// Biotech and Pharma Therapeutics
CRISPR offers genetic clues to Alzheimer’s in brain’s cleaning crew
August 16, 2022 / CRISPR / Neuroscience / Alzheimer’s
Microglia clear out the brain’s cellular debris and play a central role in maintaining the health of neurons. Changes in these special immune cells could act as potential drivers of brain diseases such as Alzheimer’s disease. Now, a research team led by the University of California, San Francisco (UCSF) has identified genetic regulators of microglia’s disease states.
Sanofi ends development of breast cancer drug in another blow to pipeline
August 17, 2022 / Sanofi / Amcenestrant / Cancer
Sanofi said Wednesday it’s giving up on a next-generation breast cancer drug called amcenestrant after a second major trial failed. The company’s shares fell by more than 6% in early trading on the Nasdaq stock exchange. The French drugmaker was testing the medicine in combination with another therapy in patients with a common form of advanced breast cancer. An interim analysis of the Phase 3 AMEERA-5 trial found results weren’t strong enough to continue, and an independent board recommended halting the study and moving patients to other treatments.
Gilead to buy remaining worldwide rights of Trodelvy from Everest Medicines
August 16, 2022 / Gilead Sciences / Everest Medicines / Trodelvy
Gilead Sciences, Inc. announced an agreement with Everest Medicines to transfer all development and commercialization rights to Gilead for Trodelvy (sacituzumab govitecan) in Greater China, South Korea, Singapore, Indonesia, Philippines, Vietnam, Thailand, Malaysia and Mongolia. In China mainland and Singapore, Trodelvy is approved for the treatment of adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) who have received two or more prior systemic therapies, at least one of them for metastatic disease. Gilead continues to work closely with regulatory bodies in Hong Kong, South Korea and Taiwan, where New Drug Applications, filed by Everest Medicines for metastatic TNBC, are currently under review.
Bluebird bio’s beta-thalassaemia gene therapy receives FDA approval
August 18, 2022 / Bluebird Bio / Mathai Mammen
Blubird bio’s Zynteglo (betibeglogene autotemcel) – also known as beti-cel – has been approved by the US Food and Drug Administration (FDA) for patients with beta-thalassaemia (beta-thal) in adult and paediatric patients who require regular red blood cell transfusions. Beta-thal is a rare genetic blood disease caused by a gene defect that impairs the ability of red blood cells to produce haemoglobin. Patients with the most severe form of beta-thal develop life-threatening anaemia and have to undergo regular blood transfusions, a lengthy process typically needed every two to five weeks.
Bayer parts ways with Exscientia, leaving one target in AI drug hunter’s hands
August 18, 2022 / Bayer / Exscientia / Artificial Intelligence
Bayer and Exscientia are parting ways on an artificial intelligence collaboration that saw the pair work together to find cardiovascular and oncology targets. Exscientia revealed the end of the collaboration in a second-quarter earnings report issued after-market Wednesday. The U.K.-based AI company said the pact was mutually ended following the achievement of a drug discovery milestone. Exscientia will retain the option to develop one of the two targets that was being worked on in the collaboration.
// 4th Industrial Revolution
AR Headset Helps Surgeons Place Implants: Interview with Louie Vogt of Enovis Surgical
August 17, 2022 / Enovis / MedTech / DJO / ARVIS
Enovis, a medtech company based in Delaware formerly known as DJO, recently announced the launch of ARVIS (Augmented Reality Visualization and Information System), an augmented reality technology that is designed to assist surgeons during implant placement in the hip and knee. The hands-free technology consists of an eyepiece that is mounted on a surgical helmet that provides the surgeon with real-time information about the position of an implant with respect to patient anatomy.
Improving health outcomes: Where now for cardiology?
August 16, 2022 / iRhythm Technologies / ZIO / Artificial Intelligence
n September 2020, iRhythm Technologies was named as a winner of the NHSX (now the NHS Transformation Directorate) AI Award, enabling the digital healthcare company to trial its Zio service at scale across a range of selected trusts in England. Before Zio’s implementation, Holter monitors were the ‘norm’ for monitoring suspected arrythmias and heart conditions. These can be viewed as outdated, burdensome, and noticeable due to their size and the need to have wires connected to electrodes. The short testing period typically seen with a Holter (24-48 hours), and low patient compliance had also led to concerns about accuracy, leading to repeat testing, or even misdiagnosis. This demonstrated a need for change within the health landscape, especially for those patients requiring longer monitoring periods.
Amazon is getting into mental health care with a Ginger partnership
August 16, 2022 / Amazon Care / Virtual Healthcare / Ginger
Amazon is everywhere: in the doctor’s office, in people’s homes, in their shopping carts, and now — in their therapist’s office, too. Amazon’s virtual healthcare program, Amazon Care, now includes a partnership with mental health company Ginger, according to an Amazon Care webpage. The new offerings were first reported by Insider.
Tick Tock (or TikTok) for US Health Care
August 16, 2022 / TikTok / ByteDance / Xiaohe Health
TikTok’s parent company ByteDance is actually buying hospitals, through two of its health subsidiaries. As first reported by South China Morning Post, and subsequently confirmed as a $1.5b deal by Bloomberg, ByteDance bought Beijing-based Amcare Healthcare, which runs eight women’s and children’s hospitals in four Chinese cities. As a private system, it targets expats and high income locals. This is not ByteDance’s first foray into healthcare; in 2020 it bought Xiaohe Medical, an internet hospital, as well as a medical information site and a telehealth service. It is using its AI expertise to aid in drug discovery. Its health business are under the umbrellas of Xiaohe Health and Xiaohe Health Technologies.
Roon wants to educate patients with freshly sourced info on their conditions
August 12, 2022 / Roon / Medical Education / Curation
As individuals try to manage medical information and understand their conditions, many typically turn to Google or WebMD — neither of which does much to verify or provide the latest information. But Roon plans to change this with a medical education platform for vetted information, sourced strictly from doctors, patients, and caregivers. By curating the data it makes available around individual conditions, Roon is meant to reassure patients and caregivers that it’s accurate and well sourced.
// Business & Markets
Orna rounds up $371M via series B, deal with Merck for circular RNAs
August 16, 2022 / Biotech / Merck / Orna / Vaccines
Biotech keeps an unpartnered ‘crown jewel’ in-house, taps pharma to advance vaccine projects and explore cancer treatments. A deal with Merck and a $221 million series B round will allow Orna’s lead circular RNA program to reach the clinic, while giving the pharma access to a platform to develop vaccines and therapeutics for infectious diseases and cancer.
Gilead, with new results, doubles down on breast cancer drug
August 16, 2022 / Gilead Sciences / Trodelvy / Everest
Gilead Sciences plans to acquire all remaining worldwide rights to its breast cancer drug Trodelvy through a new agreement announced Monday. The deal with Everest Medicines, a Chinese biotechnology company, will hand Gilead development and commercialization rights to Trodelvy across a large swath of Asia, including Greater China, South Korea, Singapore and Indonesia. In exchange, Everest will receive $280 million upfront and be eligible for as much as $175 million in additional payments that hinge on the drug achieving certain regulatory and commercial goals.
Bristol Myers Squibb successfully completes acquisition of Turning Point Therapeutics
August 18, 2022 / Bristol Myers Squibb / Oncogenesis / Tumors
Bristol Myers Squibb (BMS) has successfully completed its acquisition of Turning Point Therapeutics (Turning Point), further expanding its oncology portfolio, the company announced. Through the all-cash transaction, BMS gains a pipeline of investigational medicines designed to ‘target the most common mutations associated with oncogenesis’, including repotrectinib – a next-generation tyrosine kinase inhibitor targeting the ROS1 and NTRK oncogenic drivers of non-small cell lung cancer (NSCLC) and other advanced solid tumors.
Abbott will invest $450M in Ireland to expand EU production of FreeStyle Libre CGMs
August 18, 2022 / Abbott / Diabetes
Abbott Laboratories said it will invest €440 million ($450 million) in two facilities in Ireland in a bid to increase production of its FreeStyle Libre technology. The investment will support the construction of a new manufacturing facility and the expansion of an existing diabetes care site. The new, 250,000-square-foot facility will add capacity for Abbott’s Libre continuous glucose monitoring system.
3 ways to reform the pharmacy benefits industry
August 17, 2022 / PBMs / INVEST Conference / Health Wildcatters
Pharmacy benefit managers have been in the news a lot lately as they are an important component of the ongoing drug price debate in this country. Consolidation, legislation and litigation pepper the sector. At the upcoming INVEST Digital Health conference in Dallas set for September 28, in collaboration with Health Wildcatters, a panel discussion on the future of pharmacy benefit managers will be part of the larger conversation on employee benefits. One of the panelists for that discussion is AJ Loiacono, CapitalRx CEO.
// Legal & Regulatory
‘A watershed moment for the field of gene therapy’: bluebird bio’s gene therapy Zynteglo receives FDA approval
August 18, 2022 / Zynteglo / Beta Thalassemia / Gene Therapy
The U.S. Food and Drug Administration (FDA) has approved bluebird bio’s Zynteglo (betibeglogene autotemcel), also known as beti-cel: a one-time gene therapy custom-designed to treat the underlying genetic cause of beta thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.
Menarini Group’s Elacestrant Granted Priority Review by the U.S. FDA for Patients with ER+/HER2- Advanced or Metastatic Breast Cancer
August 11, 2022 / Diagnostics / SERD / Elacestrant
The Menarini Group (“Menarini”), a privately held Italian pharmaceutical and diagnostics company, and Stemline Therapeutics (“Stemline”), a wholly-owned subsidiary of Menarini Group, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for elacestrant, an investigational selective estrogen receptor degrader (SERD), for patients with ER+/HER2- advanced or metastatic breast cancer. The FDA has granted the application Priority Review and assigned a PDUFA date of February 17, 2023.
Astellas’ hot flashes drug will get speedy review at FDA; US opts out of Valneva vaccine
August 18, 2022 / Astellas / Vasomotor Symptoms / Fezolinetant
The FDA will decide on Astellas’ menopausal symptom drug by Feb. 22 of next year, as the Japanese pharma disclosed it had paid about $97 million to get a priority review voucher to speed up the review. Astellas said the agency has accepted the pharma’s application for fezolinetant for the treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause. VMS includes hot flashes and/or night sweats. The company said as many as 80% of women in the US experience those symptoms during or after the menopausal transition.
Regulatory tracker: Roche’s Polivy poised for $2B lymphoma verdict; Merck, AZ’s Lynparza nabs speedy prostate cancer review
August 16, 2022 / Roche / Polivy
The FDA has received Roche’s filing for Polivy and Rituxan, plus cyclophosphamide, doxorubicin and prednisone, in patients with previously untreated diffuse large B-cell lymphoma (DLBCL). With Roche’s application in hand, the FDA has set up a regulatory action date of April 2, 2023. Analysts at Jefferies have previously estimated Polivy’s frontline label expansion could mean $2.1 billion in peak sales for the drug, which was first approved in 2019.
This Week at FDA: Amazon warning letter, ANDA amendments, and more
August 12, 2022 / Amazon / Ariella Naturals / Justified Laboratories
Earlier this week, FDA sent warning letters to three companies, including online retail giant Amazon, for selling unapproved drugs for mole and skin tag removal. The other companies cited include Ariella Naturals and Justified Laboratories. In its warning letter to Amazon CEO Andy Jassy, FDA said it purchased the mole and skin tag remover products from Amazon’s website. The agency also notes that while the products were distributed by Amazon “on behalf of third parties … Each of the products was ‘fulfilled’ by Amazon.”
// Research & Development
Recent Advances In Parkinson’s Disease
August 17, 2022 / Parkinson’s / Study
Parkinson’s disease is a progressive neurodegenerative disease that induces symptoms of shaking, stiffness, uncontrollable movement, poor coordination, and balance. Development of Parkinson’s disease occurs at the age of 60 onward. This brain disorder develops as early as 50 years old in 5-10% of cases. Parkinson’s affects nerve cells deep in the brain called basal ganglia. These cells are responsible for producing dopamine and transmitting messages for bodily functions.
Genuv Cancer Drug Shows Early Potential in Alzheimer’s
August 18, 2022 / Genuv / Mekinist
South Korea-based Genuv Inc. announced the publication of a preclinical study showing that Mekinist (trametinib), approved by the FDA for melanoma, has a neuroprotective effect in Alzheimer’s disease. The data, published in Nature Molecular Psychiatry, revealed that in mice treated with Mekinist, brain cells became more adept at getting rid of clumps of protein, greatly reducing amyloid beta plaques.
Powerful new antibody neutralizes all known SARS-CoV-2 variants
August 17, 2022 / SARS-CoV-2 / Omicron / Antibodies
As SARS-CoV-2 has evolved and mutated, therapeutic antibodies that worked early in the pandemic have become less effective, and newer variants, especially Omicron, have developed ways to evade the antibodies we make in response to vaccines. A new, broadly neutralizing antibody developed at Boston Children’s Hospital could potentially improve our ability to defend against future variants. In tests, it neutralized all currently known SARS-CoV-2 variants of concern, including all Omicron variants.
Jazz takes groundbreaking epilepsy drug into kids in phase III trials
August 18, 2022 / Epidiolex / CBD / Marijuana
Jazz Pharma is breaking new ground with its cannabidiol epilepsy treatment Epidiolex, taking the drug into a Phase III study in children and adolescents. The study will include children and adolescents living with developmental and epileptic encephalopathy, known as epilepsy with myoclonic-atonic seizures (EMAS). In 2018, Epidiolex, developed by GW Pharmaceuticals, became the first cannabis-based drug approved by the FDA and required a reclassification by the U.S. Drug Enforcement Agency prior to commercialization.
Newly discovered peptide shows promising results in preventing seizures; a potential future treatment for epilepsy and Alzheimer’s
August 18, 2022 / A1R / Alzheimer’s / Epilepsy
A study published in The American Society for Clinical Investigation highlights developments made by researchers to develop A1R-CT, a peptide which disrupts the signaling between neurabin (a molecule) and the adenosine 1 receptor (A1R). This peptide can be administered through a nasal spray to try and prevent seizures related to epilepsy and Alzheimer’s Disease (AD).
Amgen, amid $10B fight with IRS, lands in Senate committee’s tax probe
August 12, 2022 / IRS / AMGEN
The Senate Finance Committee’s probe into U.S. drugmakers’ tax practices has intensified, as Amgen—already embroiled in an Internal Revenue Service (IRS) investigation over the transfer of U.S. profits to Puerto Rico—now finds itself in the crosshairs of the committee’s chair, Senator Ron Wyden. Amgen has paid an average effective tax rate of just 12% over the past four years, Wyden wrote (PDF) in a Thursday letter to the company’s CEO, Robert Bradway. Now, the Democratic senator is asking for details on Amgen’s tax tactics as part of a wider investigation into the 2017 Republican tax law.
The Top Line’ podcast: The brouhaha over the landmark drug pricing bill, what’s behind Pfizer’s $5.4B Global Blood Therapeutics buyout deal, plus more
August 12, 2022 / U.S. Senate / Medicare
Sunday, the U.S. Senate passed the landmark drug pricing bill. While the pharmaceutical industry favors the bill’s Medicare out-of-pocket cap for patients, the industry has also put up a major fight against Medicare pricing negotiations, in fact deeming them “price controls.” The bill’s passage marked a loss for the industry, but analysts aren’t sure how much the legislation will actually cut drug prices. Pfizer is riding high on its COVID-19 windfall. And with all that money in the bank, the Big Pharma has been busy scouring the biopharma landscape for new assets and companies to bring into its fold. The latest in its buying spree is a $5.4 billion acquisition of Global Blood Therapeutics and its marketed sickle cell disease drug.
Pharma’s likely to sue over Medicare negotiation. Here are the arguments they might use
August 18, 2022 / PhRMA / BIO
Democrats finally achieved a win over the pharmaceutical industry this month when they passed the most aggressive drug pricing reform in decades. But their victory could be short-lived. Even before the law passed, the pharmaceutical industry was threatening to sue to block the reforms, with both the brand drug lobby PhRMA and the Biotechnology Innovation Organization issuing veiled promises to “explore every opportunity” or “explore all avenues” to stop the law from taking effect.
Biden signs bill that extends ACA subsidies through 2025, makes drug price reforms
August 12, 2022 / Inflation Reduction Act / Affordable Care Act
“President Joe Biden has signed the Inflation Reduction Act into law, cementing major drug pricing reforms and extended subsidies for plans on the Affordable Care Act exchanges. The $430 billion legislation includes a slew of polices targeting healthcare and climate change, and aims to give Democrats a boost ahead of the midterm elections later this year.