Author Archives: admin


FDA Approves Y-mAbs’ DANYELZA® (naxitamab-gqgk) for the Treatment of Neuroblastoma

NEW YORK, Nov. 25, 2020 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, today announced that the U.S. Food and Drug Administration (“FDA”) has approved DANYELZA (naxitamab-gqgk) 40mg/10ml. DANYELZA is indicated, in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication is approved under accelerated approval regulation based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA is a humanized, monoclonal antibody that targets the ganglioside GD2, which is highly expressed in various neuroectoderm-derived tumors and sarcomas. DANYELZA is administered to patients three times in a week in an outpatient setting and the treatment is repeated every four weeks. The product has received Priority Review, Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations from the FDA.

Here is the original post:
FDA Approves Y-mAbs’ DANYELZA® (naxitamab-gqgk) for the Treatment of Neuroblastoma

Shattuck Labs to Present Virtually at Evercore ISI 3rd Annual HealthCONx Conference

AUSTIN, TX and DURHAM, NC, Nov. 25, 2020 (GLOBE NEWSWIRE) -- Shattuck Labs, Inc. (Shattuck) (NASDAQ: STTK), a clinical-stage biotechnology company pioneering the development of bi-functional fusion proteins as a new class of biologic medicine for the treatment of patients with cancer and autoimmune disease, today announced it will participate in the Evercore ISI 3rd Annual HealthCONx conference being held December 1-3, 2020.

See the original post here:
Shattuck Labs to Present Virtually at Evercore ISI 3rd Annual HealthCONx Conference

Novavax to Participate in Upcoming Conferences

GAITHERSBURG, Md., Nov. 25, 2020 (GLOBE NEWSWIRE) -- Novavax Inc. (Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today announced that it will participate in two upcoming investor conferences. Novavax’ COVID-19 vaccine candidate, NVX-CoV2373, will be a topic of discussion in each conference.

Read more:
Novavax to Participate in Upcoming Conferences

Global Contract Cell and Gene Therapy Manufacturing Market Report 2020-2026: CDMO Categorization – Primed for Business Model Disruption – Benzinga

DUBLIN, Nov. 24, 2020 /PRNewswire/ -- The "Global Contract Cell and Gene Therapy Manufacturing Market 2020-2026 - Supply Chain Optimization and Decentralized Manufacturing to Expand the Industry" report has been added to ResearchAndMarkets.com's offering.

This research service focuses on the critical role being played by CDMOs in not only supporting new product research and development but also in creating standardized manufacturing protocols.

Additionally, the study explores different cross-sections of the market and discusses market dynamics for autologous and allogeneic solutions for cell and gene therapies and for products being manufactured for clinical trial use and for commercial markets. The variability in market dynamics, manufacturing protocols, and business models across cross-sections is high. Therefore, the study also covers the emergence of non-traditional CDMOs that have thrived as a result of this variability.

The cell and gene therapy segment is one of the fastest growing segments in the biopharmaceutical space. While the science behind the therapy has grown by leaps and bounds on the back of decades worth of research, manufacturing has unfortunately lagged behind.

To fully harness the curative potential of these therapies and ensure greater reach and affordability to patients, it is imperative that aggressive investments in manufacturing technology and capacity are made today. Investments in manufacturing technology advancements including automation, single-use technologies, and GMP-in-a-box, will not only enable operational efficiency gains but also reduce project costs, generating benefits which can be transferred directly to the patients.

A large part of this growth in the cell and gene therapy product pipeline is being driven by small and mid-sized biotechs that depend on CDMOs to mitigate product development risks and diffuse infrastructure investments to be able to build future products pipeline.

The study also provides a comprehensive and critical analysis of nationally coordinated efforts towards infrastructure development and the rise of academic institutes and hospitals in meeting not only the demand from clinics, but also the demand of the commercial market.

The study also analyses the growth of specialist CDMOs focusing on vectors and plasmids, for instance. The captures the regional outlook for growth based on the currently installed capacities as well as the pipeline investments being made towards expansion. Lastly, the study tracks growth opportunities across the entire product development spectrum from supply chain to upstream and downstream manufacturing.

Key Topics Covered:

1. Strategic Imperatives

2. Growth Opportunity Analysis - CGT CDMO Market

3. Growth Opportunity Universe - CGT CDMO Market

4. Next Steps

For more information about this report visit https://www.researchandmarkets.com/r/kmx4o3

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Media Contact:

Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

View original content:http://www.prnewswire.com/news-releases/global-contract-cell-and-gene-therapy-manufacturing-market-report-2020-2026-cdmo-categorization---primed-for-business-model-disruption-301179914.html

SOURCE Research and Markets

Read the original:
Global Contract Cell and Gene Therapy Manufacturing Market Report 2020-2026: CDMO Categorization - Primed for Business Model Disruption - Benzinga

Internet hawkers of stem cell therapies raising red flags – Ophthalmology Times

This article was reviewed by Ajay E. Kuriyan. MD, MS

Patients have been blinded or have experienced retinal detachments after treatment with so-called stem cell therapies such as intravitreal injections of autologous stem cells that are not FDA approved for many ocular conditions across the US and that were marketed directly to patients.

Ajay Kuriyan, MD, MS, and colleagues had previously identified 40 such business with 70 clinics in the US that advertise these treatments directly to consumers.

Related: Improve patient comfort with intravitreal injections

He explained that the FDA began to issue permanent injunctions against these company, including US Stem Cell Clinic, LLC, US Stem Cell Inc., and the Cell Surgical Network Corporation.

Currently, the FDA has regulatory authority over such companies and since June 2019, has been issuing orders to prevent them from developing or distributing stromal vascular fraction products that are not FDA approved, he explained.

In light of this kind of marketing activity, Adam Ross-Hirsch, MD, first author of the study under discussion, and colleagues set out to determine the scope of U.S.-based businesses advertising and administering cell therapy for ocular conditions, by looking at the businesses public websites to see what has changed since they first published their findings in 2017.1

The investigators identified these business by searching the Internet using specific keywords, such as clinic locations, marketed ocular conditions, types of cell therapy offered, routes of administration, affiliation with credentialed physician, safety language, and treatment costs.

Related: Intravitreally injected hRPCs improve vision in retinitis pigmentosa cases

Results The results of the analysis showed that 13 companies had removed their advertisements for ocular conditions, and 6 discontinued their URLs.

However, despite these findings, the total number of companies promoting treatment remained almost the same as the first study.

A total of 39 businesses with 62 clinics were identified by August 2019; 14 of these and 20 clinics were newly identified, Dr. Ross-Hirsch reported.

Most of the clinics were in Texas (n=12), California (n=11), Florida (n=10), and Illinois (n=10). When the investigators compared these numbers of clinics to those previously identified, the number in California decreased by 48% and the ones in Texas more than doubled, he said.

Thirty of the 39 companies reported that they were affiliated with at least 1 credentialed physician. Six businesses claimed to be associated with ongoing clinical trials, however, only 2 were registered on the clinicaltrials.gov website.

Related: Stem cell transplantation: Restoring vision in AMD may be possible

Most of the identified companies did not show their treatment fees, but of the 4 that did the costs ranged from $4,000 to $12,000/treatment. Consultation fees ranged from $350 to $800 when they were listed.

All businesses marketed treatments for ocular diseases that included age-related macular degeneration [AMD], optic neuritis, retinitis pigmentosa [RP], diabetic retinopathy, and glaucoma, he said. Most claimed to be able to treat AMD followed by RP.

The cell therapies most often advertised were autologous adipose-derived stem cells followed by autologous bone marrow-derived stem cells, which was similar to the previous study.

Related: Exploring novel gene therapy approaches to treat ocular disease

Making claims In commenting on the findings, Kuriyan said, These websites contained various claims regarding the safety and the FDA, but the language contained variable and vague claims about the safety of the various cell therapies.

Examples of the language were the following: not approved or evaluated by the FDA, participation in patient funded clinical research, use of FDA or institutional review board approved equipment and/or protocols for cell isolation, and use of FDA guidelines for minimally manipulated same-day procedures intended for homologous use.

Ten companies did not mention the FDA, while other claims were that the advertised therapies were safe under FDA regulation and no knowledge in the current literature of serious harm transpiring due to the use of these products, he emphasized.

Related: Stem cells for dry AMD with GA show promise in early study

These companies identified in the study have the potential to leverage a faade of scientific legitimacy to capitalize on patients understandable desperation for effective sight-restoring therapies. Stem cell therapy is an area of active, intense, and scientifically rigorous research. In the future, it may safely and effectively treat a number of ocular conditions, Kuriyan stated.

Lacking evidence These companies persist despite the absence of high-quality evidence of the efficacy of the treatment, FDA approval of the procedures, the increasing regulatory oversight and legal action against some of these companies, and evidence of severe visual loss as a result of the procedures.

Based on these findings, the investigators concluded that these companies persist in their US-based direct-to-consumer marketing of cell therapies for ocular conditions. Related: Exploring safety of stem cells for dry AMD

Ross-Hirsch emphasized the importance of both counseling by ophthalmologists and optometrists for patients as a safeguard against these companies and as stem cell therapies enter well-designed trials and evidence-based practice and federal regulation. However, regulation remains limited.

Conclusion Direct marketing of cell therapies persists in the US. As of August 2019, at least 39 such companies are operating in the US. Current federal regulation is limited and the marketing on the internet is pervasive.

Counseling will serve as a safeguard for patients and become increasingly important as stem cell therapies enter well-designed clinical trials and possibly evidence-based practice. Read more by Lynda Charters

Reference 1. Nirwan RS, Albini TA, Sridhar J, et al. Assessing cell therapy clinics offering treatment of ocular conditions using direct-to-consumer marketing in the United States. Ophthalmology 2019;126:1350-1355.

Originally posted here:
Internet hawkers of stem cell therapies raising red flags - Ophthalmology Times