Sonoda Syndrome

Children with a severe form of epilepsy should be vaccinated against the flu due to the high risk of seizures being triggered by an influenza infection, according to a new study. Children with a severe form of epilepsy should be vaccinated against the flu due to the high risk of seizures being triggered by an influenza infection, according to a new study. The research, led by the Murdoch Children's Research Institute and the University of Melbourne, found the safe administration of the seasonal influenza vaccine should be a priority in those with SCN1A-postive Dravet syndrome given the likelihood of severe neurological symptoms and complications such as worsening seizures, deteriorating language and motor skills and even death after catching the flu. Paediatric neurologist, Dr Katherine Howell from the Murdoch Children's said the decision for parents to vaccinate children with this syndrome was complex because seizures could be triggered by both infection and vaccination. Dr Howell said despite the syndrome being associated with high rates of prolonged seizures during infections, the impact of influenza had not been previously studied. The research, published in Neurology, involved children with SCN1A-positive Dravet syndrome who had a confirmed influenza infection at The Royal Children's and Austin Hospital. Researchers found 21 children caught influenza 24 times, with brain complications reported in 88 per cent of cases. All presented to hospital with 75 per cent recovering quickly but death or long-term brain complications occurred in one in five infections. Between them they received 60 influenza vaccinations with most tolerating the vaccine well. "Concerns about giving the flu vaccine and incomplete routine immunisations are common in this patient group due to the risk of seizures after vaccination," Dr Howell said. However, because this syndrome is also associated with a high risk of seizures during infections, it highlights the critical need to protect patients from the complications of vaccine-preventable infections like the flu. Our research highlights that the benefits of flu vaccines for these children far outweighs the risks of seizures being triggered following vaccination." SCN1A-postive Dravet syndrome, the most common severe form of genetic epilepsy, occurs in one in 15,000 children. Melbourne mum Renae Caddy's son Fraser, 4, was diagnosed with SCN1A-Dravet syndrome after experiencing seizures as a baby. Renae said her son now received a flu shot every year to prevent prolonged seizures, which are largely triggered by an infection. At 11 months old a case of the flu caused Fraser to have a two-hour seizure and required four days of hospital treatment. "The seizures have gone from monthly to weekly, but they are much shorter now, resolving after a few minutes rather than a couple of hours," she said. But if he has a viral infection he will seize for as long as his temperature is high and may need a lot of medication and even intubation to get his seizures under control. "It can take Fraser up to four days to get back to normal after he experiences a severe seizure so anything we can do to avoid him getting sick we will do." Renae said all his influenza vaccines had been well-tolerated with no seizures. "The research is reassuring for parents that it's beneficial for children with this condition to have the flu shot to try and avoid an onset of seizures caused by an infection, which are damaging and terrifying to watch," she said. University of Melbourne Professor Ingrid Scheffer said the findings would change clinical practice. "Identifying safe strategies and strongly encouraging influenza vaccination in children and adults with SCN1A-Dravet syndrome is critical," she said. "Prior to influenza vaccination, vaccine providers should review the child's regular anti-seizure medications and ensure a seizure management plan is in place. The use of additional anti-seizure medications in the post vaccination period, such as benzodiazepines, is now recommended to reduce the risk of seizures following a vaccine and is becoming routine practice." Researchers from The Royal Children's Hospital also contributed to the study.

Laura Gault Late last year, Sage ’s chief medical officer Stephen Kanes left to head a new depression drug developer in Ancora . Now, one year later, Sage has finally found his replacement in Laura Gault . Gault comes to Sage from Alexion , AstraZeneca ’s rare disease arm, where she was head of neurology and ophthalmology and led the Ultomiris program for myasthenia gravis and NMOSD. In April, Ultomiris was approved for adults with myasthenia gravis. In moving to Sage, Gault returns to her roots as a psychiatrist. “Perhaps what’s most interesting to me about the Sage pipeline is that it enables us to actively try to address critical areas of brain health where we haven’t seen true innovation in years. As a psychiatrist who has helped many people suffering from depression, it is very exciting to look at the potential of zuranolone to get people better quickly and keep them better without chronic treatment,” she said in an email to Endpoints News , referring to Sage’s late stage depression prospect. Gault is only five days into her new role, but she has a lot ahead of her as Sage is trying to get the Biogen -partnered zuranolone approved for major depressive disorder. Sage and Biogen are touting their drug as a potential acute treatment for depression episodes instead of for chronic maintenance — an aspect that Gault said excited her about working on zuranolone. In a Phase III study , Sage and Biogen found their drug led to a significant change on a depression rating scale compared to a placebo arm after three days. But going out to 15 days, differences were not statistically significant — sparking durability concerns. To assuage said durability concerns, Sage has put out more data on the drug, looking at the occurrence of repeat treatments. Ahead of FDA filing, Sage and Biogen tout more data in hopes of alleviating durability concerns Sage and Biogen are also testing the drug for postpartum depression. — Lei Lei Wu Dannielle Appelhans → It’s a full-on red alert at a Flagship biotech that had tried to take red blood cells to the next level. On Nov. 15, COO Dannielle Appelhans will take over for Pablo Cagnoni as president and CEO of Rubius , which is chopping more than 80% of its staff and is mulling a sale or merger of what’s left. CFO Pepe Carmona and chief legal officer Maiken Keson-Brookes have also been shown the door in the second huge staff reduction we’ve seen here in the last couple months: Rubius told 75% of the workforce to pack their bags in September after massively underwhelming data at AACR made investors head for the hills. Cagnoni will stay on as chairman, ending Noubar Afeyan ’s brief run. John Carroll offers more perspective on these sweeping changes. Daniel O’Connor → You can’t say that Kate Hermans ’ turn as interim CEO of Ambrx Biopharma was uneventful: On her watch, the San Diego ADC developer trimmed 15% of the workforce and tossed its HER2 drug into the wastebasket now that Enhertu has separated itself from the pack. Daniel O’Connor walks in as the permanent CEO at Ambrx, with a heightened focus on its anti-PSMA ADC ARX517 . Formerly the CEO of OncoSec Medical and Advaxis , O’Connor also founded a SPAC called Larkspur Health Acquisition Corp. and is on the board of directors at Seelos Therapeutics . Feng Tian had been with Ambrx for nearly 18 years — rising to CEO in July 2018 — before stepping down in August . → Charles McWherter has been promoted to president of R&D at CymaBay while juggling the CSO duties he’s held at the biotech since 2013. McWherter came to CymaBay as SVP of R&D 15 years ago from Pfizer , where he was the pharma giant’s VP and head of the cardiovascular therapeutics research areas. The fatty liver disease NASH has foiled CymaBay repeatedly with setback after setback , but McWherter and the team are nothing if not persistent with seladelpar , testing the drug in patients with primary biliary cholangitis. Arturo Molina → It didn’t require a lot of waiting to find out Arturo Molina ’s next gig: After his departure from Sutro Biopharma last week, Molina will be the CMO at Protagonist Therapeutics effective Nov. 7. Molina, the medical chief at Sutro since 2016, is now tasked with directing Protagonist’s Phase III trial of rusfertide in polycythemia vera. Last year, the drug quickly pitched out of a jam when the FDA lifted a full clinical hold that it had delivered just 25 days before. Further down the pipeline, the biotech’s ulcerative colitis drug PN-943 whiffed in a Phase II trial, but CEO Dinesh Patel was undeterred. “The fact is, the primary endpoint was missed,” Patel told Max Gelman in April . “The truth is, we believe this drug works.” Frank Stegmeier → KSQ Therapeutics chief scientist Frank Stegmeier wrote on LinkedIn that he’ll be leaving to take the CSO job at an Alexis Borisy joint named Curie.Bio . As Stegmeier explains it, Curie.Bio is “a founder-focused, seed-stage venture firm with a unique model bringing extensive company building and drug discovery know-how and expertise to founders and co-piloting companies on their journey from seed to a successful Series A,” making sure to include the hashtag #FreeTheFounders in his note. Moncef Slaoui (AP) → Is Moncef Slaoui on the comeback trail? In March 2021, a GSK staffer accused Slaoui of sexual harassment and inappropriate conduct, leading to his resignation at several posts , including his new CSO appointment at Centessa , a biotech that’s been on thin ice after dropping its lead program. But the ex-Operation Warp Speed chief advisor has resurfaced as a member of health testing upstart ixlayer ’s advisory board. With his “leave of absence” from “current professional responsibilities” apparently over, Slaoui is choosing to return in this capacity alongside former Veterans Affairs Secretary David Shulkin , Paul Martino , Craig Lipset and Marcus Osborne . John Maraganore → What’s Peer Review without a John Maraganore sighting? It doesn’t feel complete without one, and the Maraganore Meter is spiking again as he chairs the board of GNS . Pharmas like Roche , Amgen and Servier have called upon GNS and its AI capabilities, and Maraganore replaces Timothy Thompson after becoming a strategic advisor here in early June in one of his countless post- Alnylam appointments. → Dutch T cell engager biotech Lava Therapeutics pours into Peer Review with Fred Powell as CFO. Powell just had the same title at Antares Pharma — now in the hands of Halozyme after a $960 million buyout in April — before hitting the C-suite at Lava, which recently teamed up with Seagen on an EGFR candidate for $50 million upfront and up to $700 million overall with milestones included. Kristen Harrington-Smith → First-year ADC Therapeutics CEO Ameer Mallik has found two key pieces to the leadership puzzle with Kristen Harrington-Smith as chief commercial officer and Peter Graham as chief legal officer. It’s a quick turnaround for Harrington-Smith after she appeared in Peer Review last year as the new CCO of another ADC outfit, ImmunoGen , and she also has Big Pharma connections, closing out a 21-year career at Novartis in 2021 as head of US hematology. The ImmunoGen release indicated that Harrington-Smith left so she could be closer to home; VP of market access Todd Talarico will step in on an interim basis. Like Powell, Graham has left Antares, after spending seven years at the New Jersey biotech as general counsel, chief compliance officer, human resources and secretary. The first day for both execs will be Nov. 17. Alan Fuhrman → Esther van den Boom will no longer be the CFO of Carlsbad, CA-based cancer biotech Tyra Biosciences , setting the stage for Alan Fuhrman to succeed her at the beginning of 2023 . Fuhrman was a board member at Checkmate Pharmaceuticals when he vaulted to interim CEO after Barry Labinger ’s departure; six months later, Checkmate was sold to Regeneron in a $250 million bargain. The ex- Amplyx CFO is on the board of directors at Esperion and SpringWorks Therapeutics . → Thought you had your fill of Actinium appointments last week? Think again: Hot on the heels of hitting the primary endpoint with its lead program Iomab-B in acute myeloid leukemia, the New York radiotherapy biotech has installed Big Pharma vet Caroline Yarbrough as chief commercial officer. Yarbrough comes to Actinium after seven years at Novartis — not staying for very long after getting elevated to portfolio general manager, US oncology — and she’s also held marketing roles at Merck , Bristol Myers Squibb and GSK. Actinium announced a busload of hires in last week’s Peer Review , including chief strategy officer Jenny Hsieh and VP of clinical development Akash Nahar . Jill Howe → In June 2021, we told you about Jill Howe taking the CFO job at DTx Pharma , but it’s time for DTx to start sifting through résumés: Howe will be the finance chief at California-based Lineage Cell Therapeutics starting Nov. 14. Howe spent a year with Amplyx as controller & director of finance, then reunited with Sheila Gujrathi and Faheem Hasnain as VP of finance and treasurer with Gossamer Bio after they all worked together at Receptos . George Nomikos → In late September, Salt Lake City-based Lipocine decided to pivot from its oral testosterone drug to CNS diseases in a move that helps the biotech “manage our resources efficiently,” CEO Mahesh Patel said at the time. To help support this change, Lipocine has selected George Nomikos as CMO and named Spyros Papapetropoulos chairman of the board. Nomikos’ packed CV includes stops at Biogen, Sage, Takeda , Astellas , Amgen and Eli Lilly , and in February 2021, he was promoted to SVP, medical & clinical sciences and head of muscle therapeutics at Scholar Rock . Papapetropoulos, ex- SwanBio CEO and current Vigil Neuro CMO, joined Lipocine’s board in April. Maryll Toufanian → In the process of closing a Long Island manufacturing facility , Amneal Pharmaceuticals has dismissed 13 employees at that site. Meanwhile, Amneal has picked up Reem Malki as chief quality officer and Maryll Toufanian as SVP, regulatory strategy and policy. Malki is no stranger to this role from her days at Alvotech , and she’s also been head of global quality operations with Mylan . And Toufanian is a longtime FDA vet who was previously the director of the Office of Generic Drug Policy at the Center for Drug Evaluation and Research (CDER). Judyanna Yu → To Canada we go: mRNA biotech Providence Therapeutics is about to push its Covid-19 vaccine candidate PTX-COVID19-B into a Phase III study as a booster — and has named Judyanna Yu as its first CFO. Jumping on board from Cerecin , where she was VP of corporate finance, operations and development, Yu has also been CFO and COO at Re-Stem Biotech . Adina Pelusio → Adina Pelusio has signed on as SVP of clinical operations at Pittsburgh oncolytic virus biotech KaliVir . Pelusio knows her way around the space after four years with Turnstone Biologics , where she had been promoted to SVP of clinical operations in March. Roche teamed up with KaliVir to develop oncolytic virus candidates in May, and although exact figures weren’t disclosed for that alliance, Astellas did shell out $56 million upfront for its own deal with KaliVir in December 2020. Abdelaziz Toumi → Twelve-year Merck KGaA vet Abdelaziz Toumi has been named CBO of CDMO KBI Biopharma and Selexis . Toumi joined Catalent in September 2021 as head of strategy execution, biologics and was quickly promoted to VP, head of the mRNA business and new ventures in February. He’s also been Lonza ’s head of biologics sales for the EMEA region and head of commercial development. → Nestlé Health Science enzyme therapy partner Codexis has welcomed Margaret Fitzgerald as chief legal officer and general counsel. Fitzgerald and CEO Stephen Dilly know each other well — Dilly was the chief executive at Aimmune while Fitzgerald was the company’s associate general counsel and privacy officer. After her nine years at Genentech , Fitzgerald also held legal posts at ZS Pharma (VP of corporate law, associate general counsel) and Allakos (general counsel, corporate secretary and chief compliance officer). → Cytek Biosciences is tapping Chris Williams as COO. Williams comes aboard from Thermo Fisher’s pharma services group, where he served as VP of engineering, technology, operations and innovation. Earlier in his career at Thermo, Williams served as VP and general manager for single use technologies and VP and general manager for bioprocess equipment and automation. → New York-based Lexeo Therapeutics is bringing in Eric Adler as CSO. Adler joins the team with plenty of experience under his belt from sitting on the boards of Rocket Pharmaceuticals , Ionis Pharmaceuticals and Sana Biotechnology . Sylvie Peltier → Shanghai-based CARsgen has brought in Sylvie Peltier as SVP of global regulatory affairs. A Teva and Pfizer alum, Peltier comes to the CAR-T biotech after leading US regulatory affairs at Servier from 2018-20 and at MorphoSys since 2020. → VC firm Longitude Capital has promoted CFO Carolyn Helms to the role of managing director. From 2014 to September 2021 Helms served as chief compliance officer of Longitude. Additionally, Longitude has brought on Lisa Chui as VP of people. Chui formerly served as VP, head of people at Longitude’s portfolio company Dascena . → Morten Frederiksen is becoming president at Grey Health & Wellness’ new EMEA office. Frederiksen comes aboard from Ogilvy , where he served as head of Ogilvy Denmark. Lisa Deschamps → As we saw with Compass Pathways ’ results in the NEJM this week , psilocybin-based treatments are gaining traction — and Reset Pharmaceuticals is looking to capitalize on the trend with its own candidate for demoralization syndrome in cancer patients. Reset has now made room for AviadoBio CEO Lisa Deschamps on the board of directors. While she was with Novartis, Deschamps was worldwide business head for neuroscience, then spent two years as CBO of Novartis Gene Therapies . → It was an arduous trek to get there, but Novavax finally got an EUA for its Covid-19 vaccine this summer, and this week the Gaithersburg, MD biotech has appointed Rick Rodgers to the board of directors. Rodgers co-founded and was CFO, secretary and treasurer at Tesaro , which GSK bought for $5.1 billion almost four years ago. Lisa von Moltke → Pruritus biotech Cara Therapeutics has added Seres Therapeutics CMO Lisa von Moltke to the board of directors. A former Sanofi Genzyme exec, von Moltke had led clinical development at Alkermes before moving on to Seres in April 2020. → Former Amneal chairman Paul Bisaro has carved out a path to the board of directors at Salt Lake City’s Myriad Genetics . Bisaro now chairs the board at Mallinckrodt . David Ryan → David Ryan has replaced founding member Ansbert Gadicke on the board of directors at Cullinan Oncology . Ryan started out at Massachusetts General Hospital in 1998, becoming clinical director in 2009 and then chief of the MGH Cancer Center three years later. → Don Nicholson is retiring from the board of directors at Kymera, while Biogen and AstraZeneca alum Victor Sandor takes a seat as the protein degradation shop’s newest board member. Sandor, the ex-CMO of Array BioPharma and former global clinical development chief at Incyte, can also be found on the boards of Prelude Therapeutics , ADC Therapeutics, Istari Oncology and Merus . → Mark Iwicki ’s eye disease biotech Kala Pharmaceuticals has elected Marjan Farid to the board of directors. The UC-Irvine clinical ophthalmology professor is also vice chair of ophthalmic faculty at the university’s Gavin Herbert Eye Institute. Illustration: Kim Ryu for Endpoints News

NEW YORK, Nov. 02, 2022 (GLOBE NEWSWIRE) -- Reset Pharmaceuticals, Inc. (Reset Pharma), a privately held biotechnology company focused on the development and commercialization of novel clinical-stage treatments for patients suffering from mental illnesses related to life-altering diseases, today announces that Lisa Deschamps, MBA has been appointed to the company’s Board of Directors. Ms. Deschamps is a highly experienced pharmaceutical executive who has worked at Board and senior management levels with global responsibilities. Ms. Deschamps is currently Chief Executive Officer and Board member of AviadoBio, a private biotech company developing potentially transformational gene therapies for people living with devastating neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). She was previously Senior Vice President and Chief Business Officer of Novartis Gene Therapies, where she was responsible for strategic planning and worldwide commercialization of drugs in the gene therapy portfolio, including Zolgensma®, the world’s most widely used gene therapy. Before this, she was Worldwide Business Head, Neuroscience at Novartis, where she was responsible for the development and commercialization of the neuroscience portfolio. She has been involved in more than a dozen successful product approvals and launches in many therapeutic areas, including in neuroscience. “We are very happy to welcome Lisa to the Board,” said Philip Taub, Chair of the Board of Reset Pharma. “Lisa’s extensive experience in development and commercialization of novel drugs and her global leadership experience, including as Worldwide Business Head, Neuroscience at Novartis, will be invaluable to the company as we prepare the late-stage Phase 2b clinical development of a novel psilocybin-related therapy for cancer patients with Demoralization Syndrome and other mental health conditions.” Lisa Deschamps commented, “I am looking forward to working closely with the Reset Pharma Board and management team to help develop treatment options for mental illness in people with life-threatening diseases like cancer. I will be able to draw on my global commercialization experience with CNS drugs to support the team as they move their lead program through the regulatory approval process.” Up to 40% of all cancer patients in the U.S., representing almost seven million individuals, may be diagnosed with a mood disorder. An estimated 20% of all patients with life-altering cancer have clinically relevant demoralization that often occurs independent of other mood disorders like depression or anxiety. These patients have symptoms, such as hopelessness, helplessness and discouragement, which are associated with a loss of confidence in one’s ability to cope with significant distress. They are also more likely to have suicidal thoughts, have more difficulties with adherence to cancer therapy, and lack effective treatment options.1 2 3 About Reset Pharmaceuticals, Inc. Reset Pharma is a privately held, US-based biotechnology company developing and commercializing novel clinical-stage treatments for patients suffering from mental illness related to life-altering diseases. The company’s lead program initially focuses on developing a psilocybin-related product to treat Demoralization Syndrome in patients with cancer. These patients have clinically relevant levels of demoralization with a perceived lack of control, hopelessness and helplessness and an increased risk of suicide. While the primary focus is the development of a psilocybin-related product for demoralization, as there are currently no effective treatments for this disorder, psilocybin also shows promise as a treatment for anxiety and depression in cancer patients. Reset Pharma has established a highly experienced biotech development team with a strong track record of successful approvals and launches of neuroscience drugs in the US. The company also has a Scientific Advisory Board of key opinion leaders in psychiatry, oncology and psychedelics, as well as a Strategic Advisory Board. Information about Reset Pharma can be found at www.resetpharma.com. Forward-Looking Statements This press release contains “forward-looking statements” concerning the development and commercialization of Reset Pharmaceuticals’ products, timing of clinical trials, the company’s business development efforts and its expectations regarding its prospects. Forward-looking statements are subject to risks, assumptions and uncertainties that could cause actual future events or results to differ materially from such statements, including the ability to obtain funding to support planned clinical activities. These statements are made as of the date of this press release. Actual results may vary. Reset Pharmaceutical undertakes no obligation to update any forward-looking statements for any reason. For further information please contact: Reset Pharmaceuticals, Inc. Jan-Anders Karlssoninfo@resetpharma.com Media inquiries: MC Services AGRaimund Gabrielreset-pharma@mc-services.eu _________________________ 1 Walker et al, Lancet Psychiatry 2014; 1: 343–502 Watson et al, European Journal of Cancer 2005; 41: 1710–17143 Vehling et al, Cancer 2017;123:3394-401.