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Ionis announces AstraZeneca’s initiation of the Phase 2b clinical study of its antisense medicine targeting PCSK9 to lower LDL-cholesterol | News

CARLSBAD, Calif., Nov. 30, 2020 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced that the biopharmaceutical company AstraZeneca has initiated a Phase 2b clinical trial of ION449 (AZD8233), an investigational antisense medicine designed to reduce blood cholesterol levels in patients with dyslipidemia by targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), an important regulator of low-density lipoprotein cholesterol (LDL-C). PCSK9 is an enzyme that controls the number of LDL receptors on the surface of cells. People with genetic variations that reduce PCSK9 function have lower LDL-C levels in the blood and a lower risk for major cardiovascular events. ION449 is a LIgand Conjugated Antisense (LICA) medicine being developed by AstraZeneca as part of a collaboration between Ionis and AstraZeneca.

The Phase 2b, randomized, double-blind, placebo-controlled trial will enroll approximately 108 participants, aged 18-75, who have LDL-C levels between 70 and 190 mg/dL and are receiving moderate- or high-intensity statin therapy as defined by the American College of Cardiology/American Heart Association (ACC/AHA) guidelines on blood cholesterol management. The primary objective is to assess the effect of different doses of ION449 on LDL-C compared to placebo at Week 12 in patients taking baseline statin therapy.  The study will evaluate three dose levels of ION449 versus placebo, all administered once a month by subcutaneous injection. Safety and tolerability will be evaluated along with a number of secondary endpoints. Learn more about the trial at: https://clinicaltrials.gov/ct2/show/NCT04641299. 

In a Phase 1 study reported at the American Heart Association (AHA) Scientific Sessions on November 13, single subcutaneous doses of ION449 (AZD8233) demonstrated dose-dependent mean reductions in circulating plasma PCSK9 and LDL-C levels of >90 percent and up to 70 percent, respectively, in subjects who had a baseline LDL-C between 100 and 190 mg/dL without concomitant statin therapy. Doses up to 120 mg were evaluated. ION449 was observed to be safe and well-tolerated at all dose levels. 

“Results from the Phase 1 study showed that ION449 potently reduces PCSK9 and LDL cholesterol. ION449 demonstrated best-in-class potential for PCSK9 inhibition and LDL-C reduction, supporting larger clinical trials that are now underway to further evaluate efficacy and safety,” said Sotirios “Sam” Tsimikas, M.D., senior vice president, clinical development and cardiovascular franchise leader at Ionis. “The growing evidence supporting Ionis’ advanced LICA technology in cardiovascular disease holds promise for more effective approaches to lower LDL-C and to address cardiovascular disease, the leading cause of death worldwide.”

Dr. Tsimikas will provide an update on Ionis’ cardiovascular programs during Ionis’ Virtual Investor Day, Dec. 7, 2020, beginning at 12 p.m. EST.

Ionis earned a milestone payment of $20 million from AstraZeneca for the Phase 2b clinical trial initiation of ION449. Ionis and AstraZeneca are collaborating on potential treatments for kidney disease, cardiometabolic disease and cancer.

About Ionis Pharmaceuticals, Inc.

As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our

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Rocket Pharmaceuticals Receives Funding from the California Institute for Regenerative Medicine for Phase 1 Clinical Trial of RP-L401 for Infantile Malignant Osteopetrosis

NEW YORK–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the California Institute for Regenerative Medicine (CIRM) has awarded Rocket a $3.7 million CLIN2 grant award to support the clinical development of its lentiviral vector (LVV)-based gene therapy, RP-L401, for the treatment of Infantile Malignant Osteopetrosis (IMO), a rare, severe monogenic bone resorption disorder characterized by skeletal deformities, neurologic abnormalities and bone marrow failure. The CIRM was founded in 2004 following the passing of Proposition 71 or the California Stem Cell Research and Cures Initiative, which allowed $3 billion in state funding for stem cell research conducted in California. This will be Rocket’s second CIRM grant after receiving one in 2019 for the development of the company’s gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I).

“We’re grateful the CIRM has recognized the promise of RP-L401 for IMO, a devastating pediatric rare disease for which the primary treatment option is allogeneic bone marrow transplant,” said Jonathan Schwartz, M.D. Chief Medical Officer and Senior Vice President of Rocket. “RP-L401 could be a potentially curative treatment for this devastating disorder that affects children at a young age, and we are thankful to have this meaningful support from the CIRM to move our program forward for these families.”

Rocket’s Investigational New Drug Application (IND) for RP-L401 was accepted by the U.S. Food and Drug Administration (FDA) in June of 2020, and the gene therapy received Fast Track designation from the FDA in August 2020. Proceeds from the grant will help fund clinical trial costs, as well as provide manufactured drug product for Phase 1 patients enrolled at the U.S. clinical trial site, University of California, Los Angeles, led by principal investigator Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los Angeles. The non-randomized, open-label Phase 1 clinical trial will enroll two pediatric patients, one month of age or older. The trial is designed to assess safety and tolerability of RP-L401, as well as preliminary efficacy, including potential improvements in bone abnormalities/density, hematologic status and endocrine abnormalities. Further information about the clinical program is available here.

About Infantile Malignant Osteopetrosis

Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption. Mutations in TCIRG1 interfere with the function of osteoclasts, cells which are essential for normal bone remodeling and growth, leading to skeletal malformations, including fractures and cranial deformities which cause neurologic abnormalities including vision and hearing loss. Patients often have endocrine abnormalities and progressive, frequently fatal bone marrow failure. As a result, death is common within the first decade of life. IMO has an estimated incidence of 1 in 200,000. The only treatment option currently available for IMO is an

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Greenwich LifeSciences, Inc. Announces Partnership with Baylor College of Medicine for its Planned Phase III Clinical Trial

Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the “Company”), a clinical-stage biopharmaceutical company focused on the development of GP2, an immunotherapy to prevent breast cancer recurrences in patients who have previously undergone surgery, today announced the formation of a partnership with Baylor College of Medicine in Houston, Texas to act as the lead clinical site for the Company’s upcoming Phase III clinical trial. Professor Mothaffar F. Rimawi has agreed to serve as the Global Principal Investigator of the Phase III clinical trial, and Professor C. Kent Osborne and Professor Rimawi are expected to serve as the first members of the Company’s Clinical Advisory Board for the development of GP2 immunotherapy across all indications and HER2 expressing cancers.

Snehal Patel, CEO of Greenwich LifeSciences, commented, “We are pleased to have entered into this collaboration with such prominent key opinion leaders who are truly committed to evaluating the potential of GP2 immunotherapy. Due to GP2’s safety profile, GP2 immunotherapy may provide clinicians with an option to deescalate treatment of patients by reducing the use of other, more toxic and expensive standard of care treatments. Both Professors Rimawi and Osborne have already introduced us to other breast cancer clinical sites and clinical leaders who have provided input into the design of the upcoming Phase III trial and who have expressed an interest in participating in the Phase III trial as high enrollment sites. In addition, we have also been jointly exploring the addition of both US and European breast cancer cooperative groups to more rapidly expand the clinical team.”

Professor Rimawi added, “We are excited to jointly evaluate the potential of GP2 immunotherapy. We believe that our patients will seek to participate in the upcoming trial as the GP2 Phase IIb clinical trial data suggests that GP2 could be both safe and effective and could be easily administered during standard of care follow-up visits. Our patients are seeking safe preventative treatments that allow them to transition away from the trauma of surgery, trastuzumab-based therapies, other HER2 targeted therapies, chemotherapy, and radiation as they seek to return to normal and healthy lives.”

Professor Osborne commented, “Bringing new alternatives to chemotherapy and improving quality of life for patients undergoing treatment for breast cancer are primary focuses of the Breast Cancer Program. GP2 immunotherapy may represent one such opportunity, and we look forward to collaborating with Greenwich LifeSciences and supporting the planned clinical trial with the resources of both the Dan L Duncan Comprehensive Cancer Center and the Baylor College of Medicine.”

Professor Mothaffar F. Rimawi is board certified in internal medicine, hematology and medical oncology, and serves as both Executive Medical Director and Co-Leader of the Breast Cancer Program at the Dan L Duncan Comprehensive Cancer Center.

Professor C. Kent Osborne is board certified in internal medicine, hematology and medical oncology, and serves as both the Tina and Dudley Sharp Chair in Oncology and the founding Director of the Dan L Duncan Comprehensive Cancer Center. Professor Osborne is also Professor of Medicine and Molecular and

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COVID hospitalizations surge as pandemic enters alarming new phase in U.S.

Americans went to the polls Tuesday under the shadow of a resurging pandemic, with an alarming increase in cases nationwide and the number of people hospitalized with COVID-19 reaching record highs in a growing number of states.

While daily infections were rising in all but three states, the surge was most pronounced in the Midwest and Southwest.

Missouri, Oklahoma, Iowa, Indiana, Nebraska, North Dakota, and New Mexico all reported record high hospitalizations this week. Nebraska’s largest hospitals started limiting elective surgeries and looked to bring in nurses from other states to cope with the surge. Hospital officials in Iowa and Missouri warned bed capacity could soon be overwhelmed.

The resurgence loomed over candidates and voters, fearful of both the virus itself and the economic toll of any new shutdowns to control its spread. The debate over how far to take economically costly measures has divided a country already sharply polarized over President Donald Trump’s turbulent four years in office.

The infection rate is definitely a leading indicator for hospitalizations, and the hospitalization rate is a leading indicator of mortality.

Meanwhile, Iowa hospital officials warned their facilities and staff could be overwhelmed without serious efforts to curtail the virus spread. The state’s seven-day rolling average of positive cases reached 36.4 percent over the weekend, the third-highest in the nation behind South Dakota and Wyoming, according to researchers at Johns Hopkins University. Hospitalizations reached a record 730 on Monday.

Suresh Gunasekaran, CEO of University of Iowa Hospitals and Clinics, said Iowa is entering its third peak, one that is higher than previous ones in May and July. He said his biggest concern is that this peak comes at the beginning of the cold weather season, when the flu and other respiratory conditions typically increase hospitalizations.

“The infection rate is definitely a leading indicator for hospitalizations, and the hospitalization rate is a leading indicator of mortality,” Gunasekaran said.

Related: Pediatric cases now account for 11% of all cases in the U.S., up from 2% in April.

Health officials in Nebraska said hospitalizations have doubled in recent weeks, reaching a record 613 on Sunday.

“No doubt if this trend continues — not just at our hospitals — but every hospital in the state could be at capacity in a very short period of time,” Dr. Cary Ward, chief medical officer for CHI Health’s network of 14 hospitals across eastern Nebraska and western Iowa said during a video call with reporters.

In Missouri, leaders of several rural hospitals raised alarms about bed capacity during a conference call Monday with Republican Gov. Mike Parson, who drew renewed fire from his Democratic election challenger for his refusal to issue a statewide mask mandate. The state health department reported 1,659 hospitalizations statewide, surpassing by 10 the previous record set a day earlier. Among the five additional deaths reported Monday was a 13-year-old boy, the first child under 14 to die from the virus in Missouri.

In Colorado, officials said more residents have been hospitalized with the

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health

Covid hospitalizations surge as virus enters alarming new phase in U.S.

Americans went to the polls Tuesday under the shadow of a resurging pandemic, with an alarming increase in cases nationwide and the number of people hospitalized with COVID-19 reaching record highs in a growing number of states.

While daily infections were rising in all but three states, the surge was most pronounced in the Midwest and Southwest.

Missouri, Oklahoma, Iowa, Indiana, Nebraska, North Dakota, and New Mexico all reported record high hospitalizations this week. Nebraska’s largest hospitals started limiting elective surgeries and looked to bring in nurses from other states to cope with the surge. Hospital officials in Iowa and Missouri warned bed capacity could soon be overwhelmed.

Full coverage of the coronavirus outbreak

The resurgence loomed over candidates and voters, fearful of both the virus itself and the economic toll of any new shutdowns to control its spread. The debate over how far to take economically costly measures has divided a country already sharply polarized over President Donald Trump’s turbulent four years in office.

Meanwhile, Iowa hospital officials warned their facilities and staff could be overwhelmed without serious efforts to curtail the virus spread. The state’s seven-day rolling average of positive cases reached 36.4 percent over the weekend, the third-highest in the nation behind South Dakota and Wyoming, according to researchers at Johns Hopkins University. Hospitalizations reached a record 730 on Monday.

Suresh Gunasekaran, CEO of University of Iowa Hospitals and Clinics, said Iowa is entering its third peak, one that is higher than previous ones in May and July. He said his biggest concern is that this peak comes at the beginning of the cold weather season, when the flu and other respiratory conditions typically increase hospitalizations.

“The infection rate is definitely a leading indicator for hospitalizations, and the hospitalization rate is a leading indicator of mortality,” Gunasekaran said.

Health officials in Nebraska said hospitalizations have doubled in recent weeks, reaching a record 613 on Sunday.

“No doubt if this trend continues — not just at our hospitals — but every hospital in the state could be at capacity in a very short period of time,” Dr. Cary Ward, chief medical officer for CHI Health’s network of 14 hospitals across eastern Nebraska and western Iowa said during a video call with reporters.

In Missouri, leaders of several rural hospitals raised alarms about bed capacity during a conference call Monday with Republican Gov. Mike Parson, who drew renewed fire from his Democratic election challenger for his refusal to issue a statewide mask mandate. The state health department reported 1,659 hospitalizations statewide, surpassing by 10 the previous record set a day earlier. Among the five additional deaths reported Monday was a 13-year-old boy, the first child under 14 to die from the virus in Missouri.

In Colorado, officials said more residents have been hospitalized with the coronavirus than at any time since a peak in April. Flags were flying at half-staff in New Mexico, where death from COVID-19 surpassed 1,000 last week and hospitalizations reached a record 380

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health

Soleno Therapeutics Announces Presentation of Body Composition Results from DESTINY PWS, a Phase III Trial of DCCR in Prader-Willi Syndrome

REDWOOD CITY, Calif., Nov. 03, 2020 (GLOBE NEWSWIRE) — Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced presentation of body composition data from the Company’s Phase III trial, DESTINY PWS (C601), evaluating once-daily Diazoxide Choline Controlled Release (DCCR) tablets for patients with Prader-Willi Syndrome (PWS). The results were presented today by Parisa Salehi, M.D., Clinical Director of the Prader-Willi Syndrome Clinic at Seattle Children’s Hospital, in a late-breaking oral presentation at The Obesity Society’s ObesityWeek® 2020 meeting. Dr. Salehi presented the data on behalf of the DESTINY PWS Investigators.

DESTINY PWS is a randomized, double-blind, placebo-controlled Phase III study of once daily oral administration of DCCR in 127 PWS patients conducted at 29 sites in the U.S. and U.K. The objective of the study was to assess the efficacy and safety of DCCR in subjects with genetically-confirmed PWS aged four years and older and weighing between 20 and 134 kg. Patients who completed the double-blind study enrolled in study C602, an ongoing open-label, extension study. Updated top-line results were previously announced in September 2020 that demonstrated DCCR’s beneficial impact on hyperphagia, the predominant symptom of PWS, other behaviors and body composition abnormalities typical of PWS.

Key results for effects on body composition and adipokines following DCCR treatment were presented at ObesityWeek 2020 as follows:

Fat mass and body mass:

  • Significant reductions in fat mass (p=0.0027) were observed after 13 weeks of double-blind treatment
    –  Greater mean reductions in fat mass were observed in those participants weighing more than 100 kg at baseline (placebo-adjusted difference -4.82 kg)

  • Linear exposure-response relationship for fat mass was significant with greater fat loss occurring at higher circulating drug concentrations (p<0.0001)

  • Trend towards increased lean body mass for DCCR compared to placebo (p=0.058)

  • Significant increase in lean body mass/fat mass ratio (p=0.001)

Leptin:

  • Significant reduction in leptin while it increased in placebo-treated subjects (p<0.0001)

  • Linear exposure-response relationship for leptin was significant with greater reductions in leptin occurring at higher circulating drug concentrations (p<0.0001)

  • Reductions in leptin were greater than would be predicted solely by the loss of body fat, suggesting an improvement in leptin sensitivity

Adiponectin:

  • Adiponectin, a cardioprotective hormone, increased in DCCR-treated subjects, but decreased in placebo-treated participants (p<0.0001)

“We are pleased to present further results from the DESTINY PWS study that demonstrate DCCR’s effect on body composition. In the DCCR group compared to placebo, we have observed a significant reduction in fat mass and leptin. Improvements in leptin sensitivity, as suggested by the results, may have an important impact on regulating hyperphagia,” said Dr. Salehi. “The data presented to date show that DCCR has the potential to manage both behavioral and co-morbid metabolic components of PWS.”

“These data represent additional means by which DCCR may address the significant unmet medical needs and the life-threatening comorbidities associated with PWS,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “We remain focused on advancing DCCR as a potential treatment

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Upadacitinib Effective in Two Phase 3 Trials

Upadacitinib (Rinvoq) appears effective for atopic dermatitis (AD) in adolescents and adults, with no unexpected safety concerns, according to two new duplicate phase 3 trials.

The Measure Up 1 and Measure Up 2 trials, each pitting both 15-mg and 30-mg doses of upadacitinib monotherapy against placebo, demonstrated major improvements in all aspects of AD with the active drug, including skin clearance, itch, and patient-reported quality of life.

The Janus kinase (JAK) inhibitor is currently approved by the US Food and Drug Administration for the treatment of rheumatoid arthritis and is being investigated for several other inflammatory conditions.



Dr Emma Guttman-Yassky

“These are exciting results in terms of quick onset of response and maintenance of response in the patient population with moderate to severe atopic dermatitis, including adolescents,” said lead investigator Emma Guttman-Yassky, MD, of the Icahn School of Medicine at Mount Sinai in New York City.

“I think the dermatological community and patients need an additional drug” for AD, “particularly an oral form,” Guttman-Yassky told Medscape Medical News. “The flexibility of dosing is also particularly important with an oral, because if patients are more severe, we may need the higher dose, and all patients may need a higher dose to get the response in the beginning.”

“Both doses are effective, but the rapidity of response is higher with the higher dose, so there’s a strong rationale for both doses,” she added.

Guttman-Yassky presented her research at the virtual European Academy of Dermatology and Venereology (EADV) Congress 2020.

The pair of randomized, double-blinded, multicenter trials included a total of 1683 participants who ranged in age from 12 to 75 years and who had experienced moderate to severe AD for 3 or more years. Between 12% and 15% of trial participants were adolescents.

In both trials, participants were evenly assigned into groups to receive upadacitinib 15 mg, upadacitinib 30 mg, or placebo once daily for 16 weeks. Coprimary endpoints at week 16 included a 75% or greater reduction in score on the Eczema Area and Severity Index (EASI 75) and the proportion of patients who achieved a validated Investigator’s Global Assessment for AD (vIGA-AD) score of 0 or 1 with two or more grades of reduction from baseline.

In both trials, the percentage of patients who achieved either EASI 75 or vIGA-AD 0/1 at week 16 was markedly higher with both doses of upadacitinib compared to placebo (P < .001).

In the upadacitinib 15-mg groups, EASI 75 was recorded in 69.6% in Measure Up 1 and 60.1% in Measure Up 2. Proportions were higher for patients who received upadacitinib 30 mg, reaching 79.7% in Measure Up 1 and 72.9% in Measure Up 2. In the placebo group, EASI 75 was recorded in only 16.3% and 13.3%, respectively.

In the upadacitinib 15-mg group, vIGA-AD 0/1 was achieved in 48.1% in Measure Up 1 and 38.8% in Measure Up 2. The upadacitinib 30-mg group logged 62% and 52, respectively. For patients who received placebo, rates were 8.4% in Measure Up 1 and

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‘Deadly’ Phase of Pandemic Looms, White House Advisor Birx Warns | Health News

By Ernie Mundell and Robin Foster HealthDay Reporters

(HealthDay)

TUESDAY, Nov. 3, 2020 (HealthDay News) — The pandemic is entering a new and “deadly” phase and the United States needs to adopt a more aggressive approach to curbing the spread of COVID-19, White House coronavirus response coordinator warned Monday.

The warning, expressed by Dr. Deborah Birx in a private memo to White House officials, was a direct contradiction to President Donald Trump’s repeated claims that the pandemic is “rounding the corner,” the Washington Post reported.

Birx’s memo painted a grim picture: “Cases are rapidly rising in nearly 30 percent of all USA counties, the highest number of county hotspots we have seen with this pandemic,” it said. “Half of the United States is in the red or orange zone for cases despite flat or declining testing.”

The memo went further, and suggested that Trump and his advisers were spending too much time focusing on preventing lockdowns and not enough time on controlling the virus.

“We are entering the most concerning and most deadly phase of this pandemic,” Birx wrote. “This is not about lockdowns — it hasn’t been about lockdowns since March or April. It’s about an aggressive balanced approach that is not being implemented.”

Until now, Birx has not criticized Trump or his administration in public, the Post reported. But her sharp critique mirrors a growing dread among government scientists and public health experts that the worst of the pandemic is yet to come.

Birx now contradicts Trump on numerous points, the Post reported:

–While he holds large campaign rallies, she warns against them.

–While he blames rising cases on more testing, Birx says testing is “flat or declining” in many areas where cases are rising.

–While he says the country is “rounding the turn,” Birx notes the country is entering its most dangerous period yet and will see more than 100,000 new cases a day this week.

The latest case counts suggest Birx is right: More than 9.2 million Americans have been diagnosed with COVID-19, and the seven-day average of new daily coronavirus infections in the United States hit a new high of 81,740 in Sunday, the Post reported.

Meanwhile, hospitals are scrambling to find enough nursing help — especially in rural areas and at small hospitals, the Associated Press reported.

Nurses are being trained in fields where they have limited experience, hospitals are scaling back other medical services, and health systems are turning to short-term travel nurses to help fill the gaps, the AP reported.

Adding to the strain, experienced nurses are “burned out with this whole [pandemic]” and some are quitting, said Kevin Fitzpatrick, an emergency room nurse at Hurley Medical Center in Flint, Mich., where several left just in the past month to work in hospice or home care or at outpatient clinics.

States say they don’t have enough money to distribute a COVID vaccine

Meanwhile, state health officials say they are frustrated about a lack of financial support from the federal government as they

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Biogen to Present Positive Phase 2 Systemic Lupus Erythematosus Data at American College of Rheumatology 2020 Meeting

  • In Phase 2 LILAC study, BIIB059 demonstrated a statistically significant reduction in joint disease activity compared to placebo in systemic lupus erythematosus patients

  • Positive results build on previously reported cutaneous lupus erythematosus data and underscore Biogen’s commitment to the lupus community

  • Systemic lupus erythematosus is a chronic and debilitating autoimmune disease that affects multiple organ systems, with periods of illness or flares alternating with periods of remission

CAMBRIDGE, Mass., Nov. 03, 2020 (GLOBE NEWSWIRE) — Today, Biogen Inc. (Nasdaq: BIIB) announced positive data from the 24-week systemic lupus erythematosus (SLE) portion of the Phase 2 LILAC study (part A) demonstrating that BIIB059 (anti-BDCA2) was associated with a statistically significant reduction in total active joint count. The study evaluated the efficacy and safety of BIIB059, a humanized IgG1 monoclonal antibody (mAb) targeting blood dendritic cell antigen 2 (BDCA2) expressed exclusively on plasmacytoid dendritic cells. These data, along with the previously reported findings from the cutaneous lupus erythematosus (CLE) portion of the LILAC study, will be presented at the American College of Rheumatology’s ACR Convergence 2020, being held virtually from November 5-9, 2020.

“People living with systemic lupus erythematosus suffer from chronic and debilitating symptoms that impact multiple organ systems as well as their social and emotional well-being,” said Nathalie Franchimont, M.D., Ph.D., Vice President and Head of the Multiple Sclerosis and Immunology Development Unit at Biogen. “These latest data highlight the potential of BIIB059 to impact disease activity and, together with the earlier cutaneous lupus erythematosus findings, reflect Biogen’s commitment to drive therapeutic innovation for lupus patients who have limited treatment options.”

The Phase 2 LILAC study (part A) met its primary endpoint of reducing joint disease activity in individuals with SLE, as measured by total active joint count. A statistically significant difference in change from baseline of 3.4 in total active joint count was observed at week 24 between participants who received BIIB059 450 mg administered subcutaneously every 4 weeks with an additional dose at week 2 versus placebo (p=0.037). Total active joint count is the total number of tender or swollen joints. Tender or swollen joints are one of the most common symptoms impacting quality of life in people living with SLE.

The study also met the secondary endpoint of SLE Responder Index-4 (SRI-4), resulting in an overall reduction in disease activity in participants who received BIIB059 versus placebo. There was a 26.35 percent higher SRI-4 response rate among participants who received BIIB059 (56.77 percent) versus placebo (30.42 percent [odds ratio=3.49, p=0.003]). The SRI-4 is a composite measure comprising criteria from different internationally validated indices of systemic disease activity.

An additional secondary endpoint from part A of the study in individuals with SLE evaluated the effect of BIIB059 on skin disease activity using the CLE Activity Disease Area and Severity Index-Activity (CLASI-A) score in a subgroup of participants with a baseline CLASI-A score of ≥8. There was a 20 percent higher response rate among participants who received BIIB059 (69.10 percent) versus placebo (49.10 percent) who achieved at

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‘We are entering the most concerning and most deadly phase of this pandemic,’ Birx wrote in a White House memo.

Dr. Deborah L. Birx, who has carefully straddled the line between science and politics as she helps lead the Trump administration’s coronavirus response, delivered a stark private warning on Monday, telling White House officials that the pandemic is entering a new and “deadly phase” that demands a more aggressive approach.

The warning — sent in a private memo to White House officials as the nation — amounted to a direct contradiction of President Trump’s repeated false assertions that the pandemic is “rounding the corner.” In it, Dr. Birx suggested Mr. Trump and his advisers were spending too much time focusing on lockdowns, and not enough on controlling the virus.

“We are entering the most concerning and most deadly phase of this pandemic,” Dr. Birx wrote, adding, “This is not about lockdowns — It hasn’t been about lockdowns since March or April. It’s about an aggressive balanced approach that is not being implemented.”

The report warned against the type of rallies that Mr. Trump has been holding. It also predicted that the United States would continue to see days when the number of new cases exceeded 100,000. Its existence was first reported by The Washington Post; a top White House official who has seen it confirmed its contents.

The blunt message was a striking one for Dr. Birx, who at least in public has resisted disagreeing with Mr. Trump. But with cases soaring around the country, and hospital intensive care units starting to fill up, government scientists and public health experts are growing increasingly concerned that the worst of the pandemic is yet to come.

Dr. Anthony S. Fauci, the government’s top infectious disease expert, has also been offering unusually blunt assessments, and is once again in Mr. Trump’s cross hairs as a result. At a campaign rally in Miami early Monday morning, Mr. Trump attacked the news media for its emphasis on the virus, which prompted the crowd to chant, “Fire Fauci! Fire Fauci!”

To that, the president responded: “Don’t tell anybody, but let me wait until a little bit after the election. I appreciate the advice.” What the president neglected to mention is that Dr. Fauci has Civil Service protections, and it would be extremely difficult for Mr. Trump to remove him.

Unlike Dr. Fauci, who has clashed repeatedly with the White House, Dr. Birx has taken a more measured approach, taking care never to openly criticize the president or his administration.

Dr. Birx was named the coronavirus response coordinator in March. The job has required her to manage the work of the White House coronavirus task force, tracking and orchestrating the government’s effort to contain the outbreak. In the early days of the pandemic, she projected a calm, authoritative presence — and a steady counterpoint to the mixed messages from Mr. Trump.

More recently, though, she has been eclipsed by Dr. Scott W. Atlas, Mr. Trump’s new pandemic adviser, who advocates allowing the virus to spread naturally among young people, while the government focuses its efforts on protecting

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