BioNTech Founders Predict Cancer Vaccine Is Only Years Away

 The husband-and-wife team who co-founded BioNTech, the biotechnology company that partnered with Pfizer to develop an effective messenger-RNA (mRNA) shot against COVID-19, has predicted that a cancer vaccine could be widely available within the next decade.

"Yes, we feel that a cure for cancer, or to changing cancer patients' lives, is in our grasp," said Professor Ozlem Tureci during an interview on BBC's 'Sunday with Laura Kuenssberg'.

The cancer vaccine, which would build upon breakthroughs achieved by the scientists during the development of the COVID-19 shot, may be widely available within just eight years, said Professor Ugur Sahin.

"We believe that this will happen, definitely, before 2030," he told Kuenssberg.

The hope is that a vaccine currently in development would train the body to recognize and attack cancers using mRNA technology.

"The goal that we have is that can we use the individualized vaccine approach to ensure that directly after surgery, patients receive a personalized, individualized vaccine, and we induce an immune response that so the T-cells in the body of the patient can screen the body for remaining tumor cells and ideally eliminate the tumor cells," Sahin explained.

BioNTech originally focused on developing mRNA-based technologies for a patient-specific approach to cancer treatment, per The New York Times.

Tureci told Kuenssberg that their experiences working in cancer wards as young physicians, who were frustrated at being unable to offer treatment to oncology patients, drove them toward their work in cancer research.

That work was the "tailwind" for the COVID-19 shot development, which, in turn, now "gives back" to their cancer research, said Tureci.

Kuenssberg asked the couple if there was "still a chance" that the cancer vaccine doesn't work.

"I don't think so," replied Tureci. "Everything we have learned about the immune system and about what we achieve with a cancer vaccine shows, in principle, the clear activity – we can induce those killer T-cells, we can direct them."

Tureci said that it remains to be seen how doctors would use other types of medical interventions in combination with the vaccine and what else needs to be tweaked to ensure that patients are cured.

"Every step and every patient we treat in these cancer trials helps us to understand more about what we are against and how to address that," said Tureci.

https://www.sciencealert.com/biontech-founders-predict-cancer-vaccine-is-only-years-away

NeuroSigma in Commercial Launch of Pediatric ADHD Treatment System

 NeuroSigma, Inc., a Los Angeles-based bioelectronics company, today announced the commercial launch of The Monarch eTNS System at the annual meeting of the American Academy of Child and Adolescent Psychiatry (AACAP) in Toronto. The launch includes a new co-pay program that will allow commercially insured children between the ages of 7 – 12 with an Attention-Deficit/Hyperactivity Disorder (ADHD) diagnosis to obtain the Monarch eTNS® System and the first month’s supply of disposable NS-2 electric patches for $100. The co-pay for each additional month of patches will be $40.

The Monarch eTNS System is a bioelectronic medical device that uses external trigeminal nerve stimulation (eTNS) to treat neurological and neuropsychiatric disorders. In 2019, the Monarch eTNS System became the first-ever medical device cleared by FDA for treating pediatric ADHD. NeuroSigma’s new Pediatric Care Co-Pay Program is the next step in commercializing the Monarch eTNS System, with the goal of making eTNS therapy available to a substantially greater number of children diagnosed with ADHD.

“NeuroSigma’s mission is to facilitate access to eTNS therapy for the millions of children worldwide struggling with ADHD,” said Dr. Colin Kealey, President of NeuroSigma. “Our new Pediatric Care Co-Pay Program is an important milestone towards achieving this goal and will significantly increase the affordability of our therapy for eligible patients and their caregivers in the United States.”

“Given emerging shortages of leading ADHD medications in the United States, the launch of a non-drug alternative to potentially addictive pharmaceutical stimulants is even more imperative in expanding long-term therapeutic options”, added Dr. Ian Cook, NeuroSigma’s Chief Medical Officer.

Attendees of the 2022 AACAP conference can learn more about the Monarch eTNS System and the Pediatric Care Co-Pay Program by visiting NeuroSigma’s booth 312 at the conference. Families interested in the Monarch can call NeuroSigma’s patient hub at 877-765-7660 or simply have their physician complete the Monarch eTNS Prescription Form and fax it to the indicated number.

https://www.biospace.com/article/releases/neurosigma-announces-commercial-launch-of-the-monarch-etns-system-for-the-treatment-of-pediatric-adhd-at-annual-meeting-of-the-american-academy-of-child-and-adolescent-psychiatry/

Biogen-Sage’s Zuranolone Impresses in Phase III Postpartum Depression Trial

 Sage Therapeutics and Biogen’s zuranolone met its primary and key secondary endpoints in the Phase III SKYLARK study of women with postpartum depression (PPD), the companies announced Monday.

After 15 days of follow-up, women who received zuranolone saw a mean decrease of 15.6 points in the 17-item Hamilton Rating Scale for Depression (HAMD-17) compared to their baseline performance. Scores dropped by an average of 11.6 points in placebo comparators. The difference in effect was statistically significant, with a p-value of 0.0007.

Treatment response, defined as 50% decrease in HAMD-17 scores or more, was also better in women treated with 50 mg of zuranolone, an effect that was apparent from the third day of follow-up and persisted through the 28th day. Remission, defined as a HAMD-17 score of 7 or below, was also higher in the zuranolone arm from days 3 through 45.

SKYLARK’s results were presented at the 35th Congress of the European College of Neuropsychopharmacology (ECNP) in Vienna, Austria.

Dr. Kristina Deligiannidis, Professor at the Feinstein Institutes for Medical Research and principal author of the study, called the results “incredibly encouraging,” especially given zuranolone’s quick action. “Rapid symptom relief is critical for women with PPD, because delays in treatment efficacy can negatively impact resolving depressive symptoms and overall clinical outcomes.”

SKYLARK is a randomized, double-blinded and placebo-controlled trial that enrolled 200 participants. Aside from evaluating zuranolone’s effects against depression, the study also used the Hamilton Anxiety Rating Scale and found that the candidate led to relief in anxiety symptoms as early as day three and persisted until the 45th day of follow-up.

Regarding safety, SKYLARK found zuranolone to be generally well-tolerated, and its safety profile was consistent with what had been observed in prior studies. Most of the treatment-emergent adverse events were mild or moderate in severity. The investigators reported four cases of severe side effects in two zuranolone-treated women but deemed unrelated to the drug.

The most common side effects observed following treatment initiation included dizziness, diarrhea, urinary tract infection, nausea, somnolence and COVID-19.

Biogen and Sage already have a rolling new drug application submission for zuranolone with the FDA. The companies are scheduled to complete their filing by the end of the year.

Biogen and Sage first entered into a partnership over zuranolone in Nov. 2020, when the pharma giant made an upfront investment of $1.52 billion to secure exclusive rights over the candidate in the U.S. The deal also includes SAGE-324, a compound under investigation for essential tremors and other neurological disorders.

“The collaboration between Sage and Biogen to jointly develop and commercialize zuranolone brings together the expertise of both organizations to advance the treatment of mental health conditions,” a Biogen spokesperson told BioSpace. “In addition to zuranolone, the collaboration agreement also includes BIIB 124/Sage-324 for essential tremor.”

Nearly a year before, in Dec. 2019, zuranolone failed a Phase III trial for major depressive disorder, forcing Sage to initiate strategic restructuring and lay off 53% of its workforce. The realignment helped the company save $170 million per year, channeling it into its depression, neurology and neuropsychiatry programs.

https://www.biospace.com/article/biogen-sage-s-zuranolone-aces-phase-iii-in-postpartum-depression/

Jaguar Gets Euro Orphan Drug Tag for Second Rare Disease Indication

 MVID, a rare congenital diarrheal disorder (CDD) condition, is a life-threatening autosomal recessive disease that affects newborns and children and leads to significant morbidity and mortality from intestinal failure, including severe secretory diarrhea

Presentation expected in December 2022 of initial results of an investigator-initiated proof-of-concept trial of crofelemer for CDD and short bowel syndrome (SBS) with intestinal failure, supporting the potential for expanded patient access to crofelemer in 2023 in Europe

https://finance.yahoo.com/news/european-medicines-agency-grants-orphan-123000861.html

Medtronic First to Receive FDA Approval for Pacing the Heart's Natural Conduction System

 Medtronic plc (NYSE: MDT) today announced it has received U.S. Food and Drug Administration (FDA) approval for expanded labeling of a cardiac lead that taps into the heart's natural electrical system, giving patients needed therapy while avoiding complications sometimes associated with traditional pacing methods, such as cardiomyopathy.1 Commonly referred to as "conduction system pacing," this approach helps ensure that pacing closely mimics the heart's physiologic contractions, allowing the heart's ventricles to work in coordination.1

Medtronic is the first and only company with therapies approved for conduction system pacing. In 2018, the FDA approved the SelectSecure™ MRI SureScan® Model 3830 cardiac lead for His-Bundle pacing, another form of conduction system pacing. Now this cardiac lead is approved for pacing and sensing at the bundle of His or in the left bundle branch area as an alternative to apical pacing in the right ventricle in a single- or dual-chamber pacing system. These implanted pulse generator systems help patients who experience slow heart rates (bradycardia).

https://finance.yahoo.com/news/medtronic-first-receive-fda-approval-130000695.html

Vera Therapeutics to Present Final Phase 2 Results of MAU868 in Kidney Transplant Recipients

 Vera Therapeutics, Inc. (Nasdaq: VERA), a late-stage biotechnology company focused on developing and commercializing transformative treatments for patients with serious immunological diseases, announced two new abstracts featuring new clinical data on the Company’s two product candidates, MAU868 in kidney transplant and atacicept in immunoglobulin A nephropathy (IgAN). These data are to be presented in oral and poster sessions, respectively, at the American Society of Nephrology Kidney Week 2022 Annual Meeting, being held November 3-6, 2022 in Orlando, Florida. The oral presentation on MAU868 includes final results from the Phase 2 clinical trial of MAU868 versus placebo to treat BKV in kidney transplant patients. The poster presentation on atacicept includes a new analysis of previously presented clinical data from the Phase 2a JANUS clinical trial evaluating atacicept in patients with IgAN.

Details of the presentations are as follows:

Title:		A Randomized Phase 2 Study of MAU868 vs. Placebo to Treat BK Viremia in Kidney Transplant Recipients
Date and Time:		November 5, 2022, 4:48 PM ET
Presentation:		Oral
Session:		Transplantation: Clinical Outcomes and Biomarkers
Abstract Number:		SA-OR43
Location:		W240, Orange County Convention Center, West Building
Presenter:		Stanley C. Jordan, M.D., FASN, FAST, Director of Nephrology & Transplant Immunology, Cedars-Sinai Medical Center, Professor of Pediatrics and Medicine at the David Geffen School of Medicine at University of California, Los Angeles
Title:		Atacicept Reduces Serum Immune Complex Levels in Patients with Immunoglobulin A Nephropathy (IgAN)
Date and Time:		November 5, 2022, 10:00 AM – 12:00 PM ET
Presentation:		Poster
Session:		Glomerular Diseases: IgA and Complement-Mediated GN
Abstract Number:		SA-PO655
Location:		Exhibit Hall, Orange County Convention Center, West Building
Presenter:		Jonathan Barratt, Ph.D., FRCP, The Mayer Professor of Renal Medicine, University of Leicester, U.K. https://www.biospace.com/article/releases/vera-therapeutics-to-present-final-phase-2-results-of-mau868-in-kidney-transplant-recipients-with-reactivated-bk-virus-infection-and-new-analysis-of-phase-2a-janus-trial/

Kineta, Merck Strike Deal to Pair Anti-VISTA Antibody with Keytruda

 Kineta, Inc. struck a collaboration deal with Merck to pair its anti-VISTA monoclonal antibody with Keytruda (pembrolizumab) as a potential treatment for advanced solid tumors, the companies announced Monday. 

Seattle-based Kineta will assess its investigational antibody KVA12123 as a standalone treatment and in combination with Merck's vaunted checkpoint inhibitor against solid tumors. The two companies anticipate the initiation of clinical trials later this year.

KVA12123 is an inhibitor of VISTA. It is a key driver of immunosuppression in the microenvironment of cancer tumors. Kineta noted in its announcement that following treatment with a checkpoint inhibitor such as Keytruda, VISTA is up-regulated. 

The up-regulation is associated with treatment failures and poor prognosis for cancer patients. KVA12123 is designed to bind to VISTA and inhibit that up-regulation.

In preclinical studies, KVA12123 demonstrated promising anti-tumor activity both as a single agent and in combination with PD-1 checkpoint inhibitor therapy. Kineta now hopes to replicate that success in the clinic.

Merck's anti-PD-1 Keytruda has become a powerhouse oncology drug that generated more than $17 billion for the company last year. Since it was first approved in 2014, Keytruda has received 33 approvals across 16 tumor types from the FDA. 

In addition to Kineta, multiple companies evaluate Keytruda in combination with their cancer therapies. Keytruda is slated to become the world's top-selling drug by 2025, according to analyst predictions.

Shawn Iadonato, chief executive officer of Kineta, noted that treatment with checkpoint inhibitors like Keytruda has become a first-line treatment in multiple cancer indications. Despite this class of drugs' success, a significant number of cancer patients still do not respond to the medication. 

Iadonato said in a brief statement that Kineta believes KVA12123 is a potential best-in-class VISTA blocking immunotherapy that could lead to improved treatments for patients. He suggested KVA12123 has the potential to deliver a differentiated product profile across a range of solid tumors.

Initial target indications for KVA12123 include non-small cell lung cancer, colorectal, renal cell carcinoma, head and neck, and ovarian cancer. These indications represent a significant unmet medical need with a large worldwide commercial opportunity for KVA12123.

Kineta will initiate a Phase I/II trial assessing KVA12123, a fully human engineered IgG1 monoclonal antibody. The trial will assess the safety, tolerability, pharmacokinetics and anti-tumor responses of KVA12123 alone and in combination with Keytruda. Topline data is anticipated in late 2023. 

Financial terms of the collaboration were not disclosed. Under terms that were announced, Kineta will be responsible for initiating the Phase I/II clinical study. The trial is expected to begin in the fourth quarter of 2022.

In addition to KVA12123, Kineta has also developed a set of anti-CD27 agonist antibodies. In preclinical studies, Kineta's selected lead anti-CD27 agonist mAbs generated T cell proliferation and secretion of cytokines involved in T cell priming and recruitment. These demonstrated the antibody's ability to provide new anti-tumor responses.

https://www.biospace.com/article/kineta-strikes-deal-to-pair-its-anti-vista-antibody-with-merck-s-keytruda/