to our SRP9001 BLA in a timely manner or at all the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business as well as the development of our product candidates and our financial and contractual obligations our dependence on certain manufacturers to produce our products and product candidates including any inability on our part to accurately anticipate product demand and timely secure manufacturing capacity to meet product demand may impair the availability of product to successfully support various programs our data for SRP9001 may not be sufficient for obtaining regulatory approval success in preclinical and clinical trials especially if based on a small patient sample does not ensure that later clinical trials will be successful and the results of future research may not be consistent with past positive results or with advisory committee recommendations or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates the commencement and completion of our clinical trials and announcement of results may be delayed or prevented for a number of reasons including among others denial by the regulatory agencies of permission to proceed with our clinical trials or placement of a clinical trial on hold challenges in identifying recruiting enrolling and retaining patients to participate in clinical trials and inadequate quantity or quality of supplies of a product candidate or other materials necessary to conduct clinical trials different methodologies assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results and even if we believe the data collected from clinical trials of our product candidates are positive these data may not be sufficient to support approval by the FDA or other global regulatory authorities we may not be able to execute on our business plans including meeting our expected or planned regulatory milestones and timelines research and clinical development plans and bringing our product candidates to market for various reasons many of which may be outside of our control including possible limitations of company financial and other resources manufacturing limitations that may not be anticipated or resolved for in a timely manner regulatory court or agency decisions such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the ongoing COVID19 pandemic and those risks identified under the heading Risk Factors in our most recent Annual Report on Form 10K for the year ended December 31 2022 and Form 10Q filed with the Securities and Exchange Commission SEC as well as other SEC filings made by the Company which you are encouraged to review
Efavirenz is not recommended in patients treated with ROCTAVIAN
For us at Jax we believe this collaboration with Actio will help to serve the rare disease community in transformative ways to build a bridge of preclinical work to therapies that are desperately needed said Cat Lutz head of Jaxs rare disease translational center in an emailed statement
Nurix already had two cancertargeting protein degraders in clinical testing both targeting a protein called BTK that is active in certain types of lymphoma
Vials of Eisai and Biogens Alzheimers drug Leqembi Courtesy of Eisai Eisai now plans to submit an approval application for subcutaneous Leqembi by the end of March at which point the company will have 12month data from its study
These forwardlooking statements involve risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forwardlooking statements
Taysha Gene Therapies Announces Fast Track Designation Granted by US FDA for TSHA102 in Rett Syndrome Fast Track Designation FTD is designed to accelerate the development and expedite the review of therapies with potential to address unmet medical needs for a serious or lifethreatening condition TSHA102 has also received Orphan Drug and Rare Pediatric Disease designations from the United States US Food and Drug Administration FDA and has been granted Orphan Drug designation from the European Commission DALLAS Aug 24 2023 GLOBE NEWSWIRE  Taysha Gene Therapies Inc Nasdaq TSHA a clinicalstage gene therapy company focused on developing and commercializing AAVbased gene therapies for the treatment of monogenic diseases of the central nervous system CNS today announced the US FDA has granted Fast Track Designation FTD to TSHA102 a selfcomplementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome
Conference Call and Webcast Information Taysha management will hold a conference call and webcast today at 830 am ET to review its financial and operating results and to provide a corporate update
Data is king and its something that is really standing true in this market. But as if often the case in biotech thats not always an easy task
Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting AMONDYS 45
Former biotechnology unicorn Intarcia Therapeutics is getting a new life less than a month before a panel of Food and Drug Administration advisers could decide the fate of the diabetes treatment its spent more than a decade trying to develop
The San Diego startup believes TRPV4 an ion channel protein could be important for treating a degenerative nerve disease called CharcotMarieTooth
Novartis licensed buparlisib to Adlai Nortye for only 95 million upfront in a heavily backloaded deal according to the biotechs IPO filing
Eisai and Biogen have been testing whether a different form of Leqembi an undertheskin injection can be as safe and effective as the already marketed version. On Wednesday at a medical conference in Boston researchers presented results from a study of nearly 400 participants that suggests the two forms are roughly comparable. the clinical trial that initially led to Leqembis approval
Researchers believe that using protein degradation could defeat the mechanisms cancer cells use to become resistant to current targeted cancer drugs
If successful we believe vutrisiran will offer convenient quarterly subcutaneous dosing with a therapeutic profile that may potentially include cardiovascular outcome benefits The FDAs decision is not a total surprise
Actio, a precision medicine startup, launches with 55M and a mouse lab partnership The two year-old San Diego biotech is allied with The Jackson Laboratory to engineer better mouse models of the rare diseases it plans to target
Urine obtained on the day of VYONDYS 53 infusion prior to the infusion or urine obtained at least 48 hours after the most recent infusion may be used
In June CellTrans won the first Food and Drug Administration approval for a cellular therapy to treat type 1 diabetes
The enzyme is already present in the body helping to modify and regulate genes
AI drug discovery startup Genesis Therapeutics has raised $200 million to push its first drug candidates into clinical testing announcing Monday a Series B funding round. Genesis plans to use the money to advance its pipeline of AI enabled drug programs and to expand its research in new directions
The presentation will be webcast live under the investor relations section of Sareptas website at httpsinvestorrelationssareptacomeventspresentations and slides will be archived there following the call for one year
Accompanying secondary biological endpoints including vector genome copies per nucleus percent positive fibers intensity and reduction in creatine kinase exploratory were also met
Analysts have predicted filings will pick up in the second half of this year but only a few have joined the queue so far
Under the deal PeptiDream will receive 40 million up front and is eligible to receive up to 1 billion in milestone payments. The new deal expands upon an initial partnership formed between the two companies in 2015 which resulted in Genentech licensing its platform system technology the following year
A cell therapy for Parkinsons disease scored positive results in an earlystage clinical trial according to an announcement Monday from its developer Bluerock Therapeutics
Also known as calpainopathy LGMD2A is caused by mutations in the CAPN3 gene and is the most common type of LGMD accounting for almost a third of cases
Sibold will take over from Friedman who has run Madrigal since 2016  a period during which the fortunes of NASH drugmakers soared on skyhigh forecasts of a large new drug market only to crash on clinical and regulatory setbacks
View source version on businesswirecom  httpswwwbusinesswirecomnewshome20230512005452en Investor Contact Ian Estepan  6172744052 iestepansareptacom Media Contact Tracy Sorrentino  6173018566 tsorrentinosareptacom Source Sarepta Therapeutics Inc
Known risk factors include among others success in preclinical and clinical trials especially if based on a small patient sample does not ensure that later clinical trials will be successful the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit different methodologies assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results and even if we believe the data collected from clinical trials of our product candidates are positive these data may not be sufficient to support approval by the FDA or foreign regulatory authorities if the actual number of patients suffering from DMD is smaller than estimated our revenue and ability to achieve profitability may be adversely affected we may not be able to execute on our business plans and goals including meeting our expected or planned regulatory milestones and timelines clinical development plans and bringing our product candidates to market due to a variety of reasons some of which may be outside of our control including possible limitations of company financial and other resources manufacturing limitations that may not be anticipated or resolved for in a timely manner regulatory court or agency decisions such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the COVID19 pandemic and even if Sareptas programs result in new commercialized products Sarepta may not achieve the expected revenues from the sale of such products and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10K for the year ended December 31 2020 as well as other Securities and Exchange Commission SEC filings made by the Company which you are encouraged to review
The Phase 3 study was fully enrolled in November 2019 and the last patient will complete two years of follow up in November 2021
JJ was also an early investor in Rapport Therapeutics  a startup that launched in March with hopes of creating precision medicines for central nervous system diseases
FDA scientists had questioned at a recent regulatory meeting how much the RNA based medicine actually benefits ATTR amyloidosis patients with heart damage
ALS drug development BrainStorm after setbacks withdraws approval application for ALS drug The company now says a Phase 3b study will be needed for its NurOwn cell therapy to have a shot at an approval
The offshoot launched in December  was backed by $40 million from Star and other investors
GerhartHines and Schwartz have published research on two proteins FFAR4 and GPR3  that regulate energy expenditure and the breakdown of fatty tissue
Besides RayzeBio and Neumora 13 other biotechs have priced IPOs so far this year though nine raised less than 100 million. The pace is in stark contrast to 2020 and 2021 when 183 biotech companies made their Wall Street debuts and raised nearly 30 billion combined
Sangamo Therapeutics is making deeper cuts to its business in an attempt to save cash announcing Wednesday plans to lay off 40 of its workforce and postpone further investment in certain research
The commercial success of drugs such as AstraZenecas Enhertu and Seagens Adcetris has prompted a recent flurry of ADCrelated dealmaking including one last month between Merck and Co and Daiichi Sankyo
Ascendis Ascendis Pharma the Ascendis Pharma logo the company logo and TransCon are trademarks owned by the Ascendis Pharma group
Novo Nordisk  Novo Nordisk is adding to its recent string of obesityrelated dealmaking announcing Wednesday it has agreed to pay 15 million euros or about 16 million to buy Embark Biotech a startup it helped found from a research center at the University of Copenhagen
The partnership seeks to find and validate new drug targets for uncommon neurodegenerative and neuromuscular diseases
Airna which was cofounded by a group of scientists including researchers at Stanford University and the University of Tübingen looks at a protein known as ADAR which stands for adenosine deaminase acting on RNA
Acelyrin which raised 540 million in a rare IPO success this May lost more than half of its market value in September when it disclosed negative data for its lead drug
ForwardLooking Statements This press release contains forwardlooking statements about the business prospects of BioMarin Pharmaceutical Inc BioMarin including without limitation statements about the potential benefits and impact of ROCTAVIAN for treating adults with severe hemophilia A including i the potential for ROCTAVIAN to transform treatment for such patients by providing years of bleed control following a single infusion of ROCTAVIAN and ii allowing such patients to produce their own FVIII and thereby limit bleeding episodes the clinical development of ROCTAVIAN including i BioMarins continued monitoring of the longterm effects of treatment with an extension study as well as postapproval studies and the duration of such monitoring and ii clinical studies regarding potential product expansion opportunities for ROCTAVIAN the commercialization of ROCTAVIAN including i BioMarins ability to manufacture sufficient ROCTAVIAN to meet commercial demand throughout the products lifecycle ii BioMarins plans to immediately educate physicians and patients about ROCTAVIAN iii BioMarins goal to ensure that every eligible adult interested in ROCTAVIAN is able to receive treatment iv BioMarins plans to offer outcomesbased warranties for ROCTAVIAN to all US insurers and v BioMarins goal that HTCs are prepared to administer ROCTAVIAN the expectation that ROCTAVIAN will offer significant savings to society the expectation that BioMarins outcomesbased warranties will further enhance the value of ROCTAVIAN to payers and BioMarins expectations regarding the number of adult patients with severe hemophilia in the US who will be eligible to receive ROCTAVIAN with the initial FDA approval
It later hopes to test its base editing approach in broader groups of people with heart disease and with other gene targets linked to heart risk
Bayer had also acquired rights to two experimental cell therapies developed by Atara Biotherapeutics as potential treatments for solid tumors
Recently it launched its Health Action Plan tool which provides health recommendations based on personal genetic reports health history survey data and blood and biomarker data
Accordingly these forwardlooking statements do not constitute guarantees of future performance and you are cautioned not to place undue reliance on these forwardlooking statements
PTC secured a renewal of that approval in 2017 but agreed to conduct another placebo controlled study of the drug in return. That trial failed to meet its goal however falling short of reaching statistical significance in the primary analysis subgroup
The CTGTACs vote while not binding will be considered by the FDA when making its decision regarding the potential accelerated approval of SRP9001
Ajinomoto and Forge expect the deal which is one of the largest gene therapy transactions this year to close in the fourth quarter of this year. A handful of other gene therapy developers  as well as one manufacturer  have also been bought by larger drugmakers over the past year
Companies such as Structure Therapeutics and Septerna have trained a spotlight on them again aiming to capitalize on advancing science and improving research tools
Investors didnt appear ready to give the Los Angeles biotech the benefit of the doubt Tuesday as the companys share price plummeted 60 to about 11 in early trading
For further information please see the full Prescribing Information About AMONDYS 45 AMONDYS 45 casimersen is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping
It plans to deploy capital in Series A through C financing rounds seeing biotechnology startups through to clinical testing said Kouki Harasaki a founding and managing partner at Bioluminescence
About Hemophilia A People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful andor potentially lifethreatening bleeds from even modest injuries
On that measure researchers said that after six months of treatment the average reduction in amyloid was 14 higher with subcutaneous Leqembi compared to intravenous
The FDA has identified deficiencies in the Companys New Drug Application NDA for TransCon PTH palopegteriparatide in hypoparathyroidism that at this time preclude s them from holding further discussions about labeling and post marketing requirements commitments
Molecular glue degraders are an exciting modality to target diseaserelated proteins that have proven challenging with more traditional treatment modalities James Sabry the global head of Roche Pharma Partnering said in a statement
Like many biotechnology companies Seraxis has an ambitious aim. The company wants to transform the lives of millions of diabetics who must take insulin every day to control their blood sugar levels
Brian Abrahams an analyst at the investment firm RBC Capital Markets wrote in a recent note to clients how bottlenecks that have hindered sales of antiamyloid drugs  including screening and monitoring of patients  should mostly be alleviated by the time subcutaneous Leqembi could enter the market
In Europe our regulatory review continues as expected and we remain on track for a European Commission decision in the fourth quarter
Clinical trial results presented in October appear to application to the FDA by the end of March meaning subcutaneous Leqembi could be cleared and on the market by early 2025
Unlike the first gene therapies approved in the US which relied on adenoassociated viruses infused into patients to deliver a genetic payload Libmeldy uses a lentivirus to correct faulty genes in stem cells that have been removed from the patient and are later reintroduced to treat the disorder
The majority of patients in the clinical trial required corticosteroids for ALT elevation median duration of corticosteroid use was 35 weeks
Despite some manufacturing roadblocks  their uptake is an encouraging sign for a new crop of startups working in the field . One of them RayzeBio  recently filed for an initial public offering in a test of a market thats been cool to young biotechs seeking public investment
The FDAs view on Zurzuvaes safety profile therefore does not appear completely Abrahams an analyst at the investment firm RBC Capital Markets