Tag: Novartis (NVS)

The U.S. FDA Approved Novartis Product Fabhalta® for the Treatment of Paroxysmal Nocturnal Hemoglobinuria

Novartis Approval Novartis (NVS) product Fabhalta (iptacopan) is a Factor B inhibitor that acts in the proximate alternative complement pathway of the immune system, It provides comprehensive control of red blood cell (RBC) destruction within and outside the blood vessels (intra- and extravascular hemolysis.   Fabhalta treatment of paroxysmal nocturnal hemoglobinuria (PNH) increased hemoglobin levels (≥ 2 g . . . This content is for paid …

Important News from Novartis MS Product Kesimpta®

From Novartis on April 20, 2023 The ALITHIOS open-label extension study demonstrated that continuous treatment with Kesimpta® (ofatumumab) for up to five years in relapsing multiple sclerosis (RMS) was associated with reduced risk of disability progression versus those who switched later from teriflunomide to Kesimpta. Outcomes related to both disability progression and brain volume change up to five years favored earlier initiation of Kesimpta in people …

23andMe Teams Up with Novartis on Lipoprotein(a) Education for its Customers

23andMe Aims To Raise Awareness of Lipoprotein(a), a Little-Known Genetic Risk Factor for Cardiovascular Disease March 24, 2023 (GLOBE NEWSWIRE) — In support of Lipoprotein(a) Awareness Day, 23andMe Holding Co. (ME) announced a collaboration with Novartis Pharmaceuticals Corporation (NVS) to increase awareness for Lipoprotein(a) (Lp(a)). The Information to Learn High levels of Lp(a) are associated with increased risk of heart attack, stroke and other life …

Alnylam Pharmaceuticals: Positive New Results of Lumasiran in Advanced Primary Hyperoxaluria Type 1

Alnylam Pharmaceuticals Alnylam Pharmaceuticals (ALNY) led the translation of RNA interference (RNAi) into a new class of innovative therapeutics, transforming the lives of people afflicted with rare and prevalent diseases with unmet needs. RNAi is a natural cellular process of gene silencing that represents  promising and rapidly advancing frontiers in biology and drug . . . This content is for paid subscribers. Please click here to …

The Omicron Variant and the Bizarre Stock Market Chaos

The Bizarre Assessment of Biotech Firms in the Stock Market The Clinical-Stage Biotechnology Firms  We are witnessing inexplicable evaluations of clinical-stage biotechnology companies. Bizarre is the best description of this group’s assessment which is based on incomes that do not exist in biotech firms that are yet to have approved and marketed products anywhere in the world. Unfortunately, the most impacted firms by the bizarre …

Novartis Announces Lift of Partial Clinical Trial Hold. See Also: Plans to Initiate a New SMA Study

Novartis Partial Clinical Trial Hold for OAV-101 IT for SMA Lifted Novartis (NVS) announced that the U.S. FDA has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. The decision to lift the hold was based on data from . . . This content is for paid subscribers. …

Novartis Announced Positive Results of Phase 3 Study Using 177Lu-PSMA-617 in Advanced Prostate Cancer Patients

Novartis Positive Results for Radioligand Therapy, 177Lu-PSMA-617, for mCRPC Novartis (NVS) reported the first interpretable results of evaluating the efficacy and safety of 177Lu-PSMA-617, a targeted radioligand therapy, in patients with progressive PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) compared to the best standard of care alone. The good news is that the trial has met both primary endpoints of overall survival (OS) and radiographic progression-free …

Regenxbio: Advancing its Gene Therapy Pipeline and its Rare Neurodegenerative Disease Platform with Non-Dilutive Capital

Regenxbio Regenxbio (RGNX) announced that it is selling a portion of its royalty rights from the net sales of Zolgensma® by Novartis to entities managed by Healthcare Royalty Management, LLC (HCR) for $200 million. This transaction provides immediate, non-dilutive capital to Regenxbio in order continue the development of its gene therapy pipeline products and complete its internal manufacturing capabilities. Morgan Stanley & Co. LLC served …

What We Expect to Hear at the Regenxbio Conference Call Today

Regenxbio Conference Call Information Regenxbio will host a conference call today, at 4:30 pm est today, November 4, 2020, to discuss its financial results for the quarter, ended September 30, 2020, and recent operational highlights. To access the live call by phone dial (855) 422-8964 (domestic) or (210) 229-8819 (international) and enter the passcode 4107819. To access a live or recorded webcast of the call …

Why We Expect a Lot from the Following Biotech Firms

Compugen Compugen (CGEN) announced the presentation of new research data that further support PVRIG as a potentially promising target for cancer immunotherapy. These data suggest that PVRIG inhibition may enhance T cell priming and infiltration into tumors and provide further evidence supporting the potential advantages of targeting PVRIG alone and in combination with TIGIT and PD-1 inhibitors, in tumors . . . This content is for …

A New Gene Therapy Series Starts Today with Regenxbio

Beyond the firms that are speeding towards bringing prophylactic vaccines against the COVID-19 infection into the clinic we have to consider the other firms that have been negatively affected by the virus pandemic; we expect them to rebound and surge when the virus loses its ability to kill. We decided to start with gene therapy companies that we believe have suffered from the delays of …
Regenxbio: Promising Results from RGX-121 Gene Therapy for Mucopolysaccharidosis Type II (MPS II)

Regenxbio: Promising Results from RGX-121 Gene Therapy for Mucopolysaccharidosis Type II (MPS II)

Regenxbio Results from RGX-121 for MPSII Regenxbio (RGNX) announced interim data from Phase I/II trial of RGX-121 for Mucopolysaccharidosis Type II (MPS II). RGX-121 is designed to deliver the gene that encodes iduronate-2-sulfatase (I2S) enzyme directly to the central nervous system (CNS) using the AAV9 vector. About Mucopolysaccharidosis Type . . . This content is for paid subscribers. Please click here to subscribe or here to log in.
Novartis to Acquire The Medicines Company at $85 a Share

Novartis to Acquire The Medicines Company at $85 a Share

Novartis to Acquire The Medicines Company Novartis (NVS) decided to buy The Medicines Company (MDCO) at $6.8 million. The Medicines Company’s shareholders will be getting $85 per share in cash  – a 45% premium to its unaffected share price and a 57% premium to its unaffected one-week average price. The Transaction is expected to be completed in the first quarter of 2020 The Medicines Company …
PTC Therapeutics’ Product Risdiplam Met Trial Endpoints for the Treatment of Spinal Muscular Atrophy

PTC Therapeutics’ Product Risdiplam Met Trial Endpoints for the Treatment of Spinal Muscular Atrophy

PTC Therapeutics Risdiplam Results for SMA PTC Therapeutics (PTCT) has positive results from part 2 of the SUNFISH trial evaluating the safety and efficacy of the product risdiplam. The results demonstrate that the trial has met its primary endpoint of change from baseline after 1 year of treatment with risdiplam compared to placebo. The change was measured by the Motor . . . This content …
When Negative Reactions to Biotechnology News Mislead. The Case of Regenxbio.

When Negative Reactions to Biotechnology News Mislead. The Case of Regenxbio.

Regenxbio Misleading Selloff A selloff of Regenxbio (RGNX) stock, following the FDA’s partial hold on the intrathecal administration of AVXS-101, is unwarranted and totally misleading.   Why? Because the selloff of RGNX gave the impression that the gene therapy Zolgensma, using the adeno-associated virus (AAV) vector , developed by Regenxbio, has failed, which is not true. The partial hold was put only on the product …
This Past Week

This Past Week

The Week in Review #43 This Past Week After hearing hints about the trade war between China and the United States being avoided the market reversed course from faltering to flying at the speed of light. This trade war replaced all important factors that would usually affect the market performances. Unemployment is the lowest since 1969 and is expected to cause a bullish market, but …
RegenxBio is Rebounding. See Also: A Scientifically Valuable Agreement Between CRISPR Therapeutics and KSQ Therapeutics

RegenxBio is Rebounding. See Also: A Scientifically Valuable Agreement Between CRISPR Therapeutics and KSQ Therapeutics

RegenxBio Rebounding RegenxBio (RGNX) has the viral vectors which could safely deliver genes into the cells. The firm has improved the adeno-associated viruses making them safe and effective gene delivery vehicles. One of the three currently approved gene therapy products, Zolgensma® for pediatric spinal muscular atrophy (SMA), marketed by Novartis, used RegenxBio’s adeno-associated . . . This content is for paid subscribers. Please click here to …