daniel-gordon/biopharma-dive huggingface dataset:
COREHH has an estimated primary completion date of December 2023 according to clinicaltrialsgov  with full completion scheduled for July 2025
The investment announced by the gene therapy maker Monday  gives Sanofi the right of first negotiation to license certain MeiraGTx technology in immunology neurology and metabolic disease settings. MeiraGTx is perhaps best known for its work on gene therapies for the eye but Mondays deal shows its progress elsewhere
That aim is on the more optimistic side wrote RBC Capital Markets analyst Brian Abrahams in a note to clients. Yet Abrahams and his still predict an upward inflection for Leqembi ultimately resulting in peak annual sales north of 10 billion
SRP9001 is supported by nonclinical evidence in addition to efficacy and safety data from studies 101 102 and 103 as well as an integrated analysis across these three clinical studies comparing functional results to a propensityscoreweighted external control EC
In 2022 the copmany executed a 1f-or-20 reverse stock split
The secondary endpoints evaluate the eicacy of patisiran vs placebo over 12 months in a hierarchical manner with the following measures Healthrelated quality of life with the KCCQ change from baseline at 12 months Composite of allcause mortality frequency of cardiovascular CV events CV hospitalizations and urgent heart failure HF visits and change from baseline in 6MWT Composite of allcause mortality and frequency of allcause hospitalizations and urgent HF visits in patients not on tafamidis at baseline and Composite of allcause mortality and frequency of allcause hospitalizations and urgent HF visits in the overall study population
While there have been some signs of life the pace of biotech IPOs has remained sluggish in 2023 with just one company that about half of successful IPOs in 2023 have at least one drug candidate in midstage clinical trials or later
In a deeper move into radiopharmaceuticals Roches Genentech unit will collaborate with the Japan-based biotechnology company PeptiDream to develop peptideradioisotope conjugates PeptiDream said Wednesday
DanaFarber spinout Precede exits stealth with blood test technology and $57M. Precede has used the money to develop a platform that reveals the activity of genes and pathways
ATTR amyloidosis is caused by the toxic accumulation of mutated transthyretin or TTR protein in the nerves and organs causing damage
In many ways Neumora fits the profile of a company that could successfully pull off an IPO amid a market downturn thats stalled other biotechs march to Wall Street. Importantly it already has two drug candidates in clinical trials giving investors nearterm catalysts on which the companys fortunes could rise
Studies supporting the application found that the new drug could reduce symptoms of schizophrenia without some of the common side effects that plague existing medications Karuna said Thursday
In BrainStorms case NurOwn failed to met its primary and secondary goals in a Phase 3 study of nearly 200 people with ALS
Most patients require fulltime use of a wheelchair in their early teens and then progressively lose the ability to independently perform activities of daily living such as using the restroom bathing and feeding
The deficiencies were not disclosed in the letter
The agency cleared the medicine for women with postpartum depression but rejected its use in a much wider pool of patients with major depressive disorder
The dose of VOXZOGO is based on body weight
BrainStorm then took the rare step earlier this year of submitting its application over the FDAs protest as part of its review of BrainStorms therapy called debamestrocel or NurOwn
Those who would like to participate may access the live webcast here or register in advance for the teleconference here
Apellis Pharmaceuticals is laying off one quarter of its workforce in an overhaul of its business designed to focus resources on a newly approved vision loss drug
Sarepta Therapeutics Announces Topline Results for Part 1 of Study 102 Evaluating SRP9001 its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy 1721  Study met the primary biological endpoint of microdystrophin protein expression at 12 weeks posttreatment as measured by western blot in SRP9001treated participants versus placebo   SRP9001treated participants showed an increase in NSAA total score compared to placebo at 48 weeks however the study did not achieve statistical significance on the primary functional endpoint of improvement in NSAA total score compared to placebo at 48 weeks posttreatment   In the prespecified analysis by agegroup by which the randomization was stratified participants aged 45 years at time of treatment with SRP9001 demonstrated a statistically significant improvement in NSAA total score versus the agematched placebo cohort achieving a 43point improvement on NSAA at 48 weeks posttreatment from baseline   No new safety signals identified for SRP9001 reinforcing the favorable safety profile observed to date   Sarepta to host conference call at 430 pm Eastern time  CAMBRIDGE Mass Jan 07 2021 GLOBE NEWSWIRE  Sarepta Therapeutics Inc NASDAQSRPT the leader in precision genetic medicine for rare diseases today announced topline results from Part 1 of Study SRP9001102 Study 102 an ongoing randomized doubleblind placebo controlled clinical trial to evaluate the safety efficacy and tolerability of a single dose of SRP9001 rAAVrh74MHCK7microdystrophin in 41 patients with Duchenne muscular dystrophy
On a nonGAAP basis the net loss for the six months ended June 30 2021 was 2437 million or 306 per basic and diluted share compared to a net loss of 1977 million or 256 per basic and diluted share for the same period of 2020
An experimental medicine from FibroGen has failed a second late stage trial in Duchenne muscular dystrophy  adding to a string of clinical setbacks that have caused the biotechnology companys shares to collapse in recent months
Interested parties participating by phone will need to register using this online form
The company licensed the drug to Australian drugmaker CSL Behring in 2020 for $450 million  providing the former the cash to continue developing AMT130 among other programs
Verve which specializes in a cuttingedge form of genetic medicine known as base editing said Monday that it had resolved questions raised by the Food and Drug Administration last fall when it first requested clearance to begin US testing
Progressive muscle weakness in the lower limbs spreads to the arms neck and other areas of the body
Contacts Investors Media Traci McCarty Andrew Villani BioMarin Pharmaceutical Inc BioMarin Pharmaceutical Inc 415 4557558 628 2697393 SOURCE BioMarin Pharmaceutical Inc 102023 805 PM US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia  Oct 20  httpsinvestorsbiomarincom20231020USFoodandDrugAdministrationApprovesBioMarinsVOXZOGORvosoritideforChildrenUnder5Yearswith 1111Our Treatments Our Company Investors  MediaOur 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
Others in cancer cardiovascular immunology and neurology are in the discovery and preclinical phases
These forwardlooking statements include statements regarding the potentially transformative benefits of SRP9001 our plan to analyze the functional results for all the patients in Study 102 once they have achieved the 48week timepoint the expectation to have biomarker and safety results from Study 103 in the second quarter of 2021 our plan to use the information and insight from Study 102 to refine and complete the protocol for our upcoming trial using commercial process material and our intention to continue to move forward with diligence and urgency to generate the evidence necessary to bring SRP9001 to waiting Duchenne patients around the world
Unlike most biotechs which develop their business around one or many drugs or even a drugmaking platform Star functions as a central hub around which its various subsidiaries orbit
The company specializes in using artificial intelligence to better model and predict the properties of proteins and their molecular partners
They would like to offer genetic information to their patients and they're just not trained on it
Emerging biotech OrbiMed raises 43B in new funds for startup investing The money will be spread across three private investment funds aimed at supporting life science companies
RayzeBio which is developing a drug that targets the same tumors as Lutathera wants to break in on the emerging market. The most richly funded of more than a dozen radiopharmaceutical startups  the company began a Phase 3 trial for its experimental neuroendocrine tumor drug RYZ101 in May
If a hypersensitivity reaction occurs institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy
I believe that the patient achieving these milestones so early in treatment coupled with the improvements in breathing patterns and quality of sleep that we have observed are highly encouraging and support the potential of TSHA102
One of its competitors Wave Life Sciences  said in early September that it planned to dose the first person in its clinical trial for an RNA editing therapy for AATD by the end of the year
Taysha gets cash lifeline from new investor RA Capital The 150 million private investment in public equity will extend Tayshaʼs operating runway from early next year to late 2025
As the disease progresses muscle weakness in the lower limbs spreads to the arms and other areas
Bluerock launched in late 2016 as part of a joint venture between Bayer and the investment firm Versant Ventures. By 2019 Bayer had agreed to buy all the startups equity it didnt already own for 240 million upfront and potentially another 360 million provided Bluerocks programs hit certain development milestones
The potentially blinding condition causes inflammation that blocks blood flow through eye vessels
The Agency recommended that the Company complete the Phase 3 Study and submit twoyear followup safety and efficacy data on all study participants
The company increased its sales estimate for Voxzogo to between 435 million and 455 million for the year up from a range of 400 million to 440 million
By extending our cash runway into the third quarter of 2025 we can focus on execution as we endeavor to deliver on key valuecreating milestones
Kidney function should be monitored in patients taking AMONDYS 45
Other big drugmakers such as Merck  Co and Bristol Myers Squibb are acquiring a stake in the new space that while enticing still faces several obstacles through development
101723 633 AM Alnylam Pharmaceuticals Press Release  Aug 03 2022  Alnylam Reports Positive Topline Results from APOLLOB Phase 3 Study of Patisira httpsinvestorsalnylamcompressreleaseid26851 19 Alnylam Reports Positive Topline Results from APOLLOB Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy Aug 03 2022  Patisiran Met the Primary Endpoint with a Statistically Significant Improvement in 6Minute Walk Test Compared to Placebo at 12 Months   Patisiran Also Met the First Secondary Endpoint with a Statistically Significant Improvement in Quality of Life as Measured by the Kansas City Cardiomyopathy Questionnaire Compared to Placebo at 12 Months  101723 633 AM Alnylam Pharmaceuticals Press Release  Aug 03 2022  Alnylam Reports Positive Topline Results from APOLLOB Phase 3 Study of Patisira httpsinvestorsalnylamcompressreleaseid26851 29 Patisiran Demonstrated Encouraging Safety and Tolerability Profile in Patients with ATTR Amyloidosis with Cardiomyopathy   Company Plans to File a Supplemental New Drug Application in US in Late 2022   Full Data Will Be Presented at the 18 International Symposium on Amyloidosis   Alnylam to Host Conference Call Today at 800 am ET  CAMBRIDGE MassBUSINESS WIREAug 3 2022 Alnylam Pharmaceuticals Inc httpsctsbusinesswirecomctCT
Forge currently has one drug in a clinical trial a gene therapy for a rare neurodegenerative condition called infantile Krabbe disease. The Phase 12 study is expected to be completed by the end of 2025. A second gene therapy with an undisclosed rare disease target is in preclinical development
In Alzheimers, Roche was among the early innovators to develop antibody drugs capable of blocking a protein called amyloid that accumulates in the brains of people with the disease
Study 102 is ongoing and remains blinded to participants investigators site staff and sponsor staff with direct site interaction
Bioluminescences investments will focus on several broad areas including breakthrough biology genetic medicine and platforms for drug discovery and computational biology
Vertex CEO Reshma Kewalramani told investors on an Aug 1 call that the decision to hold an advisory panel wasnt surprising given the new mechanism of action. The meeting will offer an opportunity to hear from patients and highlight the transformative potential of exacel she said
Exonskipping and dystrophin production in the 30 mgkg cohort were also consistently higher than the 20 mgkg cohort of MOMENTUM
Reporter: What are some things the company has not tried yet? CEO: I'm always surprised that genetics is not well adopted in clinical trials because you can use genetics to pick the right population to recruit a clinical study. The whole biotechpharma industry still hasnt quite grasped how much people want to be participants and partners in healthcare
Not all have panned out as planned  however. Under deal terms Tentarix has several opportunities to secure further payment from Gilead should the larger biotech opt to acquire one of the programspecific subsidiaries. The companys structure shares some similarities with those used successfully by Teneobio  IFM Therapeutics and Nimbus Therapeutics
The results from Study 102 reinforce the favorable safety and tolerability profile of SRP9001 with no new safety signals identified
The most common adverse reactions were balance disorder and vomiting
Startup Airna em erges from stealth to br ing RNA editing to a rare disease The biotech company initially plans to develop a drug for alpha1 antitrypsin deﬁciency
In an earlystage study that used a different dose and formulation of Zurzuvae than whats now approved a 26 year-old male patient was unresponsive to stimuli for 50 minutes and had trouble feeling pain
Companies are more mature they have quality management teams but they also have very important data points or data readout coming up once theyre public In the past the market was more receptive to innovative early stage companies
Prior to its COVID research Gritstone company was working on developing cancer immunotherapies  testing it has continued to progress with a midstage study of a personalized cancer vaccine
Like Epizyme, Fulcrum formed with a plan to develop epigenetic drugs which are meant to control molecular switches that turn genes on or off. But whereas Epizyme  now owned by Ipsen  developed cancer medicines Fulcrum targets rare genetic diseases
Urine obtained on the day of AMONDYS 45 infusion prior to the infusion or urine obtained at least 48 hours after the most recent infusion may be used
Izokibep targets a protein called interleukin17A or IL17A that plays a role in inflammation in the body
Private financing rounds are taking longer to close for some while other face a Series A cliff as they go back to investors for more money
I do not think you change from within because the incentives are just wrong. And every time I ask that question: Who profits if I stay healthy? no one has an answer for me. There is no one who makes money if I stay healthy
Furthermore the number of deaths in the allcause mortality eicacy analysis was 4 22 percent in the patisiran arm and 10 56 percent in the placebo arm determined in accordance with the predefined statistical analysis plan which excluded death due to COVID19 and treated cardiac transplant as a death event consistent with other studies in the field
The clearance spurred some debate as treatment led to allergic reactions in participants involved in its testing but Nestlé saw a market opportunity and later that year bought the rest of Aimmune that it didn't already own
Orchard closed a fundraising worth up to $188 million with private equity and venture capital firms to help fund operations through at least 2025
Verves treatment dubbed VERVE101 uses a form of CRISPR gene editing base editing that allows drugmakers to make edits to a single DNA letter or base
When we had the opportunity to hire Richard Scheller that was the beginning of the transition of 23andMe from more of a consumer company into a true healthcare company and that was not well understood by the industry
Like other drugmakers that have cut jobs Apellis is dealing with a depressed stock price
A private company founded in 2019 announced this week it acquired Intarcias assets among them the biotechs diabetes drugeluting implant and the technology on which it was built
The treatment now known as Zurzuvae was developed by Massachusettsbased biotechnology company Sage Therapeutics. Through a series of latestage clinical trials Sage tested Zurzuvae as a potential therapy for both postpartum depression and major depressive disorder
Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider
On the first measure known as the sixminute walk test study participants taking patisiran walked 15 meters further one year after the trials start than those who received a placebo
102023 805 PM US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia  Oct 20  httpsinvestorsbiomarincom20231020USFoodandDrugAdministrationApprovesBioMarinsVOXZOGORvosoritideforChildrenUnder5Yearswith 111Clinical Trials Contact Us Careers US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia       Oct 20 2023 Expanded Indication in the US Now Includes Children of all ages with Achondroplasia SAN RAFAEL Calif Oct 20 2023 PRNewswire  BioMarin Pharmaceutical Inc Nasdaq BMRN a global biotechnology company dedicated to transforming lives through genetic discovery today announced that the US Food and Drug Administration FDA has approved the supplemental New Drug Application sNDA for VOXZOGO vosoritide to increase linear growth in pediatric patients with achondroplasia with open epiphyses growth plates
Its board also recently changed with the controversial election of one new member and the departure of three others who chose not to run for reelection. Some analysts argue that Biogen was hindered in its ability to pursue business development because of board infighting. The recent board and executive team changes may suggest the company is becoming more harmonious analysts have speculated
After registering for dialin details all phone participants will receive an autogenerated email containing a link to the dialin number along with a personal PIN number to use to access the event by phone
Those results are from a trial called APOLLOB  which tested patisiran on two main goals
Two other drugmakers Adlai Nortye and Jyong Biotech also joined the IPO queue in the past month
About LNP Technology Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology
Drugs that block TL1A have become a hot commodity as pharma companies hunt for the next inflammatory disease blockbuster
Transatlantic venture capital group Abingworth has raised 356 million to invest in latestage drug development by pharmaceutical and biotechnology companies
It is not known if VOXZOGO will harm the unborn baby
The panel of 20 voting advisers will be asked to vote on whether BrainStorms clinical trial data meet the FDAs bar for substantial evidence of effectiveness and if not how a subsequent trial should be designed
Courtesy of Teva Pharmaceutical  Sanofi will pay Teva Pharmaceutical 500 million to collaborate on an experimental gut disease treatment betting the medicine could become a topselling product in one of the most competitive areas of pharmaceutical drug development
The agency uses such programs for drugs that have notable safety concerns they are designed to help doctors and patients follow best practices to ensure that a medications benefits outweigh its risks
In a note to clients Duncan wrote that in the US there are about 30000 Huntingtons patients of which roughly 90 experience chorea. And among that group approximately 70 have moderatetosevere forms of the disorder
Please see additional safety information in the full Prescribing Information and Patient Information
The No.1 reward I can imagine is at some point we have a therapy that benefits our customers she said
Novo the drugmaker itself has invested in development of cell therapies building a stem cell facility in California in 2018
Less than one year after selling an experimental medicine to Takeda for 4 billion  Nimbus Therapeutics is reloading with new funding meant to support its next wave of drug candidates among them a prospective cancer therapy
French drugmaker Sanofi has invested 30 million into MeiraGTx Holdings a London and New Yorkbased developer of gene therapies through a purchase of 4 million shares at 750 apiece
Developed by partners Eisai and Biogen, Leqembi is the only Alzheimers medicine of its type with a full approval from the Food and Drug Administration. Its specifically for patients in the early stages of the disease and is given as an hourlong intravenous infusion once every two weeks
Last year Merck expanded in Singapore with a new packaging facility to support its cancer drug Keytruda and HPV vaccine Gardasil
The Series A financing  announced Monday was coled by venture capital firm Forbion and European investor EQT Life Sciences alongside new backers that included Merck  Cos venture arm and the ALS Investment Fund. According to Forbion VectorYs financing is one of the largest funding rounds for a private biotech in Europe this year
About TransCon PTH TransCon PTH palopegteriparatide is an investigational prodrug in development for adult patients with hypoparathyroidism
Sanofi will also hold a right of first negotiation on MeiraGTxs program for radiationinduced xerostomia a condition in which salivary glands are impaired or damaged
Study randomization was stratified by age group and in the prespecified analysis of participants aged 45 n16 at the time of treatment the treatment group demonstrated a statistically significant 43point improvement on NSAA total score at 48 weeks post treatment compared to a 19point improvement in the agematched placebo group P00172
As such we are encouraged to see the potential of patisiran to improve the functional capacity and quality of life of patients living with this fatal multisystem disease
The Company remain s on track for a European Commission decision on the Marketing Authorisation Application for TransCon PTH during the fourth quarter of 2023
Its initial financing was led by Arch Venture Partners and includes investors such as ND Capital Fast Track Initiative Novalis and Codon Capital
One month later the biotech ended a Phase 3 COVID19 booster program when the shot showed limited efficacy against new virus variants
Built from several startups merged together Neumora was a major bet that advancing science could open up new drugmaking opportunities for challenging-to-treat diseases
Whats so wonderful about RNA editing is that its using a modality and a medicine that can be accessible to millions of patients the CEO said
Nestlé on Monday said it sold the peanut allergy treatment Palforzia to Swiss company Stallergenes Greer abandoning the therapy three years after acquiring it in a 26 billion deal
Sangamo which specializes in a gene editing technique called zinc fingers previously reprioritized its pipeline as it adjusted to losing development partners Novartis and Biogen