daniel-gordon/biopharma-dive huggingface dataset:
Agenus is now pinning its hopes on botensilimab and balstilimab which go after two well-known cancer immunotherapy targets CTLA4 and PD1 respectively
The company was formed as a limited liability company or LLC allowing it to house individual drug programs within subsidiaries sell them to wouldbe buyers and generate returns for investors while remaining private
OrbiMed is deeply appreciative of the continued support weve received from many longstanding partners whove invested in these funds said Carter Neild a managing partner of OrbiMed in a statement
Sana went public in early 2021  when investor appetite for emerging biotechs was still strong. The companys 588 million more than 700 million raised while Sana was still privately held giving it a particularly large valuation for a newly public biotech
AlveoGene  another gene therapy company launched Thursday with plans to develop a medicine to treat AATD lung disease
The FDA may not perceive the benefits  as ever outweighing potential safety risks of the drug  even with additional data Abrahams wrote in a note to clients Thursday. For Sage the rejection has proven to be a substantial setback
On Wednesday BioMarin said it now expects Roctavian to bring in less than 10 million in revenue this year down from a previous estimate of 50 million to 150 million
Hypomagnesemia was identified in patients taking SRP5051
Emily Whitehead who in 2012 became the first pediatric leukemia patient given the cancerfighting CART cell therapy Kymriah  has been in remission since her treatment
For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward looking statements as well as risks relating to Ascendis busi ness in general see Ascendis Annual Report on Form 20 F filed with the US Securities and Exchange Commission SEC on February 16 2023 and Ascendis other future reports filed with or submitted to the SEC
Tax amounts can vary substantially from period to period due to tax adjustments that are not directly related to underlying operating performance
Obesity drugs An obesity drug biotech gets buyin from Eli Lilly venture firms Startup OrsoBio raised 60 million in a Series A round co led by Longitude Capital and Enavate Sciences and joined by the pharmaʼs venture arm
Known risk factors include the following 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 productdemand 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 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 filed with the Securities and Exchange Commission SEC as well as other SEC filings made by the Company which you are encouraged to review
Stifel analyst Benjamin Burnett wrote in a note to clients that the initial data was not obviously better than competitors which dampened our appetite into combo readouts We believe that the company will emerge more aligned with current investor sentiment wrote Leerink Partners analyst Andrew Berens
Vertex told investors earlier this month that the FDA plans to hold an advisory committee meeting to review its option
It is designed to target and silence TTR messenger RNA thereby reducing the production of TTR protein before it is made
In December 2019  Roche partnered with Sarepta to combine Roches global reach commercial presence and regulatory expertise with Sareptas gene therapy candidate for Duchenne to accelerate access to SRP9001 for patients outside the United States
The company will continue testing VTX958 in Crohns disease for which it will conduct an interim efficacy analysis early next year
The current model which is daily pills and intermittent injections does not really work that well. The hope is that a medicine like VERVE101 can address that unmet need with a onetime therapy that produces deep and durable lowering of LDL Verve is testing the drug first in people with heterozygous familial hypercholesterolemia or HeFH an inherited condition that causes extremely high LDL levels and earlyonset atherosclerotic cardiovascular disease
The company is also testing the drug in uveitis and psoriatic arthritis and plans to start a Phase 3 trial in axial spondyloarthritis
Our deep understanding of biology and our ability to employ the technology and tools necessary to effectively explore and manipulate that biology are critical differentiators that we believe will ultimately enable us to develop therapeutics DCruz said in a statement
Apellis is now advising doctors to immediately stop using any kits with the 19 gauge needle and instead use an 18 gauge option thats also in distribution
The reorganization will strengthen our ability to invest in growth opportunities and support the companys longterm success said CEO Cedric Francois in a statement
The companys most advanced program is designed to stimulate two members of a protein family that control whether important neurological processes  gets released in the brain. Its an approach shared by Karuna Therapeutics which is developing a similar closely watched drug called KarXT
But the drug is used for severe forms of hypophosphatasia leaving patients with more moderate symptoms without any options approved by the Food and Drug Administration
The public markets are ultimately where biotechs have to go to fund largescale innovation but were really happy that were in a very strong position from a balance sheet perspective, Neumoras CFO said in an interview
May 2023 Ascendis Pharma AS
Conference Call Information Management will discuss the APOLLOB topline results via conference call on Wednesday August 3 2022 at 800 am ET
Viking is known for backing socalled crossover rounds  which often act as a bridge for private companies to prepare for an IPO
But last week the company said that it uncovered irregularities in some of the needles used to withdraw the medicine from vials before an injection
This condition is caused by a change in the FGFR3 gene a negative regulator of bone growth
The drug is currently only approved in the US but could by cleared in Europe in early 2024
For each of the six months ended June 30 2021 and 2020 the Company recorded amortization of inlicensed rights of approximately 03 million
The cell therapy Lantidra  approved by the Food and Drug Administration in June  is intended for Type 1 diabetes patients who have trouble managing blood sugar levels and have experienced repeated episodes of hypoglycemia
By one 2017 estimate  approximately onethird of medicines target the ubiquitous and multifunctional receptors in some way
Shares in Sanofi recovered slightly Monday rising by more than 3
Under the current deal Royalty will have rights to 81 of tiered royalties for Evrysdi up to a 13 billion total cap and 67 of royalties above the cap
QA Inside Bavarian Nordicʼs late stage stumbles  and drive to become a vaccine giant Despite the companyʼs recent Phase 3 setbacks in RSV and COVID19 CEO Paul Chaplin sees a path forward for the Danish biotech in the travel vaccine market
Receiving FTD for important aspects of the disease is a critical milestone that furthers our ability to accelerate the development of TSHA102 with the potential to address a serious condition and significant unmet medical need in patients living with this devastating disease
Previously VOXZOGO was indicated for children who were 5 years of age and older
The company has supported a number of approved drugs with its codevelopment strategy including Pfizers Besponsa Eisais Lenvima and Apellis Pharmaceuticals Empaveli. In the latter case Abingworth provided 28 million to help Apellis run a Phase 3 trial of the drug
The company is appealing the decision and expects to review a new opinion in January 2024. PTC shares fell by about 15 Friday to trade around 22 apiece
In December the company shook things up further replacing CEO RA Session II with current chief executive Sean Nolan  who had been serving as chairman of the board. Tayshas difficulties have come as other gene therapy developers have struggled to attract new funding and deliver on ambitious development goals
Actio is aiming at another form Type 2C that causes severe muscle weakness and respiratory complications
The CEO of Airna said the company is ﬁrst focusing on developing a drug for alpha1 antitrypsin deﬁciency Permission granted by AIRNA  Airna a biotechnology startup focused on RNA editing to make more precise medicines emerged from stealth Tuesday with 30 million in hand
Research and development Research and development expenses were 2396 million for the three months ended June 30 2021 compared to 1885 million for the same period of 2020 an increase of 511 million
BioMarin and VOXZOGO are registered trademarks of BioMarin Pharmaceutical Inc
ForwardLooking Statements This press release contains forwardlooking statements about the business prospects of BioMarin Pharmaceutical Inc including without limitation statements about i the development of BioMarins valoctocogene roxaparvovec program generally ii further regulatory interactions with the FDA valoctocogene roxaparvovec iii possible requirements for approval of valoctocogene roxaparvovec including the suitability of Study 270301 to meet such requirements iv the conduct of ongoing clinical trials for valoctocogene roxaparvovec v and the continued review of the Marketing Authorization Application for valoctocogene roxaparvovec by the European Medicines Agency
Since the acquisition Bluerock has operated as an independent subsidiary
For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward looking statements as well as risks relating to Ascendis business in general see Ascendis Annual Report on Form 20 F filed with the US Securities and Exchange Commission SEC on February 16 2023 and Ascendis other future reports filed with or submitted to the SEC
Developers see these treatments as working well for psoriasis and in other inflammatory conditions like psoriatic arthritis and Crohns disease  both indications Bristol Myers is currently studying Sotyku in
The study enrolled 360 adult patients with ATTR amyloidosis hereditary or wildtype with cardiomyopathy at 69 sites in 21 countries
All relevant nonGAAP measures are reconciled from their respective GAAP measures in the attached table Reconciliation of GAAP Financial Measures to NonGAAP Financial Measures About EXONDYS 51 EXONDYS 51 uses Sareptas proprietary phosphorodiamidate morpholino oligomer PMO chemistry and exonskipping technology to bind to exon 51 of dystrophin premRNA resulting in skipping of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping
Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51 5321 Results suggest a highly potent nextgeneration treatment that could offer greater efficacy with less frequent dosing SRP5051 dosed monthly at 30 mgkg delivered mean exon skipping of 1079 and mean dystrophin expression of 655 consistently higher than the other SRP5051 dosing cohorts at 12 weeks and weekly eteplirsen at 24 weeks Sareptas predictive model indicates that SRP5051 at 30 mgkg will achieve greater than 10 dystrophin with monthly chronic dosing CAMBRIDGE Mass May 03 2021 GLOBE NEWSWIRE  Sarepta Therapeutics Inc NASDAQSRPT the leader in precision genetic medicine for rare diseases today announced positive results from Part A of the MOMENTUM study Study 5051201 a global Phase 2 multiascending dose clinical trial of SRP5051 its nextgeneration peptide phosphorodiamidate morpholino oligomer PPMO treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping
We have combined our teams proven experience in gene therapy drug development and commercialization with the worldclass UT Southwestern Gene Therapy Program
Cargos lead program dubbed CRG022 is in Phase 2 clinical testing for patients with large Bcell lymphoma that is relapsed or refractory to existing CART therapies
Important Safety Information for AMONDYS 45 Kidney toxicity was observed in animals who received casimersen
The exterior of the Food And Drug Administration headquarters is seen on July 20 2020 in White Oak Maryland
Seagen would be responsible for conjugation and then advancing the drug candidates into preclinical and clinical research as well as commercialization
What has always surprised me is just how well the machine works meaning that selfreport works really well most people want to opt into research and most people when I recontacted them and they see the message they are interested in participating in research. So our ability to create this realtime research engine has really been extraordinary
As part of the bemdaneprocel study participants were asked to record when they were in on states meaning their symptoms were well controlled and off states when their symptoms seemed to worsen. According to Bluerock one year after treatment participants in the highdose group said they were experiencing a little over two hours more in the on state without troubling involuntary movements and just under two hours less in the off state
Stop administering VOXZOGO if instructed by your healthcare provider
In the meantime Biogen expects its revenue in 2023 to be lower than last years total  although not by as much as previously thought
Kapeller said the company's investors chose to stake the company with additional funding so it could show whether its experimental drug might work in humans
Some well-known drugs that target GPCRs include the diabetes drug Victoza and the multiple sclerosis treatment Gilenya
Access Biotechnology Atlas Venture and RA Capital Management incubated the company which in 2021 announced a Series A investment of $75 million
With the May 29 action date our top priority we will work collaboratively with the FDA to complete the review of our BLA for SRP 9001
As part of the deal announced Thursday  both companies retain options for a sale that would transfer even more of the rights to Royalty potentially bringing in as much as 500 million more for PTC
Any statements contained in this press release that are not statements of historical fact may be deemed to be forwardlooking statements
FDA cited concerns related to the manufacturing control strategy for variability of delivered dose in the TransCon PTH drugdevice combination product
For additional information please visit wwwbiomarincom102023 805 PM US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia  Oct 20  httpsinvestorsbiomarincom20231020USFoodandDrugAdministrationApprovesBioMarinsVOXZOGORvosoritideforChildrenUnder5Yearswith 911ForwardLooking Statements This press release contains forwardlooking statements about the business prospects of BioMarin Pharmaceutical Inc BioMarin including without limitation statements about the potential impact and role of VOXZOGO in children with achondroplasia including younger children as well as in the treatment of hypochondroplasia and other genetic short stature conditions the development of BioMarins VOXZOGO program including BioMarins expectations regarding timing and the ability to obtain final approval decision from the European Commission for the marketing authorization to expand the indication for VOXZOGO BioMarins plans for the continued clinical development of VOXZOGO including ability to maintain continued approval in the US based on ongoing labelextension studies and confirmatory trials BioMarins plans to initiate a pivotal development program in hypochondroplasia and BioMarins expectations regarding the number of children with achondroplasia that could be prescribed with VOXZOGO and related potential benefits
The company's goal is to provide innovative drug delivery solutions that are needed to raise standards of care Graves said in a statement
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 or result in an assessment that SRP9001 provides a safe or effective treatment benefit different methodologies or assumptions than 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 are positive the data may not be sufficient to support approval by the FDA or foreign regulatory authorities 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 many of which are outside of our control including possible limitations on 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 the impact of the 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 2019 and most recent Quarterly Report on Form 10Q filed with the Securities and Exchange Commission SEC as well as other SEC filings we make which you are encouraged to review
The agency also detailed its issues with how BrainStorm intends to manufacture NurOwn which is built from mesenchymal stem cells collected from each patient. These stem cells once injected into the cerebrospinal fluid are meant to secrete proteins that help neurons survive
TYK2 is part of a family of proteins known as Janus kinases or JAKs which help control the bodys immune regulation
Emerging biotech Abingworth raises 356M to invest in latestage drug development The ﬁrmʼs newest fund extends its codevelopment strategy of ﬁnancing broader study of medicines far along in testing or already on market
About Sarepta Therapeutics At Sarepta we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease
Nimbus handed some of the proceeds of the Gilead and Takeda deals to its investors and used the rest to fund research. The approach contrasts with that typically followed by most biotech startups which are often built for initial public offerings or acquisitions down the road. It appears to have resonated with investors at a time when startups are having difficulty raising money
For additional information please visit wwwbiomarincom
The conference call may be accessed by dialing 844 5347313 for domestic callers and 574 9901451 for international callers
Known risk factors include the following we may not be able to comply with all FDA postapproval commitments and requirements with respect to our products in a timely manner or at all 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 may fail to meet regulatory approval requirements for the safety and efficacy of product candidates if the actual number of patients suffering from the diseases we aim to treat 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 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 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 the COVID19 pandemic and 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 those risks identified under the heading Risk Factors in our most recent Annual Report on Form 10K for the year ended December 31 2020 and most recent Quarterly Report on 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
The FDA has pledged to take a flexible approach in its review of ALS drugs and recently approved medicines from Amylyx Pharmaceuticals and Biogen despite mixed results in support of their effectiveness
Jaxs models could give Actio the tools to make that jump which many drugmakers aspire to more reliably
MapLight Therapeutics a San Francisco-based biotechnology company has raised another 225 million in its pursuit to develop new drugs for brain disorders
NonGAAP selling general and administrative expenses are defined by the Company as GAAP selling general and administrative expenses excluding depreciation and amortization expense stockbased compensation expense and other items
Cell therapies are viewed as potential onetime treatments able to cure or at least reduce the burden of disease in chronic or fatal conditions
At the same time we have not fully understood the root cause of diabetes as a disease. The disease severity increases over time and weve not really found a key to stop disease progression to slow it down reverse or even prevent it
Since its founding 20 years ago Alnylam has led the RNAi Revolution and continues101723 633 AM Alnylam Pharmaceuticals Press Release  Aug 03 2022  Alnylam Reports Positive Topline Results from APOLLOB Phase 3 Study of Patisira httpsinvestorsalnylamcompressreleaseid26851 79to deliver on a bold vision to turn scientific possibility into reality