FDA’s research guidance will help take guesswork out of drug dosing in newborns

STAT | October 01, 2019

When we need to give medicine to newborns in our hospital, we often have to guess at the correct dose to give them. That’s a problem: give too little and the medicine might not work; give too much and it might cause harm. Dosing newborn babies with greater precision has been a challenge because it is difficult to conduct studies in this population, many of whom are very small and often critically ill. For researchers trying to determine the appropriate dosing for a medicine for adults, the FDA has clear guidelines on how to give the medicine, collect blood samples, examine the drug level in the blood, apply math to analyze how the level changes over time, and make decisions based on these analyses. But these guidelines don’t necessarily work for studying drugs in full-term and premature babies, who could weigh as little as 1 pound. Not only are newborns smaller, but their organs aren’t yet fully developed, so it may take longer for them to process or excrete medicines compared to older children and adults. It is also harmful to take as many blood samples from a newborn as can be taken from an adult.

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PHARMA TECH

iXensor Receives Strategic Investment from Rohto Pharmaceutical with A Separate Technology Licensing Agreement

iXensor | April 28, 2022

iXensor, the pioneer of mobile health, announced that Rohto Pharmaceutical Co., Ltd, a multinational pharmaceutical corporation headquartered in Japan, has become the new strategic partner and shareholder. iXensor received Rohto Pharmaceutical's strategic investment to expand its mobile health business globally and accelerate its PixoTech® platform licensing business. Building on this strategic partnership, Rohto Pharmaceutical will deploy iXensor's patented PixoTech® technology to digitize its new products through a separate technology licensing and joint development agreement, thereby providing Rohto Pharmaceutical's consumers with innovative self-testing portfolios including women's health and infectious diseases in Japan market. iXensor's first product, PixoTest® Glucose Monitoring System, developed based on its PixoTech® platform, is the world's first US FDA-approved smartphone camera-based blood test dated in 2017. The PixoTech®, currently protected by more than 70 international patents, is iXensor's core technology platform that transforms smartphones into in vitro diagnostic instruments for biomedical analysis in point-of-care testing and self-testing. PixoTech® controls smartphones' camera sensing modules and screen lighting emissions with multiple wavelengths to analyze all types of immunoassay and enzymatic tests through the proprietary algorithm. "By investing in iXensor, we can deepen our joint efforts on a series of new product developments, thus expanding our testing and diagnostic business through a wave of digital transformation. As the first step, we will receive a technical license for smartphone-based diagnostic devices and apps from iXensor to develop and sell innovative self-testing products." Mr. Hidetoshi Segi, Chief Strategy Officer of Rohto Pharmaceutical Dr. Carson Chen, CEO of iXensor, expressed, "This partnership represents the unlimited potential and technical advantage of the PixoTech® platform to transform smartphones into precision medical diagnostic devices. The licensing business of PixoTech® technology started shifting in as the new engine to drive iXensor's growth." In the post-pandemic era, iXensor endeavors to accelerate the growth of the at-home self-testing market and empower the evolution of the digital health ecosystem through licensing the PixoTech® technology. Thanks to that, PixoTech® can be flexibly applied to the digitalization of various lateral flow tests. In addition, it can shorten the lead time for IVD manufacturers venturing into the digital health business. It can also be applied to develop companion diagnostic or remote monitoring tools for pharmaceuticals to prove the efficacy of new classes of medications with actual test results collected from patients' smartphones with comfort at home. About Rohto Pharmaceutical Founded in 1899, Rohto Pharmaceutical is a renowned Japanese company that has successfully commercialized its research and development of consumer and pharmaceutical products worldwide. Rohto Pharmaceutical has established a robust product portfolio, including eye care, skincare, gastrointestinal medicines, and functional foods. Within the test drug business, the novel coronavirus antigen rapid test for medical institutions was launched in response to the global epidemic in addition to the existing pregnancy test, ovulation test, and flu rapid test products. About iXensor iXensor, the pioneer of mobile health, turns smartphones into lab-grade mobile medical diagnostics. In 2017, iXensor introduced the PixoTest® Blood Glucose Monitoring System as the world's first US FDA-approved smartphone camera-based blood test. Based on the PixoTech® platform, iXensor has ventured into at-home self-testing and clinical point-of-care diagnostics across infectious diseases, women's health, diabetes, and cardiovascular diseases.

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BUSINESS INSIGHTS

Basetwo Launches No-Code Artificial Intelligence Platform for Pharmaceutical Manufacturing, Raises $3.8 Million

Basetwo | May 27, 2022

Basetwo, a SaaS AI platform for manufacturers, announced it secured a $3.8M seed round led by Glasswing Ventures and Argon Ventures with additional funding from Caffeinated Capital, Graphite Ventures, MaRs IAF, Pareto Holdings, Plug and Play, and Quiet Capital. The funding supports the launch of the category-making Basetwo AI platform, a first-of-its-kind solution that enables process engineers to rapidly build digital twins of their manufacturing plants leveraging a familiar no-code interface. Basetwo promotes the digitalization of manufacturing systems to foster intelligent manufacturing across the global supply chain. Basetwo intends to be the default platform for continuous improvement in the current Good Manufacturing Process (cGMP), beginning with the pharmaceutical market and expanding to the food and beverage and chemical markets. The pharmaceutical manufacturing market has been challenged in the past two years due to the pandemic and associated supply chain issues. In addition, the rate of drug development and demand for pharmaceuticals has increased at an unprecedented rate, fueled by technological and economic advancement globally. However, manufacturing optimization to meet this demand is still rudimentary and inadequate to meet today’s and future needs. According to ABI Research the pharma industry is predicted to spend over $4.5 billion on digital transformation by 2030, growing at a CAGR of 27%. In addition, the market for digital twins is forecasted to reach $35.5 billion by 2026, a 37% CAGR, according to a recent ResearchandMarkets report, primarily driven by the growing applications of digital twin technology in the pharmaceutical and healthcare industries. Basetwo’s platform powered by “physics-informed machine learning” is well suited for pharmaceutical manufacturing. The Company’s no-code approach brings highly sophisticated capabilities from pre-built models to data pipelines and integrations within a complex manufacturing process to a format familiar to process engineers. Basetwo's ability to predict and understand manufacturing processes improves as more data is gathered, improving return on investment over time. “The introduction of our no-code AI platform lets manufacturers of any size leverage the power of the Internet of Things (IoT) and machine learning (ML) to develop digital twins easily, quickly, and economically. We are excited to have the backing and the expertise from Glasswing Ventures, Argon Ventures, and all our investors as we scale the business.” Thouheed Abdul Gaffoor, Co-Founder and CEO of Basetwo Having previously co-founded and exited Emagin, an AI startup in the heavy industry, Thouheed has seen first-hand the challenges that manufacturers face with maximizing value from machine learning initiatives. To address this challenge, Thouheed left his previous role as Head of AI at Autodesk, and partnered with Thamjeeth Abdul Gaffoor, who co-founded and served as the CFO at Emagin, Tawfeeq Abdul Gaffoor, former Software Engineer at Lyft and Databricks, and Kiefer Eaton, a pharmaceutical industry specialist from Johnson & Johnson. Together, the Basetwo founding team brings deep domain expertise in manufacturing, technology, and pharmaceuticals. “The pharmaceutical manufacturing market is at a critical point, having been heavily challenged by the pandemic and supply chain issues. By creating a digital duplicate of a pharmaceutical manufacturing plant, the Basetwo AI platform allows process engineers to analyze where things might break down — or run more efficiently, and increase resilience,” said Rudina Seseri, Founder and Managing Partner of Glasswing Ventures. “As a category maker, Basetwo has delivered a revolutionary AI-based no-code solution that enables manufacturers to use digital twins for optimization. We anticipate that Thouheed and his team will disrupt the status quo in manufacturing and affect significant change in the industry.” About Basetwo Basetwo is the first no-code AI platform that enables process engineers to rapidly build and operationalize digital twins of their manufacturing plants. From process development to commercial manufacturing, manufacturers use the digital twins they build with Basetwo AI to improve process efficiencies across the value chain. About Glasswing Ventures Glasswing Ventures is an early-stage venture capital firm dedicated to investing in the next generation of AI and frontier technology startups that enable the rise of the intelligent enterprise. The firm was founded by visionary partners with decades of experience in these markets, a disciplined investment approach, and a strong track record of industry-leading returns. Glasswing leverages its deep domain expertise and world-leading advisory councils to invest in exceptional founders who transform markets and revolutionize industries. About Argon Ventures Argon Ventures is a venture fund with a mission to amplify the energy of founders to launch breakthrough products to build impactful, global businesses. The firm’s Intelligent Industry Solutions investment thesis is centered on cutting-edge software platforms that accelerate productivity & drive top-line revenue, transform markets through radical efficiency and create enterprise value through data at scale. Argon’s portfolio spans a number of markets, including digital health, future of work, manufacturing, and others. The founding General Partners collectively bring over four decades of experience as successful operators, investors, advisors, founders and C-level executives of global companies.

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BUSINESS INSIGHTS

MedAvail Announces Pricing of $50 Million Private Placement

MedAvail | April 01, 2022

MedAvail Holdings, Inc. a technology-enabled retail pharmacy company, announced it has entered into a definitive securities purchase agreement for the sale of common stock and warrants to purchase common stock in a private placement with certain institutional and other accredited investors for gross proceeds to MedAvail of $50 million, before deducting placement agent commissions and other offering expenses. Pursuant to the terms of the securities purchase agreement and following the completion of all closings of the private placement, MedAvail will issue approximately 47.1 million shares of common stock and warrants to purchase approximately 23.5 million shares of common stock. The shares of common stock will be sold for a price of $1.0625 per share. Each purchaser will also receive a warrant to purchase 50% of the number of shares purchased under the securities purchase agreement by such purchaser. The warrants will have a per share exercise price of $1.25 and will be exercisable by the holder at any time on or after the issuance date for a period of five years. In addition, the warrant terms provide MedAvail with a call option to force the warrant holders to exercise up to two-thirds of the warrant shares subject to each warrant, with one-third of the warrant shares being callable beginning on each of the 12 month and 24 month anniversaries of the warrant issuance dates, in each case until the expiration of the warrants, and subject to the satisfaction of certain pricing conditions relating to the trading of MedAvail’s shares. If all warrants that are sold and issued in the private placement following the completion of all closings are fully exercised, then MedAvail would receive gross proceeds of approximately $29.4 million. The private placement is expected to have a first close on or about April 1, 2022, subject to the satisfaction of customary closing conditions. Additional details regarding the private placement will be included in a Form 8-K to be filed by MedAvail with the Securities and Exchange Commission. MedAvail intends to utilize the net proceeds for general corporate purposes and to fund its strategic initiatives. The securities to be sold in the private placement have not been registered under the Securities Act of 1933, as amended or any state or other jurisdiction’s securities laws, and may not be resold absent registration under, or exemption from registration under, the Securities Act. MedAvail has agreed to file a registration statement with the SEC registering the resale of the shares of common stock to be issued and sold in the private placement, together with the shares of common stock underlying the warrants issued in the private placement, within 60 days of the applicable closing of the private placement in which such securities were purchased. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction. Cowen acted as the placement agent for the private placement. Lake Street acted as financial advisor. About MedAvail MedAvail Holdings, Inc. is a technology-enabled pharmacy organization, providing turnkey in-clinic pharmacy services through its proprietary robotic dispensing platform, the MedAvail MedCenter, and home delivery operations, to Medicare clinics. MedAvail helps patients to optimize drug adherence, resulting in better health outcomes.

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VIEWS AND ANALYSIS

CHMP Recommends Approval of Dupixent® (dupilumab) for Children Aged 6 to 11 Years with Severe Asthma with Type 2 Inflammation

Regeneron Pharmaceuticals, Inc | January 31, 2022

Regeneron Pharmaceuticals, Inc. and Sanofi today announced that the European Medicines Agency's Committee for Medicinal Products for Human Use has adopted a positive opinion, recommending to extend the approval of Dupixent® (dupilumab) in the European Union (EU) to include add-on maintenance treatment for children aged 6 to 11 years with severe asthma with type 2 inflammation characterized by raised blood eosinophils and/or raised fractional exhaled nitric oxide (FeNO) who are inadequately controlled on two maintenance therapies. The European Commission is expected to announce a final decision on the Dupixent application in the coming months. The CHMP positive opinion is supported by Phase 3 data recently published in the New England Journal of Medicine showing that Dupixent added to standard of care significantly reduced the rate of severe asthma attacks and rapidly improved lung function within two weeks, with sustained improvement up to 52 weeks, in children with uncontrolled moderate-to-severe asthma. The safety results from the trial were generally consistent with the known safety profile of Dupixent in patients aged 12 years and older with uncontrolled moderate-to-severe asthma. Adverse events more commonly observed with Dupixent compared to placebo included injection site reactions, viral upper respiratory tract infections and eosinophilia. Helminth infections were also more commonly observed with Dupixent compared to placebo in patients aged 6 to 11 years. Asthma is one of the most common chronic diseases in children. Up to 85% of children with asthma may have type 2 inflammation and are more likely to have higher disease burden. Despite treatment with current standard-of-care inhaled corticosteroids (ICS) and bronchodilators, these children may continue to experience serious symptoms such as coughing, wheezing and difficulty breathing. Severe asthma may impact children's developing airways and cause potentially life-threatening exacerbations. Children with severe asthma also may require the use of multiple courses of systemic corticosteroids that carry significant risks. Uncontrolled severe asthma can interfere with day-to-day activities like sleeping, attending school and playing sports. On October 20, 2021, Dupixent was approved by the FDA as an add-on maintenance treatment for patients aged 6 to 11 years with moderate-to-severe asthma characterized by an eosinophilic phenotype or with oral corticosteroid-dependent asthma. The use of Dupixent in children aged 6 to 11 years with uncontrolled severe asthma is investigational in the EU and is not yet approved. About Dupixent Dupixent, which was invented using Regeneron's proprietary VelocImmune® technology, is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. IL-4 and IL-13 are key and central drivers of the type 2 inflammation that plays a major role in atopic dermatitis, asthma and chronic rhinosinusitis with nasal polyposis (CRSwNP). About Regeneron Regeneron is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to nine FDA-approved treatments and numerous product candidates in development, almost all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, pain, hematologic conditions, infectious diseases and rare diseases. Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite technologies, such as VelocImmune, which uses unique genetically humanized mice to produce optimized fully human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world. About Sanofi Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions. With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.

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