// Biotech and Pharma Therapeutics

October 10, 2022 / RNA / Startup / Chronic Liver Disease –Ochre Bio conducts its research with donor livers. These livers aren’t suitable for organ donation, but they can still be used for drug research. These livers are kept “alive” for days at a time by machines that mimic physiological conditions. Using machine learning, the biotech analyzes the liver data in order to identify and validate targets for its drugs. The company describes its approach as “deep phenotyping.” The company is developing therapies to treat chronic liver disease. Read Full Article on MedCity News >>
Stalicla signs licence deal for Evgen Pharma’s lead asset SFX-01 October 11, 2022 / Stalicla / Licensing Deal – More than a year after Eli Lilly received a subpoena centered on quality control snafus and alleged document tampering in New Jersey, U.S. drug inspectors have turned up a fresh batch of problems at the same site. The U.S. FDA has logged multiple new quality control lapses at Lilly’s plant in Branchburg, New Jersey, Reuters first reported, citing an agency report seen by the news outlet. Read Full Article on Fierce Pharma>>
Positive results from trial of Arecor’s insulin candidate AT247 October 11, 2022 / Inslin / Arecor / AT247 – AT247 is an ultra-rapid acting novel formulation of insulin that has been designed to accelerate the absorption of insulin post injection. The superior pharmacokinetics (PK) and pharmacodynamics (PD) profile of a single AT247 dose, compared with gold standard insulins NovoLog and Fiasp, has been previously demonstrated in a phase 1 study. This second clinical study further confirms that AT247 has a superior PK profile compared with NovoLog and Fiasp – showing a statistically significant difference when meeting the trial’s co-primary endpoint. AT247 also demonstrated a statistically superior early glucose lowering effect in the trial’s second primary endpoint compared with NovoLog. The trial further demonstrated that AT247 can be safely and effectively delivered via continuous SC infusion using an insulin pump. Read Full Article on Pharma Times>>
AlzeCure demonstrates disease-modifying effects of NeuroRestore ACD856 October 11, 2022 / AlzeCure / NeuroRestore / ACD856 / –Data from the study shows that ACD856 has a potential neuroprotective effect in a cellular mode. AlzeCure – a company that develops a broad portfolio of small molecule candidate drugs for diseases affecting the central nervous system – has released data. The abstract demonstrates new preclinical data with the company’s leading drug candidate NeuroRestore ACD856, which is being developed with a focus on Alzheimer’s disease. The abstract – ACD856 improves mitochondrial function and increase BDNF levels in primary cortical neurons – was presented by Pontus Forsell, head of research and discovery at AlzeCure, and includes new preclinical data on ACD856, the lead drug candidate in the NeuroRestore project. Read Full Article on Pharma Times>>

// 4th Industrial Revolution

How telemedicine and digital therapeutics can improve orthopedic care and PT
October 11, 2022 / Digital Therapeutics / Telemedicine / Orthopedic care –Minority and lower-income populations are less likely to have orthopedic surgery – and more likely to experience poor outcomes when they do. Untreated musculoskeletal conditions can result in sedentary behavior that leads to or worsens co-morbidities, including diabetes, obesity, depression and opioid misuse. Access challenges are partly to blame. Disadvantaged populations face many barriers to care, including low referral rates, lack of Medicaid acceptance and transportation difficulties. Read Full Article on Healthcare IT News >>
How medtech can crack the patient engagement conundrum and transform the patient experience
October 11, 2022 / Digital Health / Medtech –Patient engagement is a critical component of remote patient monitoring. Part of real world evidence (RWE) collection involves patient-reported data. But if medtech companies are to build a collaborative relationship with patients, building trust is critical to get opt-in to share their data. If patients understand why their data is needed and how it will be used, they will be more likely to view this request in a positive light. They’ll see this as a way to help them manage their condition and not as a cynical transactional relationship. As well as ensuring that accurate, timely data is collected from devices, capturing patient reported outcomes (for example, chronic pain) is also important to build a fuller view of the patient. In a recent webinar on remote patient monitoring and RWE, sponsored by Huma, panelists discussed how they are using patient engagement to trigger earlier interventions, support patients through their care journey and de-escalate the stress and anxiety often associated with managing acute and chronic conditions. Full Article on MedCity >>
Capital Blue Cross to bring low-cost pharmacy option to members and non-members in 2023
October 11, 2022 /Low cost pharmacy / Capital Blue Cross / Digital Health –Come 2023, members of Capital Blue Cross—and even those that are not—will be able to bypass their traditional pharmacy benefits manager and buy prescription drugs from Mark Cuban Cost Plus Drug Company. The Dallas, Texas-based company launched in 2022 as a disruptor to the traditional pharmacy industry, and last week both companies announced that Capital Blue Cross is the first health plan to formally collaborate with the pharmacy startup. The Harrisburg, Pennsylvania-based insurer serves 21 counties in Central Pennsylvania. Read Article on MedCityNews >>
Forge Biologics’ Gene Therapy Shows Positive Signals in Krabbe Disease
October 11, 2022 / Forge Biologic / Gene Therapy / Krabbe Disease – Data from a Phase I/II study of an investigational adeno-associated virus developed by Forge Biologics for infants with Krabbe disease showed positive signals, the company announced Tuesday. Krabbe disease is a fatal neurodegenerative disorder. Two patients treated with FBX-101, a novel gene therapy, have shown increased galactocerebrosidase (GALC) enzyme activity in plasma and cerebrospinal fluid, Ohio-based Forge reported. The patients are also showing normal white matter myelination and normalization of motor development – which is of significant importance due to the disease’s tendency to rob children of these milestones. Read Full Article on BioSpace >>
Developing an organic transmembrane device to host and monitor 3-D cell cultures October 12, 2022 / Transmembrane Device / 3-D cell cultures –Researchers have used 3D cell culture models in the past decade to translate molecular targets during drug discovery processes to thereby transition from an existing predominantly 2D culture environment. In a new report now published in Science Advances, Charalampos Pitsalidis and a research team in physics and chemical engineering at the University of Science and Technology in Abu Dhabi, UAE and the University of Cambridge describe a multi-well plate bioelectronic platform named the e-transmembrane to support and monitor complex 3D cell architectures. Read Full Article on Phys.org >>

// Business & Markets

Illumina and AstraZeneca look to drug target discovery with ‘pivotal research collaboration’
October 11, 2022 / Illumina / AstraZeneca / Collaboration – The collaboration will evaluate whether a combined framework of these technologies can increase the yield and confidence of target discovery to find promising drugs based on human omics insights. “Illumina and AstraZeneca are uniquely positioned to improve the efficiency of pharma pipelines by leveraging industry-leading abilities to identify genetic variants that contribute to human disease,” said Joydeep Goswami, chief strategy and corporate development officer, interim chief financial officer at Illumina. Read Full Article on Labiotech >>
PureTech and Nektar Call Off Rumored Merger
October 11, 2022 / PureTech / Nektar / Merger –Last week, rumors spread that PureTech Health was preparing to merge with Nektar Therapeutics. Now the deal, never officially announced, has been called off. When the rumors first broke, PureTech released a statement on Oct. 6 confirming the two companies were in negotiations, but that they were “non-binding” and “there can be no certainty that any firm offer will be made, nor as to the terms of any such offer.” Tuesday, in a K-6 filing and release, PureTech announced the two companies had agreed to terminate discussions. Read Full Article on BioSpace >>
NeuroPace Announces First Patient Implanted in NAUTILUS Pivotal Study for the Treatment of Idiopathic Generalized Epilepsy (IGE)
October 11, 2022 / NeuroPace / NAUTILUS / Idiopathic Generalized Epilepsy – NeuroPace, Inc. (Nasdaq: NPCE), a commercial stage medical device company focused on transforming the lives of people living with epilepsy, today announced the first patient implanted in the NAUTILUS clinical study. The pivotal study will evaluate the safety and effectiveness of the RNS® System in individuals aged 12 and older with drug-resistant idiopathic generalized epilepsy (IGE), also known as primary generalized epilepsy. The first procedure took place at Vanderbilt Health in Nashville, Tenn. with co-investigators Angela Crudele, MD, and Dario Englot, MD, PhD. “Today, we have limited treatment options for patients who have drug-resistant, idiopathic generalized epilepsy,” said Dr. Crudele. “This condition can be very difficult to treat and has a significant impact on a patient’s and family’s quality of life. I am excited about the possibility of having an FDA-approved treatment for this population, such as a brain-responsive neuromodulation, and giving these patients a better future.” Read Full Article on BioSpace >>
Bayer, Hua Medicine snag first-in-class nod in China for Roche castoff diabetes drug, but commercial value uncertain
October 10, 2022 / Bayer / Hua Medicine / Type 2 Diabetes / Drug Approvals – China’s Hua Medicine has spent over a decade on one medicine—a diabetes candidate in-licensed from Roche. Now under a partnership with Bayer, the drug has snagged a global first-in-class nod in China. Hua’s dorzagliatin is a glucokinase activator (GKA), and the Chinese green light ends a 10-year drought in Type 2 diabetes in terms of no medicines with new mechanisms of action entering the market, the company said (PDF) Sunday. The drug is allowed for use either alone for new patients or in combination with metformin for treatment-experienced patients.Read Full Article on Fierce Pharma >>
In $500M expansion, Merck opens one Singapore facility and breaks ground on another October 5, 2022 / Merck / Gardasil / Keytruda / Prefilled Syringe – Wednesday, the New Jersey-based company opened a new secondary packaging facility to churn out vaccines and biologics. It also broke ground on a plant that will produce inhalers and is set for completion in 2026. Merck’s fill-finish facility in Singapore now includes a vial-packaging line to produce cancer treatment Keytruda and three syringe-packaging lines to manufacture another Merck mega blockbuster, HPV vaccine Gardasil. The site began the sterile fill of Keytruda into vials last year. Its expansion includes cold storage and a new quality control laboratory. Read Full Article on Fierce Pharma >>

// Legal & Regulatory

U.S. FDA Accepts for Priority Review the Supplemental Biologics License Application for Gilead’s Trodelvy® for Pre-Treated HR+/HER2- Metastatic Breast Cancer
October 11, 2022 / FDA / HR/+HER2 / – Gilead Sciences, Inc. today announced the U.S. Food and Drug Administration (FDA) has accepted for priority review the supplemental Biologics License Application (sBLA) for Trodelvy® (sacituzumab govitecan-hziy) for the treatment of adult patients with unresectable locally advanced or metastatic hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative (IHC 0, IHC 1+ or IHC 2+/ISH–) breast cancer who have received endocrine-based therapy and at least two additional systemic therapies in the metastatic setting. The FDA grants priority review for therapies that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. The Prescription Drug User Fee Act (PDUFA) target action date is currently set for February 2023. Read on BioSpace >>
FDA nixes EUA ambitions for Eiger’s COVID treatment, proposes full approval pathway instead
October 10, 2022 / FDA / Covis’ Makena / Withdraw –Makena (hydroxyprogesterone caproate), as well as five generic forms of the drug, is the only drug approved by the FDA to decrease the risk of preterm birth in women with a singleton pregnancy with a history of spontaneous preterm birth. AMAG Pharmaceuticals marketed the drug until Covis acquired it in Oct. 2020. Specifically, the drug is an injectable progestin therapy to decrease preterm births, or births that occur before 37 weeks of pregnancy have been completed. Preterm babies can develop health issues, including respiratory distress syndrome, neonatal sepsis and intraventricular hemorrhage. Read on Fierce Biotech >>
Blue Shield of California sues state over Medi-Cal changes that benefit Kaiser Permanente
October 10, 2022 / Medi-Cal / Blue Shield of California / Kaiser Permanente / Doctors –Blue Shield of California sued the state’s Department of Health Care Services (DHCS) over an alleged failure to produce documents about how it plans to contract with for-profit health insurers across the nation who want to participate in the state’s Medicaid program, Medi-Cal. A new law goes into effect in January 2024 that would affect approximately 14.6 million lower-income individuals covered by Medi-Cal. However, the state has failed to document exactly what that impact might be, according to BSC. Read on Fierce Healthcare >>
Nanoscope’s optogenetic therapy receives Fast Track designation from FDA
October 11, 2022 / Nanoscope / Optogenetic Therapy / FDA – Clinical-stage biotechnology firm Nanoscope Therapeutics has received Fast Track designation (FTD) from the US Food and Drug Administration (FDA) for its optogenetic monotherapy, MCO-010, to treat retinitis pigmentosa (RP). MCO-010, the lead asset of the company, is an ambient-light activatable multi-characteristic opsin (MCO) monotherapy, which has been designed to restore vision in blind patients. It has also received orphan drug designations from the US FDA for the treatment of RP and Stargardt. Read on Pharmaceutical Technology >>
AstraZeneca and GSK settle talent feud in court — again
October 11, 2022 / Pfizer / Biohaven Pharmaceuticals – AstraZeneca and GSK have settled their latest talent skirmish, clearing the way for 18-plus-year AstraZeneca veteran Chris Sheldon to start a new gig at the rival pharma next month. Read on Endpoints News >>

// Research & Development

Albireo Drug Makes “Profound” Difference in Rare Alagille Syndrome October 11, 2022 / Albireo / New Drug / Alagille Syndrome – Albireo Pharma reported positive Phase III ASSERT data of Bylvay for Alagille syndrome (ALGS) Tuesday. The company is headed to U.S. and European regulators with expectations of regulatory filings in Q1 of 2023. In a conference call, Ron Cooper, president and CEO of Albireo, said he was “pleased to announce a positive outcome for [the] trial, the first and only gold-standard” study for Alagille syndrome. Read Full Article on BioSpace >>
Forge Biologics’ Gene Therapy Shows Positive Signals in Krabbe Disease October 11, 2022 / Forge Biologics’ / Gene Therapy / Krabbe – Data from a Phase I/II study of an investigational adeno-associated virus developed by Forge Biologics for infants with Krabbe disease showed positive signals, the company announced Tuesday. Krabbe disease is a fatal neurodegenerative disorder. Two patients treated with FBX-101, a novel gene therapy, have shown increased galactocerebrosidase (GALC) enzyme activity in plasma and cerebrospinal fluid, Ohio-based Forge reported. Read Full Article on BioSpace >>
TILT Biotherapeutics Announces First US Patient Dosed in Immunotherapy Clinical Trial in Ovarian Cancer
October 11, 2022 / TILT Biotherapeutics / Patient / Clinical Tri / Ovarian Cancer – This open-label, phase I, dose-escalation, multicenter, and multinational, clinical trial of TILT-123 in combination with MSD’s (a tradename of Merck & Co., Inc., Rahway, NJ., USA) anti-PD-1 therapy, KEYTRUDA® (pembrolizumab), is for platinum resistant or refractory ovarian cancer (PROTA, also called TILT-T563). The US patient, treated at the Mayo Clinic, should complete enrollment in the first cohort of three patients, the other two located in Finland. The Phase I trial is expected to enroll up to 15 patients. Read Full Article on BioSpace >>
Major worldwide study clusters long COVID symptoms into 3 groups
October 11, 2022 / University of Washington / Long COVID / World Health Organization / Chronic Fatigue – Healthcare systems around the world facing the challenge of diagnosing long COVID might benefit by separating the symptoms of the disease into three general clusters: persistent fatigue with bodily pain or mood swings, cognitive problems and ongoing respiratory problems that persist at least three months after acute infection. That’s what hundreds of expert authors known as the Global Burden of Disease Long COVID Collaborators did in a Bayesian meta-regression analysis published Monday in JAMA Network. Read Full Article on Fierce Healthcare >>
Yale New Haven Health signs to acquire Connecticut systems from Prospect Medical Holdings
October 7, 2022 / Yale New Haven Health / Prospect Medical Holding / Mergers and Acquisitions – Yale New Haven Health has signed an agreement to acquire two Connecticut health systems, Waterbury HEALTH and Eastern Connecticut Health Network), from Prospect Medical Holdings. The deal would give Connecticut’s largest health system the businesses, real estate, physician clinic operations and outpatient services of three hospitals: 357-bed Waterbury Hospital, 249-bed Manchester Memorial Hospital and 102-bed Rockville General Hospital. Also included are Prospect Provider Group of Connecticut and Visiting Nurse and Health Services of Connecticut, according to a release. Read Full Article on Fierce Healthcare >>
AstraZeneca’s COVID-19 nasal vaccine spray comes up a bust in small study
October 11, 2022 / AstraZeneca / Oxford University / COVID-19 – Since the coronavirus enters the body through the nose, researchers have figured a nasal spray may be one way to defend against its spread. But developing an effective point-of-entry vaccine can be elusive—even for companies that are able to throw the most money at the job. Read Full Article on Fierce Pharma >>

// Politics

Biden administration finalizes rule to get rid of ACA’s ‘family glitch’
October 11, 2022 / US Department of the Treasury / Affordable Care Act (ACT) / Health Insurance Exchanges / Insurance Premiums – The Biden administration finalized on Tuesday a rule that fixes a glitch in the Affordable Care Act that can inadvertently raise insurance costs on certain families. The Treasury Department released a final rule on Tuesday that targets a lingering issue that has led some low-income families to not receive premium assistance. The new rule will go into effect next month. “About one million Americans will either gain coverage or see their insurance become more affordable as a result of the new rule,” said President Joe Biden in a statement Tuesday. “It builds on our progress so far, which has brought the rate of uninsured Americans to a record-low eight percent. Read Full Article on Fierce Healthcare >>
Inflation drives pharma manufacturing focus on sustainability
October 11, 2022 / Inflation / Pharma Manufacturing / Sustainability – “The problem is that it’s not just the cost of materials going upwards, but also that the cost of energy is only spiralling upwards,” says Elisabeth Stampa, the president of Medicines for Europe, the nonprofit that wrote the recent letter. The difficult financial climate and growing calls for accountability for manufacturing’s impact on the environment mean that contract development and manufacturing organizations (CDMOs) must evaluate how to consider sustainability despite potential financial constraints. Read Full Article on Pharmaceutical Technology >>
Maiden Pharma row: Govt to review product quality of PLI scheme beneficiaries
October 10, 2022 / Product Quality / Department of Pharmaceuticals – The Department of Pharmaceuticals (DoP), under the government of India, has asked the state drug controllers to review manufacturing units and product quality of the companies benefitting from the central government run Production Linked Incentive Scheme (PLI) for pharmaceuticals. The move follows the controversy surrounding Delhi-based Maiden Pharma’s cough syrups, which have been linked to deaths of over 66 children in Gambia. Calling the development a “wake up call” for Indian pharmaceutical industry, officials in the DoP have also urged the states to verify, if the products manufactured and exported from pharmaceutical companies come under the Micro, Small and Medium Enterprises (MSME) comes -PLI scheme. Read Full Article on BusinessToday.In >>