daniel-gordon/biopharma-dive huggingface dataset:
Alnylam has a deep pipeline of investigational medicines including multiple product candidates that are in latestage development
Predictive modeling for dystrophin accumulation that includes assumptions of known turnover of dystrophin in the muscle and an analysis of data generated with the PPMO platform indicates that SRP5051 at 30 mgkg is likely to deliver greater than 10 dystrophin over time with monthly dosing
nearly 5 billion drawing backers such as RA Capital Management Venrock and the Bill and Melinda Gates Foundation to fund a technology that can steadily deliver a precise amount of a drug into the body
The totality of data submitted  does not demonstrate substantial evidence of effectiveness Gumei Liu the agencys clinical reviewer said during a presentation
The companys fieldbased sales staff and medical employees are minimally affected by the reorganization which will be completed during the third quarter Apellis said
Many of these risks and uncertainties are beyond our control
Directors of the San Diego-based drugmaker approved a liquidation plan that will be up for a shareholder vote in the fourth quarter. In the meantime Histogen has stopped all clinical work and laid off almost all of its staff
Now Sana has a cell therapy called SC291 in Phase 1 testing for Bcell lymphomas and leukemias and expects to deliver data this year and next. Its also seeking regulators approval to test the treatment in patients with autoimmune diseases
Nestle gives up on peanut allergy treatment after sluggish sales The food and beverage company has sold oﬀ the FDA approved therapy Palforzia three years aer acquiring it in a 26 billion deal
To reduce the risk of a decrease in blood pressure and associated symptoms dizziness feeling tired or nausea patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO
Bristol Myers Squibb is acquiring an antibody-drug conjugate to treat bone and blood cancers from multinational biotechnology startup Orum Therapeutics the companies announced Monday
Genentechs deal with Orionis is not its first foray into targeted protein degradation. The company announced a collaboration in 2022 with Chinabased Jemincare to develop an experimental protein degrader for a target linked to prostate cancer, which poured 40 million into a collaboration with PeptiDream  a Japanese drugmaker working on radiopharmaceuticals
And very importantly Study 102 has provided us with a wealth of information and insight which we will use to refine and complete the protocol for our upcoming trial using commercial process material
The new combined company eventually gave up on the baldness drug researched emricasan as a treatment for COVID19 and tried at various times to refocus on a more promising path forward
The Food and Drug Administrations list of cleared or approved companion diagnostics includes more than 20 tissuebased tests for the HER2 biomarker alone
A second experimental drug dubbed NMRA511 is in early clinical evaluation as a potential treatment for agitation related to Alzheimers disease
Star Therapeutics adds another $90M to grow its galaxy of drug startups. The biotech has already formed two oﬀshoots and aims to start up more with the additional funding
We are excited to have chosen our target dose for further development
On Wednesday the company said its experimental flu shot succeeded in a Phase 3 trial although previous data have been mixed
The companys lead therapy AIO 001 is designed to block TSLP a protein linked to inflammation in AstraZenecas approved medicine Tezspire . Aiolos says its experimental treatment has unique properties that allow for a significantly extended halflife. The company plans to advance AIO001 into a Phase 2 trial for patients with moderatetosevere asthma after promising early results
In its regulatory filing  RayzeBio wrote that it sees an opportunity for innovative radiopharmaceutical therapeutics to transform how cancer is treated
Kenvue JJs consumer division also completed an IPO in May as part of a spinout. A handful of other companies are lining up to test the waters too offering a glimpse into what could be a busier fourth quarter
It plans to start a Phase 3 trial by the end of September and will soon begin a Phase 1 trial in bipolar depression as well
Under a revamped executive team and board of directors the company says its now diving deeper into research areas like immunology and rare diseases. To that end Biogen in September completed the 7 billion acquisition of Reata Pharmaceuticals  which gave it a newtomarket Friedreichs ataxia. That drug called Skyclarys is off to a strong start according to RBCs Abrahams
Nimbus wrapped the second last year when it sold its TYK2 medicine to Takeda. Those deals validated the unorthodox path pursued by Nimbus  which Atlas and Schrödinger launched during the global recession in 2009
Tevas was originally in development for asthma but the company stopped testing after disappointing results in a midstage study
ONPATTRO Indication and ISI Indication ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin mediated amyloidosis in adults
Novo Nordisk and AstraZeneca  meanwhile have both entered new collaborations in the space this year
Despite having four products on the market that earned it 638 million in the first half of 2023 the company still lost 450 million over that period
The data backing both Relyvrio and Qalsody were mixed with the latter drug having failed the main objective of the key trial that supported its approval
However manufacturing and supplying radioisotopes is a long and complex process. Earlier candidates struggled to take off after development and Novartis faced initial problems with supply
Claire Henchcliffe, one of the studys principal investigators and the chair of the neurology department of the University of California Irvine School of Medicine, called the data collected for bemdaneprocel extremely encouraging. While this is a small openlabel study meeting the studys primary objective for safety and tolerability along with initial improvements seen in clinical outcomes represents a great step forward Henchcliffe said in a statement from Bluerock
Sage Therapeutics reeling from a regulatory decision that will sharply curtail the potential of its newest medicine is slashing jobs pausing some earlystage research and reshuffling its executive team
In March the company picked up a portion of royalty rights to an experimental schizophrenia drug being developed by Karuna Therapeutics. And in August it bought royalties on Ferring Pharmaceuticals  Adstiladrin treatment for bladder cancer
But by August 2022 ProQR said it would seek a partner for its eye drugs and focus instead on the Axiomer RNA editing platform
The Food and Drug Administration is reviewing Orchard Therapeutics approval application for a gene therapy to treat the rare disease metachromatic leukodystrophy setting a decision deadline of March 18 2024 the company said Monday
November brings new flurry of biotech layoffs Seres Therapeutics Kronos Bio and Rani Therapeutics joined Sangamo in announcing job cuts while private biotech LocanaBio revealed itʼs shutting down
Forward  looking statements do not reflect t he potential impact of any future licensing collaborations acquisitions mergers dispositions joint ventures or investments that Ascendis may enter into or make
About SRP5051 SRP5051 uses Sareptas PPMO chemistry and exonskipping technology to skip exon 51 of the dystrophin gene
We want to be able to really derisk the biology for the target before we ever get into the clinic said Actios co-founder and CEO in an interview
With Shapes help Otsuka aims to develop adenoassociated virus based gene therapies that can deliver into eye cells the genetic code for molecules and antibodies its identified to treat chronic ocular diseases
These risks and uncertainties include among others results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec including the possible one year102023 651 PM BioMarin Receives Complete Response Letter CRL from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A  Au httpsinvestorsbiomarincom20200819BioMarinReceivesCompleteResponseLetterCRLfromFDAforValoctocogeneRoxaparvovecGeneTherapyforSe 56 extension of Study 270301 the content and timing of the expected follow up meeting with the FDA the content and timing of decisions by the FDA the European Commission and other regulatory authorities and those other risks detailed from time to time under the caption Risk Factors and elsewhere in BioMarins Securities and Exchange Commission SEC filings including BioMarins Quarterly Report on Form 10Q for the quarter ended June 30 2020 and future filings and reports by BioMarin
The data Verve plans to present next month will give an early indication of how well VERVE101 might work in people with of the US who received one of four different doses of VERVE101 and are the same as what Verve submitted to the FDA
Forwardlooking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forwardlooking statements
These risks and uncertainties include among others results and timing of current and planned preclinical studies and clinical trials of VOXZOGO any potential adverse events observed in the continuing monitoring of the patients in the clinical trials the content and timing of decisions by the European Commission and other regulatory authorities and those factors detailed in BioMarins filings with the Securities and Exchange Commission SEC including without limitation the factors contained under the caption Risk Factors in BioMarins Quarterly Report on Form 10Q for the quarter ended June 30 2023 as such factors may be updated by any subsequent reports
Rett syndrome caused by a pathogeniclikely pathogenic MECP2 mutation is estimated to affect between 15000 and 20000 patients in the US EU and UK
These forwardlooking statements include statements regarding market opportunities the potential benefits of PPMO including offering greater efficacy with less frequent dosing being a step order improvement over our current PMO platform and profoundly impacting the course of Duchenne our prediction that SRP5051 at 30 mgkg will achieve greater than 10 dystrophin over time with monthly dosing and our plans including to work with great urgency to discuss the results with regulatory agencies and gain their insights including the development path to support an accelerated approval of SRP5051 in the United States and to present full results at a future medical meeting
Although these are recurring charges to operations the Company believes the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within the Companys control
Verve Therapeutics plans to soon start testing a gene editing therapy for heart disease in the US after federal regulators lifted an order that had blocked the biotechnology company from enrolling people into its study
Taysha is a little more than a year on from a business reorganization in which it trimmed its research pipeline and laid off approximately onethird of its workforce
Secondary endpoints include certain timed functional tests micro dystrophin expression measured by immunofluorescence IF fiber intensity and microdystrophin expression measured by IF percent dystrophin positive fibers
JJ will retain rights to aprocitentan for pulmonary hypertension
The restructuring will reduce Sangamos annual operating expenses by about half to between 115 million to 135 million from approximately 240 million to 260 million previously
Monitor patients during the infusion for signs and symptoms of IRRs
Companies are now increasingly attracting investors and partners by attempting more targeted adjustments in DNA using technologies such as base editing, prime editing, and RNA editing
With only a few hundred patients eligible for Qalsody Viehbacher said the drug is not going to by any means move the needle commercially Sales expectations for Zurzuvae have diminished too since the FDA decided against approving it in major depressive disorder  a far more common condition than postpartum depression
Females and Males of Reproductive Potential
Selling general and administrative expenses were approximately 1435 million for the six months ended June 30 2021 compared to 1565 million for the same period in 2020 a decrease of 130 million
Based on the companys headcount of 689 fulltime workers as of Feb 8  its planned reduction could become one of the largest layoff rounds within the biotech sector this year and follows a major restructuring in April 2020
A nonsignificant result pvalue 00574 was found on the secondary composite endpoint of allcause mortality frequency of cardiovascular events and change from baseline in 6MWT over 12 months compared to placebo
This indication is approved under accelerated approval based on an102023 805 PM US Food and Drug Administration Approves BioMarins VOXZOGO vosoritide for Children Under 5 Years with Achondroplasia  Oct 20  httpsinvestorsbiomarincom20231020USFoodandDrugAdministrationApprovesBioMarinsVOXZOGORvosoritideforChildrenUnder5Yearswith 511improvement in annualized growth velocity
In addition to Cormorant Fidelity and Goldman Sachs, the Series B round received investment from Logos Capital Perceptive Advisors Sofinnova Investments Surveyor Capital and funds and accounts advised by T Rowe Price
In addition we expect to enroll the first patient in a German early access program  if approved during the second quarter of 2023
Only urine expected to be free of excreted AMONDYS 45 should be used for monitoring of urine protein
The company develops and commercializes targeted therapies that address the root cause of the genetic conditions
UniQure will still develop drugs for forms of epilepsy ALS and Fabry disease
BioMarin conducted a randomized doubleblind placebocontrolled Phase 2 clinical trial evaluating the safety and efficacy of VOXZOGO in children aged 5 and under Study 111206
We are grateful for the collaboration of the achondroplasia community physicians and the children and their families who have played a crucial role in advancing this clinical program
Comparisons between baseline and posttreatment measures were statistically significant p0001 for positive fibers and p0002 for intensity
The path to regulators was not straightforward however. Intercept reported positive Phase 3 data in 2019 but the FDA asked for longerterm data rejecting Intercepts first NASH application
Shape uses high throughput screening to parse a vast array of possible adenoassociated viruses or AAVs that could work as a delivery chassis for payloads like those Otsuka has in mind. It then applies machine learning to select the AAVs besttargeted to specific tissues and less likely to stray from their cellular destination.
Primary efficacy endpoints are patient assessments by clinicians using the Clinical Global Impressions Scale  Improvement CGII Rett Syndrome Hand Function Scale and Revised Motor Behavior Assessment RMBA
The company raised 540 million in May and the IPO was seen as a potential bellwether for a market that has been lagging
Among Bioluminescences current investments are Ensoma  a cell and gene therapy company backed by Takeda and Nido Biosciences  which raised more than 100 million ahead of its emergence from stealth earlier this year
Please visit ascendispharmacom to learn more
Selling general and administration Selling general and administrative expenses were approximately 723 million for the three months ended June 30 2021 compared to 737 million for the same period in 2020 a decrease of 14 million
SRP9001 is an investigational gene transfer therapy intended to deliver its microdystrophinencoding gene to muscle tissue for the targeted production of the microdystrophin protein
After decades of research cell therapies have become a reality with six immune cell therapies now available in blood cancers and a pancreatic cell replacement therapy recently approved for Type 1 diabetes
Initial public offerings for drugmakers have remained slow with only 18 biotechs making their public debut
These are not all the possible side effects of VOXZOGO
Even more schizophrenia patients only get a partial benefit from existing medicines or cant handle them because of side effects. Doctors are eager for alternative treatments and appear to view the KarXT data favorably according to RBCs Abrahams. He expects the medicine to win approval and bring in peak sales of 12 billion
The variability of amounts and lack of predictability of collaboration and licenserelated upfront and milestone payment makes the identification of trends in the Companys ongoing research and development activities more difficult
The FDAs complete response letter didn't identify any other issues regarding clinical safety study conduct drug quality or manufacturing the biotechnology company said
Bavarian pivoted in 2019 toward building a more traditional commercial tickborne encephalitis and dedicating its RD efforts to infectious disease and cancer vaccines. But the transition has been bumpy, in July the company dropped out of the respiratory syncytial virus vaccine race after a Phase 3 trial failed to meet its main goal
RayzeBio Neumora price some of biotechʼs largest IPOs this year Both companiesʼ oﬀerings brought in signiﬁcant funds with RayzeBio raising 311 million and Neumora getting 250M
More information is available at wwwtayshagtxcom
These patients have limited treatment options and disease progression is common
The company announced plans to develop ORM6151 in late 2022 as the second experimental drug of this type in its pipeline
Its treatments Ozempic and Wegovy are now household names thanks to their potent weight loss effects and surging use
The savings will help extend the cash runway for the Seattle based company further into 2025 according to a Tuesday statement. Sana will also postpone a planned regulatory filing seeking permission for human studies of a cell therapy for a type of blood cancer
Instead Taysha chose to focus on research into Rett an inherited neurological disorder that affects girls and another central nervous system condition called general axonal neuropathy
Serum cystatin C urine dipstick and urine proteintocreatinine ratio should be measured before starting AMONDYS 45
Therefore the Company believes that excluding these charges facilitates comparisons of the Companys operational performance in different periods
Alnylam explicitly disclaims any obligation except to the extent required by law to update any forwardlooking statements
There are approximately 800 children under 5 with achondroplasia in the US VOXZOGO is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates
Rampart believes its approach can address both delivery and the issue of redosability which may be necessary as hypophosphatasia progresses according to the company
Study 102 reinforces our confidence in the potentially transformative benefits of SRP9001 including among other things the fact that in the Studys prespecified analysis the participants in the 45 age group robustly achieved a statistically significant and clinically meaningful improvement in NSAA over placebo as predicted by our prior Study 101
During treatment monitor urine dipstick every month and serum cystatin C and urine proteintocreatinine ratio every three months
Orionis headquartered in Boston with research facilities in Ghent Belgium has a handful of preclinical cancer drug programs in its pipeline
Approvals in Canada Australia the UK and Switzerland could come next year as well
BioMarin has recently been able to secure increased fillfinish commitments in 2024 and beyond to meet this additional demand
As part of the new collaboration Alexion will use Verges technology to select novel targets with highpotential for other indications
They also have partnered on an antisense drug Roche is testing in kidney disease and Ionis is studying as a treatment for an age related eye disorder
Xphozah works by blocking absorption in a different way. Importantly it is taken twice daily as a single tablet compared to the more frequent dosing with larger pills required for existing phosphate binders
The Company excludes upfront and milestone payments associated with its license and collaboration agreements from its nonGAAP results and research and development expenses because the Company does not consider them to be normal operating expenses due to their nature variability of amounts and lack of predictability as to occurrence andor timing
And Lilly in June announced plans to buy cell therapy developer Sigilon Therapeutics after working with the company on type 1 diabetes treatments
Significantly and representing years of work and enormous investment into process and analytical development for SRP9001 our gene therapy for Duchenne muscular dystrophy we announced very positive results from our first cohort of Study SRP9001103 ENDEAVOR to test the performance of our commercially representative material
But the company used a rare tactic in drug regulations  a file over protest  to force an evaluation. The agency subsequently said it would have an approval verdict by Dec 8
Researchers believe searching those elements for clues to how disease develops could unlock new types of medicines
This class of medicines commonly link a targeting antibody to a tumor killing toxin guiding the potent drug to tumors while sparing healthy tissue. Rather than a toxin Orum is using a small molecule designed to degrade target proteins
In 2014 a National Institutes of Health Phase 2 study of Ocaliva in people with NASH was stopped early because it had met its primary goal  improvement in disease activity score  at an interim analysis
Study 102 remains blinded and we will analyze the functional results for all patients including crossover participants once they have achieved the 48week timepoint in Part 2
Apellis shares shot up 36% in early trading Wednesday amid optimism that there may be an answer to the safety issues unconnected to Syfovre itself
Current executives Amy Pooler and Gregory Davis will be appointed head of research and head of technology respectively Shares in Sangamo fell by more than 15 Thursday morning
Late last month Beam Therapeutics which held more than 1 billion in cash at the end of June said it would cut 100 jobs to focus its resources on its most promising projects
Major depressive disorder  which affects tens of millions of people in the US by some estimates  was central to the Wall Street models that had pegged Zurzuvae as a blockbuster product
Ardelyx did not disclose the price it plans to charge for Xphozah indicating it would do so during an upcoming earnings call
When Rapport with offices in Boston and San Diego launched in March with 100 million from a Series A financing round its newly appointed CEO said the money would help to get that lead program to the point where it had proofofconcept data
But the market for genetic medicine companies and biotechs more broadly has soured over the past year narrowing companies funding options and forcing difficult decisions about which research to prioritize
SRP9001 is an investigational gene transfer therapy being developed for the treatment of Duchenne muscular dystrophy
To make and administer the therapy is laborious first requiring a referral from a hematologist. If the patient is eligible their cells are collected and shipped to a manufacturing facility where theyre genetically edited to express a form of an essential protein called hemoglobin. The cells are then shipped back to a treatment facility that infuses them into the patients bone marrow
Tell your doctor about all of the medicines the patient takes including prescription and overthecounter medicines vitamins and herbal supplements
If the patient is pregnant or plans to become pregnant
VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates epiphyses