Novartis' SMA gene therapy would not be cost-effective if priced over $1.5M: ICER

FiercePharma | April 04, 2019

The pricing analysis done by the Institute for Clinical and Economic Research (ICER) on new therapies for spinal muscular atrophy (SMA) has been no small undertaking. One of the therapies ICER reviewed—Zolgensma from Novartis’ AveXis unit—isn't yet FDA-approved or priced, forcing the influential agency to assess it using hypothetical price ranges. Now, after considering input from Novartis, ICER has released its final word on the subject—and it hasn't changed. The agency stood by its initial assessment, saying a price of $900,000 or so would make the most sense, but it definitely shouldn't carry a price higher than $1.5 million. The FDA is expected to decide on Zolgensma in May, and Novartis will reveal its pricing plans then. But Dave Lennon, president of AveXis, has already had plenty to say on the subject. Lennon, who argues that gene therapy cures need an entirely different type of evaluation, created quite a stir last year when he said the gene therapy would be cost-effective at $4 million to $5 million.

Spotlight

Quickly and easily quantitate immune checkpoint markers using a fraction of the sample volume required by traditional ELISA. In this technical note, we show how AlphaLISA can be used to detect and quantify additional immune checkpoint molecules within the PBMC population with and without activation using Dynabeads.


Other News
BUSINESS INSIGHTS

Athira Pharma Announces Topline Results from ACT-AD Phase 2 Proof of Concept Study of Fosgonimeton in Mild-to-Moderate Alzheimer’s Disease

Athira Pharma, Inc. | June 27, 2022

Athira Pharma, Inc. a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today announced topline results from its exploratory ACT-AD Phase 2 study of fosgonimeton in patients with mild-to-moderate Alzheimer’s disease. Fosgonimeton is a small molecule designed to enhance the activity of Hepatocyte Growth Factor and its receptor, MET, which are expressed in the central nervous system to promote brain health and function. “Following compelling ERP P300 latency biomarker data from a small Phase 1b trial over eight days in Alzheimer’s patients on fosgonimeton monotherapy, this Phase 2 trial provides valuable insights into the nature of this novel intervention over 26 weeks. ACT-AD was designed as a learning study to further investigate the ERP P300 biomarker signal over 6 months, assess safety in a patient population more representative of the real world, by allowing the use of add-on standard-of-care acetylcholinesterase inhibitors (AChEIs, e.g., donepezil), and explore fosgonimeton’s effect on psychometric outcomes, including ADAS-Cog11, to inform the ongoing Phase 3 LIFT-AD study. To that end, this study achieved its goal,” said Hans Moebius, M.D., Ph.D., Chief Medical Officer of Athira. “The study was intended to show differences on the biomarker ERP P300 latency. This primary endpoint was not met by protocoled analysis, however a pre-specified subgroup analysis indicated a potential diminished effect of fosgonimeton when given in combination with AChEIs. A subsequent post hoc analysis of the data from patients on fosgonimeton monotherapy showed a meaningful improvement in both ERP P300 latency and cognitive performance compared to placebo at 26 weeks. “These data points are very encouraging as they indicate the expected pharmacological activity of fosgonimeton by parallel improvement on ERP P300 latency and ADAS-Cog11 and show a favorable safety profile over six months. This is the first time monotherapy fosgonimeton has shown an effect on ADAS-Cog11, suggesting a potential cognitive benefit. We will use these insights for a rational optimization of the ongoing LIFT-AD trial. We plan to seek advice from our scientific advisors, investigators, and ultimately regulators on how to expeditiously analyze and potentially adapt the LIFT-AD study,” Dr. Moebius “The data from the fosgonimeton monotherapy analysis are encouraging and show biologic activity that may support the potential role of the HGF/MET pathway in neurodegenerative diseases,” said Marwan Sabbagh, M.D., FAAN, professor of neurology at Barrow Neurological Institute, Phoenix, AZ. “ACT-AD adds to the body of literature suggesting ERP P300 latency as an important biomarker for cognitive status.”

Read More

PHARMACY MARKET

HKSTP and Boehringer Ingelheim Venture Fund Launch Co-Incubation Programme to Fast-track R&D in Infectious Diseases and Immunology

BIVF | June 18, 2022

Hong Kong Science and Technology Parks Corporation is partnering global research-driven biopharmaceutical leader, Boehringer Ingelheim Venture Fund Limited in a strategic co-incubation collaboration to promote and nurture startups on research and development for infectious diseases and immunology. HKSTP is dedicated to collaborating with sector leaders in building the strongest I&T eco-system to help startups via business development, mentorship and investment initiatives. In the last five years, the number of biotech companies at HKSTP have tripled from 50 to over 150, which has also synched with the Government’s strategic focus on biomedical technology with allocation of HK$10 billion to develop life and health technology as a key future growth sector for Hong Kong. This partnership between HKSTP and BIVF marks a key milestone to drive the Hong Kong’s biomedical technology development. Both local and global qualified startups in infectious diseases and immunology can apply to the incubation programme to access the full capabilities of the HKSTP ecosystem and BIVF’s extensive biotech funding network. Incubatees can access one-on-one coaching and assessment to track key research milestones, while receiving expert guidance from HKSTP on commercialisation, manufacturing, scaling-up and marketing strategy to ensure successful innovation, plus vital funding opportunities and investment insight from BI. “Hong Kong is now Asia’s largest and the world’s second largest fundraising hub for biotech. As HKSTP marks our 20th year of propelling success and innovation, our mission is to drive the growth of biotech to another level with world class leaders like BIVF. We will maximise the GBA growth opportunities for high-potential tech talents and early-stage startups to ensure the region emerges as a global I&T powerhouse.” Mr. Albert Wong, CEO of HKSTP Dr. Grace Lau, Head of Institute for Translational Research of HKSTP, said: “Our partnership with BIVF, provides early-stage startups and promising university spinoffs with vital support at the most critical stage of their long and challenging biotech innovation journey. Incubatees will have access to our Incu-Bio Programme, with total incubatees doubling up in the last five years. The startups can also access funding support of up to HK$6 million, with financial subsidies and upfront grants to cover regulatory activities such as clinical trials.” “The world has faced growing public health challenges in recent years. As Boehringer Ingelheim’s strategic investment arm, BIVF focuses on the development of new science in areas with huge unmet medical needs such infectious diseases and immunology,” said Dr. Frank Kalkbrenner, Global Head of the BIVF. “The co-incubation program jointly initiated by HKSTP and BIVF will enable us to identify more breakthrough technologies in the early stage of development. With the funding and infrastructure support offered by HKSTP and BI‘s expertise and experiences in the successful development of breakthrough mediactions for patients, we foster the startup companies and bridge the gap between science and industry for the local ecosystem. We look forward to developing together with HKSTP next-generation therapies in the fields of infectious diseases and immunology with long term partnerships.” “The innovation competency of biopharmaceuticals in Asia is rising quickly. To grab the opportunities in this market, Boehringer Ingelheim has set up the External Innovation Hub in China which brings our Research Beyond Borders, Business Development & Licensing and Venture Fund groups under one umbrella. Now we’re thrilled to see that the biopharmaceutical industry is picking up rapidly in the Guangdong–Hong Kong–Macau Greater Bay Area, it’s a great opportunity for us to partner with HKSTP to develop the local ecosystem, and further enhance China’s dual-circulation scheme. We hope to offer our continuous support to more home-grown innovations to be recognized on the global market and eventually benefit the patients worldwide.” said Mr. Felix Gutsche, President & CEO, Boehringer Ingelheim China. Programme incubatees will also benefit from HKSTP’s rapidly-growing biotech R&D capabilities including the HKSTP Institute for Translational Research (ITR), enabling biomedical startups to turn their innovative biomedical technologies to life-changing impact for patients and society. Also available to startups is HKSTP’s Incu-Bio program providing dedicated biotech mentorship, business matching, entrepreneur-in-residence, and access to R&D facilities and the Science Parks’ diverse talent pool. About Hong Kong Science and Technology Parks Corporation Hong Kong Science and Technology Parks Corporation (HKSTP) has for 20 continuous years committed to building up Hong Kong as an international innovation and technology hub to propel success for local and global pioneers today and tomorrow. HKSTP has established a thriving I&T ecosystem that is home to three unicorns and Hong Kong’s leading R&D hub with over 11,000 research professionals and over 1,000 technology companies focused on healthtech, AI and robotics, fintech and smart city technologies. Established in 2001, we attract and nurture talent, accelerate and commercialise innovation and technology for entrepreneurs on their journey of growth in Hong Kong, to the Greater Bay Area, Asia and beyond. Our growing innovation ecosystem is built around our key locations of the Hong Kong Science Park in Shatin, InnoCentre in Kowloon Tong and three modern INNOPARKs in Tai Po, Tseung Kwan O and Yuen Long. The three INNOPARKs are realizing a vision of re-industrialisation for Hong Kong. The goal is sectors like advanced manufacturing, electronics and biotechnology are being reimagined for a new generation of industry. Through our infrastructure, services, expertise and network of partnerships, HKSTP will help establish innovation and technology as a pillar of growth for Hong Kong, while reinforcing Hong Kong’s international I&T hub status as a launchpad for global growth at the heart of the GBA innovation powerhouse. About BIVF Created in 2010, the Boehringer Ingelheim Venture Fund GmbH invests in groundbreaking therapeutics-focused biotechnology companies to drive innovation in biomedical research. searching for significant enhancements in patient care through pioneering science and its clinical translation by building long-term relationships with scientists and entrepreneurs. BIVF’s focus is to target unprecedented therapeutic concepts addressing high medical needs in immuno-oncology, regenerative medicine, infectious diseases and digital health. These may include novel platform technologies to address so far undruggable targets, new generation vaccines and/or new biological entities, such as oncolytic virotherapy. About Boehringer Ingelheim External Innovation Hub China Established in 2020, Boehringer Ingelheim External Innovation Hub China unites three external innovation functions including Research Beyond Borders (RBB), Business Development & Licensing and the Boehringer Ingelheim Venture Fund. Through this innovative business model, Boehringer Ingelheim External Innovation Hub China is committed to providing one-stop solutions for biomedical innovators in China and. It is also dedicated to enriching the company's global R&D pipeline through external innovation collaborations so as to benefit more patients worldwide.

Read More

PHARMACY MARKET

SoniVie receives IDE approval from FDA for its Pilot study to treat Hypertension with its Renal Artery Denervation TIVUS™ technology

SoniVie | June 20, 2022

SoniVie, an Israeli company developing a novel proprietary Therapeutic Intra-Vascular Ultrasound System to treat a variety of hypertensive disorders, announced that on May 5th 2022 the U.S. Food and Drug Administration granted IDE approval for its "REDUCED1" Pilot study to treat Resistant Hypertension Patients with Renal Artery Denervation using TIVUS™, its innovative Ultra-Sound Ablation System. Resistant hypertension is defined as blood pressure higher than 140/90 mmHg despite use of three antihypertensive medications of different classes at the best tolerated doses, one of which must be a diuretic. Millions of people world-wide suffer from resistant hypertension which substantially increases the risk of heart attack, stroke and kidney failure. "We are very pleased that FDA has approved the REDUCED1 study. Sites initiation has started, and many clinical teams have responded very favourably about participating to the study. There is a significant number of patients that may benefit from our technology and we are genuinely happy for this important step towards the introduction of TIVUS™ in the US. There is a lack of effective therapeutic solutions for patients suffering from resistant hypertension, and physicians are looking forward to a safe, effective and easy to use device treatment," Christian Spaulding, CMO, SoniVie Ltd "This is a significant US Regulatory milestone for SoniVie, starting the feasibility study using the Ultra-Sound ablation platform in the US for the Renal Denervation indication. This is a major step and priority in the company's history," says Tomaso Zambelli, CEO, SoniVie LTD. The REDUCED1 study will further expand the Company's clinical experience based on two clinical trials in Renal Denervation performed using the earlier generation of the TIVUS™. Renal Denervation with TIVUS™ is a minimally invasive procedure that uses high-frequency non-focused Ultra-Sound energy to ablate nerves in the renal artery. This causes a reduction in the nerve activity, which may decrease blood pressure. This procedure is designed for patients who suffer from resistant hypertension. About SoniVie SoniVie is a medical device company developing the TIVUS™, the only Ultra-Sound Denervation platform with active development programs in three therapeutic areas: pulmonary artery denervation for pulmonary hypertension, renal artery denervation for resistant hypertension, and lung denervation for chronic obstructive pulmonary disease with chronic bronchitis. These diseases affect millions of patients in the United States and Europe.

Read More

BUSINESS INSIGHTS

Hyfe Joins Forces with Merck to Deploy Its Artificial Intelligence-Powered Cough Detection and Counting Technology

Hyfe, Inc. | July 20, 2022

Hyfe, Inc., the global leader in AI-powered cough detection, tracking and classification, has entered into an agreement with Merck, known as MSD outside the United States and Canada, to integrate its artificial intelligence-powered (AI) cough tracker technology into Merck’s consumer disease educational efforts.. Hyfe’s AI-powered technology passively and remotely monitors the number of times a person coughs and the sound of the cough through any smart device, such as a mobile phone. This data collected over time provides the user with helpful information by revealing patterns and potential triggers like seasonal allergies that may be causing the cough that would otherwise go unnoticed or unaddressed. “The overwhelming majority of people cannot even come close to accurately estimating the number of times they cough per day. In order to help diagnose and treat patients, cough frequency and sound should be monitored and measured with the same accuracy as blood pressure, temperature, heartbeat and other biomarkers that are essential for assessing health and medical conditions.” Peter Small, M.D., chief medical officer of Hyfe, Inc Users of the app will be able to determine when and how long to track their coughing. The AI-powered technology records cough-related sounds and allows the user to compare the frequency and quality of their coughs against their own benchmark. This personalized approach provides users with more detailed information and confidential data about their individual situations to share with their health care providers. Hyfe, Inc. is the global leader in AI-powered cough detection and classification that provides insight into cough patterns and correlations that can greatly improve treatment and prevention. With more than 280 million samples, Hyfe maintains the largest cough dataset in the world enabling the building of powerful models to track, manage and diagnose respiratory illnesses. Hyfe provides platforms and data for pharmaceutical companies, medical researchers, government agencies, health care providers and patients and has partnerships with leading academic institutions including Johns Hopkins University and the University of California at San Francisco. The company was founded in 2020 and is headquartered in Wilmington, Delaware.

Read More

Spotlight

Quickly and easily quantitate immune checkpoint markers using a fraction of the sample volume required by traditional ELISA. In this technical note, we show how AlphaLISA can be used to detect and quantify additional immune checkpoint molecules within the PBMC population with and without activation using Dynabeads.

Resources