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Important Safety Information InfusionRelated Reactions Infusionrelated reactions IRRs have been observed in patients treated with ONPATTRO

About BioMarin BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and lifethreatening rare and ultrarare genetic diseases

The European Medicines Agency EMA review of the Companys Marketing Authorization Application MAA for valoctocogene roxaparvovec is ongoing

The idea of fundamentally altering diabetes at the cellular level has drawn interest from companies big and small

Cargos focus is on CART therapies a type of cellular treatment for several kinds of blood cancers

These other items include collaboration revenue and transaction cost related to the Roche transaction upfront and milestone payments acquired inprocess research and development expense gain from sale of PRV and loss on contingent consideration

The company is based in Cambridge Massachusetts with research facilities in Tübingen Germany

The FDA issues a CRL to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form102023 651 PM BioMarin Receives Complete Response Letter CRL from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A Au httpsinvestorsbiomarincom20200819BioMarinReceivesCompleteResponseLetterCRLfromFDAforValoctocogeneRoxaparvovecGeneTherapyforSe 26Having previously agreed with the Agency on the extent of data necessary to support the BLA the FDA introduced a new recommendation for two years of data from the Companys ongoing 270301 study Phase 3 to provide substantial evidence of a durable effect using Annualized Bleeding Rate ABR as the primary endpoint

The Companys portfolio consists of six commercialized products and multiple clinical and preclinical product candidates

Ardelyx which has been developing the therapy for more than a decade expects to make Xphozah available in November and is hiring new sales staff to support the drugs launch in the US

SRP9001 is Sareptas investigational gene therapy for the treatment of Duchenne muscular dystrophy

Concentra m akes bi d for struggling bi otech Rain The proposal from Concentra which earlier this year bought Jounce Therapeutics offers to buy Rain for 125 per share plus a contingent value right

Seraxis a cell therapy company aiming to cure insulin dependent diabetes closed a second round of venture capital financing bringing the total equity investment in the company to more than 50 million

The cancerfocused biotech has struggled to maintain its footing this year following a clinical trial setback

Bioluminescence joins a class of new venture funds that have emerged in 2023 including Cure Ventures and Dimension

Reduced Serum Vitamin A Levels and Recommended Supplementation ONPATTRO treatment leads to a decrease in serum vitamin A levels

The model combines artificial intelligence and insights into an epigeneticbased biological program that is shared by most cancers. Earlier this year Harbinger found its test has 82 overall sensitivity of cancer detection at 95 specificity in a study of 1046 subjects. Most or 621 of the subjects had newly diagnosed treatmentnaive cancer

Exon skipping is intended to allow for production of an internally truncated dystrophin protein

Everything is cyclical Harasaki who was most recently part of Microsofts venture arm M12 previously worked as an investor at Andreessen Horowitz and in various roles at Novartis and Baxalta

There was no such expense recognized during the same period of 2020

An unusually high response rate among patients on placebo combined with dropouts in the group receiving izokibep impacted the results Acelyrin said

Companies like Triveni are trying to move other inflammatory disease drugs in the same direction

This indication is approved under accelerated approval based on an improvement in annualized growth velocity

FDA denies expanded approval for Alnylam RNA drug. The agency rejected Alnylamʼs application for approval of its medicine patisiran in people with a rare heart condition setting back the companyʼs plans

As part of the plan announced Monday, Histogen will delist its shares on the Nasdaq stock exchange seek to sell assets and distribute any remaining cash to stockholders after paying debts and obligations. The company will also continue looking for buyers for pipeline assets which may produce more cash for investors

Words such as believes anticipates plans expects will intends potential possible and similar expressions are intended to identify forwardlooking statements

For a detailed description of risks and uncertainties Sarepta faces you are encouraged to review the SEC filings made by Sarepta

VOXZOGO is approved in the US and indicated to increase linear growth in children with achondroplasia with open epiphyses

Previously Sage indicated that studies of its drug had not shown signs of suicidal thoughts or behaviors

General and Administrative Expenses General and administrative expenses were 60 million for the three months ended June 30 2023 compared to 99 million for the three months ended June 30 2022

ALS drug development Forbion-backed biotech raises $138M to develop ALS drug. Three-yearold startup VectorY plans to use the Series A funding to advance a vectorized antibody for ALS into clinical testing

Actual results or events could differ materially from the plans intentions expectations and projections disclosed in the forward looking statements

Beam is at least the 11th gene therapy or gene editing company to lay off staff in 2023 joining others like Editas Medicine, Graphite Bio, Sangamo Therapeutics, and UniQure

Sarepta Therapeutics Announces Positive Vote from US FDA Advisory Committee Meeting for SRP9001 Gene Therapy to Treat Duchenne Muscular Dystrophy 51223 Advisory committee voted 86 in support of accelerated approval of SRP9001 Regulatory action date is May 29 2023 CAMBRIDGE Mass

Leonid Sorokin via Getty Images Ventyx Biosciences on Monday said it will stop testing an experimental inflammatory disease drug in two conditions after results from a midstage study fell short of its expectations

Last week the Food and Drug Administration lifted a regulatory hold that had blocked Verve from enrolling patients in the US into a Phase 1 trial of the medicine

Its pursuing that strategy in response to the fatigue investors have had with experimental cancer drug combinations many of which broke hearts and broke wallets Keiper said

2. They join a list of more than 130 biotechs that have laid off employees this year as the sector struggles with a shaky stock market and diminished funding prospects

Launched in 2015 Verge has raised over 100 million from a variety of investors including the asset management firm Blackrock and large pharmaceutical companies like Eli Lilly and Merck Co. Rather than follow the typical path for identifying new drug targets which often involves animal or cell models Verge uses machine learning to analyze genomic data from human tissue samples

About Sarepta Therapeutics Sarepta is on an urgent mission engineer precision genetic medicine for rare diseases that devastate lives and cut futures short

Contacts Investors Media Traci McCarty Marni Kottle BioMarin Pharmaceutical IncBioMarin Pharmaceutical Inc 415 4557558 415 2187111 SOURCE BioMarin Pharmaceutical Inc102023 715 PM US Food and Drug Administration Approves BioMarins ROCTAVIAN valoctocogene roxaparvovecrvox the First and Only Gene Thera httpsinvestorsbiomarincom20230629USFoodandDrugAdministrationApprovesBioMarinsROCTAVIANTMvaloctocogeneroxaparvovecrvoxthe 1111Our Motivation Our Treatments Our Company Investors MediaOur 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

Aer 12 months all patients will receive patisiran in an openlabel extension period

Additionally people with the most severe form of hemophilia A FVIII levels 1 often experience painful spontaneous bleeds into their muscles or joints

The 37 million decrease was due to lower compensation expense as a result of reduced headcount and fewer manufacturing batches and raw material purchases

The startup incubated by Flagship Pioneering recently brought an experimental monoclonal antibody to treat COVID19 into clinical testing. Dubbed GB0669, the drug is the first of Generates programs to enter the clinic

In Europe as expected Ascendis has received the comprehensive Day 120 response from the European Medicines Agency

The company had previously said it was weighing a sale or partnership of the drug and Raffat noted there was high level of interest in a deal

While similar to achondroplasia people with hypochondroplasia typically present with milder disproportionality and less severe short stature compared to achondroplasia

Novartis still stands to receive additional milestone payments and if the drug is approved sales royalties

Two patients experienced serious adverse events transaminase elevation in one patient and nausea and vomiting in a second patient that fully resolved

More broadly neuroscience , viewed for years as a highrisk research area by many of the worlds leading drugmakers, has the startup Neumora Therapeutics for example has raised more than 600 million from a deep bench of investors that includes Amgen Arch Venture Partners and other venture firms

However the split came as a biotech bull market fizzled constraining many companies ability to raise new funds

But sepofarsen failed in a latestage clinical trial in February 2022 sending shares tumbling

Two patients have severe vision impairment which is unlikely to be resolved and the other three patients are still being evaluated Apellis said

By comparison in 2021 60 of biotechs that went public were in preclinical or Phase 1 testing

Words such as believe anticipate plan expect will may intend prepare look potential possible and similar expressions are intended to identify forwardlooking statements

Cash Cash Equivalents Investments and Restricted Cash and Investments The Company had approximately 17 billion in cash cash equivalents and investments as of June 30 2021 compared to 19 billion as of December 31 2020

Harbinger plans to launch a laboratory developed test for early cancer detection in 2025. Flagship Pioneering joined with new Harbinger investors including Pictet Partners Investment and Catalyst to provide money to execute the strategy

FDA documents hint at ʻuphill battleʼ for broad approval of Sageʼs depression drug New information from the agencyʼs review show it had concerns about the side effects experienced by people with major depressive disorder who took Sageʼs drug Zurzuvae

We look forward to expanding the clinical evaluation to earlier stages of disease progression following recent FDA clearance to initiate clinical development of TSHA102 in pediatric patients in the United States Rumana HaqueAhmed Senior Vice President Regulatory Affairs of Taysha added Rett syndrome is a devastating neurodevelopmental disorder that can lead to motor and respiratory impairment loss of communication and ultimately shortened life expectancy

On a GAAP basis for the six months ended June 30 2021 the Company reported a net loss of 2487 million or 312 per basic and diluted share compared to a net loss of 1683 million reported for the same period of 2020 or 218 per basic and diluted share

EXONDYS 51 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental

Other items The Company evaluates other items of expense and income on an individual basis

It then put the drug into Phase 2 testing for inflammatory bowel disease just before Prometheus and Pfizers drugs attracted attention for promising early data. Pfizer later set up a new company with Roivant handing away majority rights

Lilly buys out Beam in heart drug alliance with Verve Already a partner to Verve the pharma is paying 200 million upfront and investing another 50 million in Beam to acquire option rights to several gene editing programs

Internet Posting of Information We routinely post information that may be important to investors in the For Investors section of our website at wwwsareptacom

VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping

Early entrants struggled to find a market niche but Novartis has had greater success with its radiopharmaceutical drugs Lutathera and Pluvicto respectively approved for neuroendocrine and prostate cancers

The companies plan to do this by using Verges technology which uses both AI and human tissue data to predict what drug targets are most likely to succeed in clinical testing

The company completed its application to the Food and Drug Administration in midJuly requesting the agency assess the drug under priority review. If granted the expedited process would shorten the FDAs review window from 10 months to six

The Series A funding Actio announced Tuesday is meant to get the TRPV4 program into the first stages of clinical testing and make meaningful progress on at least one other program

The company at first made plans to shed staff and overhaul research as it tried to find a way forward for sepofarsen while developing ultevursen

The application was based on the Phase 3 study interim analysis of study participants treated with investigational product manufactured by the tobecommercialized process and threeyear data from the Phase 12 Study

Priya Singhal Biogens, head of development who has been serving as interim head of research told investors in April that the company is looking at every program in great depth several times over and thinking about what are the options what are the operational and strategic and regulatory challenges but also the opportunities Soon Grogan will have substantial influence over what research programs Biogen funds

According to Cantors Duncan around 47 and 72 of participants in that key Ingrezza study reported depression or suicidal ideation respectively

ElevateBio, which in May raised 401 million in one of biotechs largest private financings this year is cutting 13 of its workforce Fierce Biotech reported Oct 26

In earnings released Wednesday the Cambridge Massachusetts based drugmaker said it recorded 253 billion in revenue between July and September, up 1 from the same three-month period a year prior. Whether the growth increases or even continues depends on how well the companys newer products perform

Alnylams commercial RNAi therapeutic products are ONPATTRO patisiran GIVLAARI givosiran OXLUMO lumasiran AMVUTTRA vutrisiran and Leqvio inclisiran being developed and comm ercialized by Alnylams partner Novartis

Abingworth targets for clinical codevelopment drugs that are either in or through Phase 3 testing with a high probability of reaching approval and successful commercialization within a few years according to the companys website

Although it is passed down from parents to102023 651 PM BioMarin Receives Complete Response Letter CRL from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A Au httpsinvestorsbiomarincom20200819BioMarinReceivesCompleteResponseLetterCRLfromFDAforValoctocogeneRoxaparvovecGeneTherapyforSe 46children about 13 of cases are caused by a spontaneous mutation a new mutation that was not inherited

Critically Karunas submission came before Oct 1 when hard line Republicans may force a government shutdown that prevents the FDA from accepting new applications. By acting now Karuna should get an answer from regulators around this time next year RBC Capital Markets analyst Brian Abrahams wrote in a note to clients

While the company had no clinical data to offer investors in 2021 it had lofty ambitions with a pipeline of preclinical candidates based on engineered cells

It is used to monitor the progression of the disease and treatment effects which makes it suitable as an endpoint in clinical trials for Duchenne

Important Safety Information About EXONDYS 51 Hypersensitivity reactions including rash and urticaria pyrexia flushing cough dyspnea bronchospasm and hypotension have occurred in patients who were treated with EXONDYS 51

Source Sarepta Therapeutics Inc Investor Contact Ian Estepan 6172744052 iestepansareptacom Media Contact Tracy Sorrentino 6173018566 tsorrentinosareptacom

2Seventy isnt an outlier in making the decision to retrench. Many other small and midsized drugmakers have cut back this year in response to funding challenges and a difficult market

To accomplish all of this Moderna said it expects to invest 25 billion on research and development from 2024 through 2028 a sizable step up from the 13 billion to 32 billion its spent annually on RD the past few years

The FDA was concerned with the drugs mechanism . An oral medication FTX6058 is meant to spur production of fetal making shortly after birth. Boosting levels of fetal hemoglobin is associated with a reduction in the sickling of red blood cells

Going forward the company will only pursue high potential opportunities in retinal and central nervous system diseases

You have to find ways that healthcare can scale. Other industries all scale because they have found out how to do that without having to have a onetoone interaction. So we have been able to deliver genetic data now to 14 million people and none of those customers had precounseling or post counseling

Since its inception in 2019 the company has raised 125 million in private financing including a 40 million seed round from OrbiMed

FDA leadership has noted publicly that FDA is interested in exploring the use of surrogate endpoints biomarkers and innovative approaches like accelerated approval to advance cell and gene therapies particularly for rare lifeending degenerative diseases

Their pricings could indicate a coming resurgence according to Jordan Saxe, head of healthcare listings for the Nasdaq stock exchange. This weeks listings indicate that the appetite to invest in innovative healthcare companies is back and the pipeline of biotech companies looking to tap the public markets is continuing to grow Saxe said

If the infusion is interrupted consider resuming at a slower infusion rate only if symptoms have resolved

PTC to sell spinal muscular atrophy drug royalties for 1B A second deal with Royalty Pharma will allow PTC to retire Blackstone debt and keep more cash for future operations

About BioMarin Founded in 1997 BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery

A panel of experts earlier this year voted to raise the approval standards for the medicines at least when considering broader use in lymphomas and leukemias

RayzeBio also raised more funds in its stock sale than it had initially projected a positive indicator for a queue of other young biotechs attempting to go public. The San Diegobased company is developing an experimental radiopharmaceutical drug called RYZ101 for neuroendocrine tumors. . The therapy which is aimed at the same target as Novartis approved medicine Lutathera entered Phase 3 testing in May

Its backed by clinical trial data showing it can help people with the condition live longer and avoid heart related hospitalizations

Unlike CRISPRbased therapies RNA editing can target specific sites in an RNA transcript without permanently changing a patients genome