// Biotech and Pharma Therapeutics
Drug companies set sights on new Covid medicines to boost depleted arsenal
It was not that long ago that the medical community had a small arsenal of new drugs to prevent and treat COVID-19 in people most susceptible to severe disease. But the arrival of new variants, as well as the loss of two treatments for immunocompromised people, has amped up the urgency to develop a stronger generation of medicines.
Opus Genetics Announces Acquisition of the Rights to Two Gene Therapy Product Candidates for Inherited Retinal Diseases
Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced it has acquired the rights to two preclinical-stage AAV-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio. Opus will develop the novel gene therapy candidates to address bestrophin-1 (BEST1)-related inherited retinal diseases and rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), respectively.
Should Patients Have the Right to Own Their Clinical Trial Data?
Participants in clinical trials donate their health information so researchers can learn more about the risks and benefits of new treatments. During a clinical study, all kinds of personal information are collected: from genetic sequencing to cholesterol measurements, CT scans to IQ results. This data is then analyzed by researchers to answer specific research questions. But what happens to a patient’s individual data? More specifically, do participants have the right to own their own clinical research data?
Biostage Selects IQVIA to Manage its FDA-Approved Clinical Trial in Severe Esophageal Disease, including Cancer
The FDA-approved clinical trial is an open-label trial assessing both safety and efficacy in up to ten patients requiring the removal of up to 6cm of their esophagus for any reason (including cancer, trauma or birth defects) at up to five hospitals in the U.S. The primary endpoint is the establishment of a continuous biological conduit at three months following implantation.
What Will the Pharma Industry Face in the Year Ahead?
Life science organizations faced down an array of daunting challenges during the last three years: a global health crisis, supply chain upheaval, and uncertain economic climate, just to name a few. But if we can say the dust is beginning to settle, we can also look to the future with the knowledge that uncertainty will persist – and that we should be prepared. How can companies prepare for an unpredictable year ahead?
// 4th Industrial Revolution
Emergency Drone Can Deliver First Aid Medication by Itself
The uFAST relies on a drone — more accurately, an unmanned aerial vehicle (UAV) — to deliver first-aid therapeutics to the scene of an accident or other medical emergency. If there are people already on site, they might not be trained in medical care or drug administration. The system has that covered, as the uFAST is designed to administer medications on its own. It hovers over the patient and releases a weighted canister. When it contacts the patient’s body, the device applies a patch covered with microneedles that are loaded with the required therapeutics.
Real-World Study Uses AI to Identify Undiagnosed Dementia in Primary Care
Few primary care practices are designed for the timely detection of Alzheimer’s disease. The limited time that primary care clinicians have to spend with patients, the need to focus on the health problems which brought the patient to the clinic, as well as the stigma of dementia are the major reasons for the lack of recognition of the condition, according to Dr. Boustani. In addition, he notes, there is no demand from the public for dementia diagnoses, most likely driven by the stigma of dementia, lack of public knowledge about the benefits of early recognition of Alzheimer’s, and issues related to health insurance coverage.
Smart Belt Monitors Heart Failure Patients
More than 6 million adult patients in the U.S. have heart failure, a progressive condition that prevents the heart from pumping an adequate supply of blood to the body. Treatment typically includes medication, which must be adjusted as the patient’s condition changes. The sooner that changes can be detected, the sooner adjustments can be made. If adjustments are made early enough, you can avoid potentially life-threatening complications. As a result, constant monitoring can be the key to tracking a patient’s condition.
Sound and Light Create 3D Images of Deep Tissues
Blinking lights shining on our skin to make biometric measurements have become commonplace. Photoplethysmography (PPG) is used in fitness trackers, smart rings, and even some earbuds to measure heart rate, blood oxygen levels, and more. But like beauty, the sensitivity of these devices is only skin deep. Their sensors cannot penetrate deeply into the body. Researchers at the University of California San Diego (UCSD) have come up with a possible solution that relies on not just light, but sound as well.
How Automation Can Cut Into the $500B of Administrative Waste
In 2018, David Cutler, a professor of economics at Harvard University, submitted testimony on administrative waste to the U.S. Senate Committee on Health, Education, Labor and Pensions. In his research, Cutler estimated that administrative costs account for twice what the U.S. spends on cardiovascular disease care annually and three times the spend on cancer care.
// Business & Markets
Amgen, Merck KGaA add to budding constellation of ADC deals amid the modality’s meteoric rise
The pool of Big Pharmas competing to develop game-changing antibody-drug conjugates continues to grow, with the water now at a boil after two more sizable licensing pacts. Amgen and Merck KGaA each entered the holiday weekend with separate collaborations aimed at developing the burgeoning cancer-targeting modality, with the deals’ combined value eclipsing $2 billion.
Novavax Announces Closing of $175.25 Million Offering of Convertible Senior Notes Due 2027 and $74.75 Million Public Offering of Common Stock
On December 20, 2022, Novavax, Inc. (Nasdaq: NVAX), a biotechnology company (the “Company” or “Novavax”) dedicated to developing and commercializing next-generation vaccines for serious infectious diseases, completed the closing of its previously announced offering of $150 million aggregate amount of 5.00% Senior Convertible Notes due 2027 (the “Notes”), and on December 23, 2022, the Company completed the closing of the initial purchasers’ option to purchase up to an additional $25.25 million aggregate principal amount of the Notes, which was exercised in full.
Teleradiology Market Primed to Hit $3.7Bn by 2026 – What’s Driving the Surge in Demand?
4.7bn diagnostic imaging procedures were performed globally in 2021, representing a strong recovery over 2020. Healthy demand for teleradiology services resulted in the global penetration of teleradiology reads into diagnostic imaging procedures bouncing back to 1.9%, and the overall teleradiology reading services and IT market revenue elevating past its pre-pandemic level to USD $1.7bn.
A wave of overdoses is hitting an already vulnerable population
Francesca Coleman, known to friends as “St. Louis,” has been unhoused since the age of 15. Now 24, Coleman sleeps in Tongva park, across from the famous Santa Monica pier in California. Like many unhoused people in the Los Angeles area, Coleman uses meth in an attempt to ease chronic pain, anxiety, and depression. On a Wednesday morning in July, physician Coley King checked in with Coleman in Tongva Park. King practices street medicine, which means the majority of his patients are unhoused, and Coleman is one of his patients.
Many Women Need Mental Health Services But Aren’t Able To Access Them
About 50% of women ages 18 to 64 said they needed mental health services in the last two years, but only half of them were able to get an appointment, according to a recent survey. The Kaiser Family Foundation (KFF) survey includes a sample of 5,145 women and 1,225 men between the ages of 18 and 64. It was conducted online between May 10 and June 7.
// Legal & Regulatory
By Eliminating Requirement for Animal Testing, FDA Modernization Act Allows Faster, More Cost-effective Drug Development
Congress passed the FDA Modernization Act 2.0 last week removing the requirement to use animal testing in drug development. This will allow drug companies the option to use alternative safety-testing models when making new drug submissions. This is important because the Federal Food, Drug and Cosmetics Act of 1938, which is still in force, mandates animal testing for every new drug development protocol. Advocates of non-animal experimentation have argued that the cost of animal research is high—in dollars, in time, and in delays in approvals of beneficial drugs for human use.
Roche gets FDA nod for Lunsumio, offering convenient option to gene therapies in follicular lymphoma
Upon the FDA approval for its bispecific antibody Lunsumio (mosunetuzumab) as a third-line therapy for follicular lymphoma, Roche was quick to point out that its treatment was an “off-the-shelf, accessible” option. The reference, of course, was to the competition Lunsumio now faces as it takes on Novartis’ Kymriah, Bristol Myers Squibb’s Breyanzi and Gilead’s Yescarta.
Smart Insulin Pen Device Gets New FDA Clearance
Mallya is coming to America. The U.S. Food and Drug Administration (FDA) recently granted clearance to the smart medical device from the French manufacturer Biocorp. First launched in French pharmacies in spring 2021, Mallya is an add-on that fits atop a range of disposable insulin pens to transmit injection data via Bluetooth to companion software in real time. According to its makers, Mallya is the first system approved for use in the United States that can automatically connect different kinds of insulin and GLP-1 medications that lower blood glucose levels.
MediWound Announces FDA Approval of NexoBrid® for the Treatment of Severe Thermal Burns in Adults
“We are pleased and excited that the FDA has approved NexoBrid, an innovative, non-surgical alternative for the treatment of severe burn injuries,” said Ofer Gonen, Chief Executive Officer of MediWound. “We appreciate and thank the burn patients who participated in our trials, the clinical investigators, and our researchers for their commitment and efforts to attain this significant achievement. We also thank our partner, BARDA, for their unwavering support since 2015, and our commercial partner, Vericel, who will launch NexoBrid in the U.S.
After FDA’s Ukoniq pull, TG Therapeutics wins commercial mulligan with Briumvi to enter crowded MS arena
Half a year after the FDA snatched back an approval for TG Therapeutics’ first-in-class lymphoma med Ukoniq, the company has landed a regulatory win. Wednesday, TG snared U.S. approval for its monoclonal antibody ublituximab in the crowded relapsing multiple sclerosis field. Now christened Briumvi, TG’s CD20-targeting drug will rely on its twice-a-year dosing to provide a potential convenience edge against a host of competitors like Roche’s Ocrevus, Novartis’ Kesimpta and Biogen’s Tecfidera.
// Research & Development
ML Model Accurately Predicts Need for Massive Transfusion During Surgery
Published in JAMA Network Open, a recent study described how adding preoperative data and intraoperative hemodynamic monitoring data to a machine learning (ML)-based prediction model led to accurate real-time predictions of the need for massive transfusion during surgery. Many healthcare providers are using predictive analytics to improve the timeliness and quality of treatment while reining in costs.
Pfizer says hemophilia B gene therapy controlled bleeding in key study
Drugmakers have long studied hemophilia gene therapies, seeking to replace the infusions that people with the disease take for life to control their bleeding. In recent years, their research has delivered clinical successes in both the “A” and “B” forms of the disease and, in 2022, the first regulatory approvals. Health authorities in Europe in August authorized BioMarin Pharmaceutical’s gene therapy for hemophilia A, while last month the Food and Drug Administration approved CSL and UniQure’s gene therapy for hemophilia B.
Pfizer’s hemophilia B gene therapy is better than standard of care in phase 3 trial
Pfizer’s gene therapy for severe hemophilia B has not only matched standard of care but beat it in a phase 3 study testing whether the treatment can help patients make the protein responsible for blood clotting on their own. Fidanacogene elaparvovec, which Pfizer licensed from Spark Therapeutics, was being tested in the late-stage trial with 45 male patients who have moderately severe or severe hemophilia B.
Despite missing the mark in a phase 2, Lexicon is standing by its neuropathic pain therapy
Lexicon Pharmaceuticals has missed the mark in a phase 2 aiming to treat a common complication of shingles known as postherpetic neuralgia. However, the Texas biotech is standing by its therapy, saying the trial still demonstrates “clear evidence of effect.”
Researchers Develop ML Tool for Studying Characteristics of Single Cells
Researchers from the Children’s Hospital of Philadelphia (CHOP) and the New Jersey Institute of Technology (NJIT) developed new machine-learning (ML) software to analyze multiple characteristics of a single cell, which may lead to insights into the causes of genetic disease. The press release states that the tool enables researchers to perform single-cell sequencing, which helps them see how one change in a cell can lead to several others and how specific aspects of a cell impact its interaction within its microenvironment.
// Politics
‘Rife with irregularities’: Congressional investigation reveals FDA’s approval of Aduhelm marked by secret discussions, breaches of protocol
The Food and Drug Administration’s review and approval of a controversial Alzheimer’s drug was “rife with irregularities,” including dozens of undisclosed calls and emails with Biogen and an “inappropriate” level of coordination between the drugmaker and regulators, a congressional investigation concluded.
Biogen, FDA’s inappropriate Aduhelm coordination detailed in blistering congressional report
A scathing congressional report has detailed communications described as “inappropriate” and “atypical” between Biogen and the FDA regarding the approval of Alzheimer’s disease drug Aduhelm plus the decision-making behind the pricing and marketing of the now-benched medicine.
FDA, Biogen, and an Alzheimer’s drug approval: 8 key takeaways from congressional investigation
Congressional investigators sorted through more than 500,000 pages of documents from the Food and Drug Administration and Biogen to chronicle how a controversial Alzheimer’s disease drug was approved, and how it went from what was supposed to be the biggest drug launch in history to a financial catastrophe.
Justice Dept. Sues AmerisourceBergen Over Role in Opioid Crisis
The Justice Department filed a lawsuit on Thursday against AmerisourceBergen, one of the country’s largest pharmaceutical distributors, accusing the company of knowingly distributing opioids that were later resold illegally.