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

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.


(PRNewsfoto/Ionis Pharmaceuticals, Inc.)

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 drug

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This is ‘most deadly phase’ yet

An internal memo from Dr. Deborah Birx that circulated among top officials in President Trump’s administration pokes holes in the his claim that the country is “rounding the corner” in the fight against the coronavirus and will soon have defeated it.



Donald Trump, Deborah Birx are posing for a picture: Internal memo shows Birx contradicting Trump on pandemic: This is 'most deadly phase' yet


© getty: White House Coronavirus Response Coordinator Dr. Deborah Birx
Internal memo shows Birx contradicting Trump on pandemic: This is ‘most deadly phase’ yet

“We are entering the most concerning and most deadly phase of this pandemic … leading to increasing mortality,” Birx said Monday in a memo reported by The Washington Post. “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.”

Trump has repeatedly attempted to reassure voters that his administration is helping the country “round the corner” and suggested the virus would soon disappear with or without a vaccine for widespread use.

Birx, a leading member of the coronavirus task force led by Vice President Pence, has served as the nation’s top expert on COVID-19 data and appeared almost daily on television and during briefings with reporters during the pandemic’s early days.

A top administration official told The Post that Birx has become increasingly frustrated by what she has characterized as feeling “ignored” by White House officials as she and others have warned against a potentially deadly second and third wave of the virus this fall and winter.

Video: Twitter flags doctored video purportedly showing Biden addressing wrong state (FOX News)

Twitter flags doctored video purportedly showing Biden addressing wrong state

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The Monday memo specifically referenced Trump’s recent campaign rallies, many of which have involved little social distancing and mask wearing by attendees.

Late on Sunday, Trump, who downplayed the dangers of the virus in the spring before contracting it himself last month, encouraged a crowd of supporters who chanted for him to “fire Fauci.”

Dr. Anthony Fauci is another leading member of the task force and a close colleague of Birx’s with whom Trump has publicly broken with.

“Don’t tell anybody, but let me wait until a little bit after the election,” Trump said of Fauci. “He’s a nice man, but he’s been wrong on a lot.”

Brix’s memo reportedly insists on a “much more aggressive action from messaging, to testing, to surging personnel around the country before the crisis point.”

For weeks, several states in the upper Midwest and rural south have reported spikes in coronavirus cases, in many cases a trend public health officials attribute to loosened lockdown regulations and smaller family gatherings ahead of the holiday season.

“This is about empowerment [sic] Americans with the knowledge and data for decision-making to prevent community spread and save lives,” the memo said.

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First-Ever Positive Phase III Trial in Epidermolysis Bullosa

A topical treatment derived from tree bark significantly increased healing of epidermolysis bullosa (EB) lesions versus standard care in an international multicenter clinical trial.

After 45 days of treatment, 41.3% of patients randomized to oleogel-S10 (Filsuvez) had complete wound closure as compared with 28.9% of the control group. A subgroup analysis showed that the beneficial effects were limited to patients with recessive dystrophic, which accounted for almost 80% of the study population.

The wound-healing advantage of oleogel-S10 emerged at about 30 days and persisted out to 90 days, when the proportion of patients with healing became similar in the two treatment groups, reported Dedee Murrell, MD, of the University of New South Wales in Kensington, Australia, during the European Academy of Dermatology and Venereology virtual conference.

“The time to event, which is wound healing over 90 days … was not statistically significant overall,” she said. “The wound healing trajectories demonstrated that oleogel-S10 accelerates wound healing in a subset of the wounds. However, as expected, with good wound care, the control group begins to catch up later by 90 days. The difference in the proportion of healed target wounds had narrowed between treatment groups at 90 days, but the control group never overtook the oleogel arm.”

“This is the first time that a phase III trial in EB has met its primary endpoint,” she added.

Background of Development

A rare genetic skin-fragility disorder, EB characteristically emerges as a pattern of recurring healing and break-down wounds, along with chronic slow-healing or nonhealing wounds. The condition has no approved therapy, and standard of care consists of nonadhesive bandages, topical antimicrobial agents, topical steroids, and various unapproved therapies that are not specific for EB, Murrell noted.

The primary active ingredient in oleogel-S10 is betulin, a naturally occurring triterpene found in the bark of certain types of birch trees. Dry betulin extract is mixed with sunflower oil to form a gel, which is applied directly to EB lesions and to the contact surface of bandages. The mechanistic rationale for its use in EB includes evidence that triterpenes help modulate inflammation and are involved in keratinocyte proliferation, migration, and differentiation.

Preliminary clinical research provided evidence of accelerated wound healing in patients with dystrophic EB. The work subsequently led to the international phase III EASE trial. Investigators at 58 sites in 28 countries enrolled 223 patients, primarily with dystrophic EB but also junctional EB or Kindler syndrome. Eligible patients had a partial thickness wound 10-50 cm2 in size, persisting for 21 days to 9 months.

Patients were randomized to oleogel-S10 or control gel, each in addition to standard dressings changed at least once every 4 days. The primary endpoint was the proportion of patients who had a first complete closure of a target wound within 45 days. Secondary endpoints included time to wound healing, proportion of target wounds healed within 90 days, incidence and severity of wound infections, change in total body wound burden, change in itching, and adverse events.

Patients ages 4-12 years accounted for

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Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Clinical Trials of Setmelanotide in The Lancet Diabetes & Endocrinology

Largest studies in POMC and LEPR deficiency obesities demonstrate that treatment with setmelanotide reduced body weight and hunger

BOSTON, Oct. 30, 2020 (GLOBE NEWSWIRE) — Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, announced today that results from two pivotal Phase 3 studies evaluating setmelanotide in proopiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity were published in The Lancet Diabetes & Endocrinology. As previously reported, data from the studies demonstrate that treatment with setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, led to statistically significant and clinically meaningful reductions of weight and hunger.

“Results from Rhythm’s pivotal Phase 3 studies, which are the largest studies to date in POMC and LEPR deficiency obesities, provide evidence regarding the safety and efficacy of setmelanotide and we believe they validate its potential long-term use as a novel treatment for severe obesity and hyperphagia,” said co-author Peter Kühnen, M.D., Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin Berlin, Germany. “It is important to recognize the signs of these rare genetic disorders because we may soon have a targeted treatment option available for the first time for obesity disorders caused by impairments of the MC4R pathway.”

Rhythm initially reported positive topline data from the Phase 3 studies in August 2019 and subsequently presented updated data in a late-breaking research forum during the 37th Annual Meeting of The Obesity Society at ObesityWeek® 2019.

Eight of 10 participants with POMC deficiency obesity (80%; P<0.0001 compared with historical data) and five of 11 participants with LEPR deficiency obesity (45%; P=0.0001 compared with historical data) achieved at least 10 percent weight loss at approximately one year. The mean percent change in “most hunger” score in participants aged 12 years and older was -27.1 percent (n=7; P=0.0005) in POMC deficiency obesity and -43.7 percent (n=7; P<0.0001) in LEPR deficiency obesity. Consistent with prior clinical experience, setmelanotide was generally well-tolerated in both trials. The most common adverse events were injection site reaction, skin hyperpigmentation, and nausea.

“These results are significant because, as we know from natural history data, individuals living with POMC or LEPR deficiency obesity consistently experience substantial weight gain each year beginning in early childhood, and we would not expect any of these patients to be able to achieve 10 percent weight loss over the course of a year without continued treatment,” said co-author Karine Clément, professor of nutrition at Pitié-Salpêtrière hospital and Sorbonne University in Paris. “These data and the significant unmet need to address the obesity and hyperphagia caused by rare genetic disorders of obesity underscore the importance of testing for genetic variants that may impair MC4R activation and lead to severe obesity.”

In May 2020, Rhythm announced that the U.S. Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity, granted Priority Review of the NDA and assigned a Prescription Drug

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Norwalk Returns To Phase 2 Reopening As Coronavirus Cases Rise

NORWALK, CT — Norwalk is reverting to Phase 2 reopening efforts, as the city grapples with a continued rise in coronavirus (COVID-19) cases, Mayor Harry Rilling announced Thursday. The rollback will take effect at noon on Nov. 1 to allow businesses to prepare.

The city was placed on red alert status last week by Gov. Ned Lamont and state health officials, due to Norwalk exceeding 15 new coronavirus cases per 100,000 people. The number of red alert communities in Connecticut now stands at 30, up from 19 last week, according to the state.

At that time, the city’s rate of infection was 18.9 new coronavirus cases per 100,000, but that number more than doubled to 40.5 and then to 48.9 per 100,000 between Oct. 18 and Oct. 24. Norwalk also reported its first coronavirus-related deaths this week, after more than three weeks without one.

To date, 151 Norwalk residents have died as a result of the virus.

This week, Rilling has been under quarantine due to exposure to a family member who tested positive for COVID-19. The mayor has tested negative twice since then.

As of Thursday, 54 new coronavirus cases were reported in Norwalk, bringing the city’s total to 2,984 since the pandemic began in March, according to health officials. No new deaths were reported.

The city was at Phase 3 reopening, which meant restaurants, personal services, gatherings and sporting events could operate at 75 percent capacity. On Nov. 1, that number rolls back to 50 percent.

Additionally, Phase 2 also calls for:

  • Private gatherings to drop from 100 people to 25 people indoors, and 150 people to 100 people outdoors

  • Religious gatherings to decrease from up to 200 people to a maximum of 100 people indoors

“This is a difficult decision, as I do not want to see our local businesses impacted, but my priority remains the health and safety of our residents,” Rilling said. “Our cases are rising, and I am deeply concerned. We are now seeing increased cases for those over 70 years of age, and we know this population is at higher risk of serious illness and death from this virus.”

The city will host another free, drive-thru coronavirus testing session on Saturday between 10 a.m. and 2 p.m. in the parking lot at Norwalk Community College on Richards Avenue.

“We have ramped up testing to try to slow this virus down, but it continues to spread rapidly, as people are not following all public health guidelines,” Rilling said. “Residents must take this seriously. Please, stay home if you can, limit travel and errands whenever possible, and always wear a face covering in public.”

This article originally appeared on the Norwalk Patch

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Stamford Returning To Phase 2 Reopening As Coronavirus Cases Rise

STAMFORD, CT — Mayor David Martin announced Thursday the city will return to “phase 2” of reopening following an increase in cases of the coronavirus in the city and across the state.

According to the state health department’s COVID-19 data tracker, Stamford is now at 15.6 cases per 100,000, qualifying the city as a “red zone,” Martin said in a news release.

The city also recently received data from the Wastewater Early Detection Program indicating the highest levels of COVID-19 residue in Stamford’s wastewater since the program began in August, Martin said.

See also: High Virus Concentrations Found In Stamford, Bridgeport Sewage

“This is a difficult decision,” Martin said in a statement, “but every indicator we’re monitoring suggests we’re at the beginning of a second wave. Unfortunately, this means we must change our behavior immediately.”

Martin also emphasized the urgency of residents increasing their caution as the city transitions back to phase 2.

(To sign up for Stamford breaking news alerts and more, click here.)

“This second wave is no longer speculative or a possibility, it is happening right now,” Martin said. “There is no feasible way to get our community and economy close to normal if everyone is getting sick. I am reluctant to make this decision because I know how it will impact our businesses and community, but the city of Stamford must rollback to phase 2 as soon as possible.”

Similar to phase 3, residents are required to maintain 6 feet of distance from others, wash or sanitize their hands frequently and wear either a mask or a face covering that covers both their nose and mouth.

The following restrictions are also in place under phase 2:

A full list of restrictions during phase 2, including specific guidelines for various businesses and establishments, can be found on the city website.

According to Jennifer Calder, the city’s director of health, the best defense against this virus is to avoid getting infected and avoid activities that could lead to infection.

“Any interaction with individuals outside your household puts you at risk,” Calder said in a statement. “This is especially true now as we report more cases per day. While many residents are fatigued of health and safety guidelines, unfortunately the virus does not get fatigued and will continue to spread if we let it.”

Residents can monitor daily coronavirus cases by visiting the state health department’s COVID-19 data tracker.

This article originally appeared on the Stamford Patch

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Vosoritide Ups Height in Achondroplasia in Year-Long Phase 3 Study

A year of daily subcutaneous vosoritide was associated with a 0.6-inch (1.5-cm) greater increase in height than placebo in children with achondroplasia, the most common form of human dwarfism.

There was no difference in the incidence of side effects in patients receiving the drug or placebo in this phase 3 study of the investigational drug from BioMarin Pharmaceuticals.

“To our knowledge, this study provides the first, robust evidence for an effective, precision therapy for achondroplasia that could fundamentally change the clinical management policies, growth trajectory, and treatment recommendations for children affected by this condition,” Ravi Savarirayan, MD, University of Melbourne, Australia, and colleagues report.

The study was recently published online in The Lancet.

“Over many years [this therapy] could lead to quite a significant increase in height [and] there were very few side effects that were attributable to the medication,” coauthor Joel Charrow, MD, Ann and Robert H. Lurie Children’s Hospital of Chicago, Illinois, summarized to Medscape Medical News.

And, he noted, it is also hoped “that it will affect other features of achondroplasia and prevent them, such as foramen magnum stenosis and shortening of the nasopharynx.”

“In theory, [infancy] would be the ideal time to start treatment to maximize the benefit,” he continued, and a trial of vosoritide that is enrolling infants with achondroplasia has just begun.

However, therapy for children with achondroplasia to make them taller is controversial, Charrow noted.

“In the dwarf community there are many people who are opposed to this kind of treatment because they don’t think that dwarfism should be medicalized or treated like a disease,” he said.

Asked to comment, David Ornitz, MD, PhD, who was not involved with the study, told Medscape Medical News that vosoritide appeared to be safe during 1 year of use, with no treatment-related adverse effects.

The efficacy results were “encouraging” for this “first potential pharmacological therapy to increase growth of achondroplasia patients.”

The drug could be “a pharmacological alternative to traditional surgical treatments or to not treating this genetic disease,” or it “could be used to augment the effectiveness of surgical therapies,” speculated Ornitz, of the Washington University School of Medicine, St Louis, Missouri.

Caveats include that daily injections might limit patient compliance and drug effectiveness, and it is not known whether efficacy would be sustained over the many years of treatment that would be required or if adverse effects would emerge later on, he noted.

Rare Genetic Mutation, Medical Complications in Addition to Short Stature

Achondroplasia is caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene that results in inhibited mineralization of chondrocytes (cartilage cells) in the growth plate (growing tissue near the ends of the long bones).

The mutation occurs in about one in 25,000 live births worldwide. And more than 80% of children with achondroplasia have parents of average height.

Achondroplasia results in a disproportionately short stature and disordered architecture in the long bones, spine, face, and base of the skull; affected children have an average-sized

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Mustang Bio Announces Initial Phase 1 Data on MB-105 for Patients with PSCA-positive Castration Resistant Prostate Cancer

Data presented by City of Hope at 27th Annual Prostate Cancer Foundation Scientific Retreat

WORCESTER, Mass., Oct. 26, 2020 (GLOBE NEWSWIRE) — Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that one patient‘s experience on the Phase 1 trial of MB-105, a prostate stem cell antigen (PSCA) chimeric antigen receptor (CAR) T administered systemically to patients with PSCA-positive metastatic castration-resistant prostate cancer (mCRPC), was presented at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat.

Tanya Dorff, M.D., City of Hope Associate Clinical Professor, Department of Medical Oncology & Experimental Therapeutics and Head of its Genitourinary Cancer Program and the trial’s principal investigator, presented a description of the correlative science from the ongoing Phase 1 open-label clinical trial of MB-105, one of the first CAR T trials for prostate cancer in the nation. In a 73-year-old male patient with PSCA-positive mCRPC who was treated with MB-105 and lymphodepletion (a standard CAR T pre-conditioning regimen) after failing eight prior therapies, MB-105 demonstrated on day 28 a 94 percent reduction in prostate-specific antigen (PSA), near complete reduction of measurable soft tissue metastasis by computerized tomography, and improvement in bone metastases by magnetic resonance imaging. The therapy was associated with cytokine release syndrome, which was clinically managed with tocilizumab (anti-IL-6 receptor antibody), and hemorrhagic cystitis requiring transfusion which clinically resolved in 30 days.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “We are encouraged by the initial data presented by City of Hope from the ongoing Phase 1 trial of Mustang’s CAR T cell therapy MB-105. We see potential for this PSCA-targeted CAR T in the treatment of prostate cancer, as well as other difficult-to-treat solid tumor cancers. We look forward to the continued progression of this trial and anticipate providing further data in the second half of 2021.”

According to the American Cancer Society (ACS), prostate cancer is the most common cancer in American men, excluding skin cancer. ACS estimates 191,930 new cases of prostate cancer in the U.S. will be diagnosed this year, and roughly one out of every nine men will be diagnosed with prostate cancer during his lifetime. The median survival for men with CRPC is less than two years, according to the American Urological Association.

About MB-105 (PSCA CAR T technology)
MB-105 was developed in the laboratory of Saul Priceman, Ph.D., assistant professor in City of Hope’s Department of Hematology & Hematopoietic Cell Transplantation and associate director of translational sciences in the T Cell Therapeutics Research Laboratory led by Stephen Forman, M.D., leader of City of Hope’s Hematologic Malignancies and Stem Cell Transplantation Institute and the laboratory’s director.

The Phase 1 clinical trial of MB-105 will continue to enroll up to 33 patients. Its primary endpoints are to define safety and optimal dosing of PSCA CAR T cells in treating patients with PSCA-positive mCRPC. Secondary

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Crinetics Pharmaceuticals Announces Phase 2 ACROBAT Edge Study with Paltusotine in Acromegaly Met Primary Endpoint

ACROBAT Edge Results Showed Maintenance of IGF-1 Suppression with Paltusotine After Switching from Depot Somatostatin Receptor Ligand Monotherapy  

Paltusotine was observed to be well tolerated among the 60 participants in the ACROBAT Edge and Evolve Studies

On Track to Initiate Phase 3 Paltusotine Program in 1H 2021

SAN DIEGO, Oct. 26, 2020 (GLOBE NEWSWIRE) —  Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, today announced positive topline results from the company’s Phase 2 ACROBAT Edge and ACROBAT Evolve studies of paltusotine (formerly CRN00808), the company’s lead candidate for the treatment of acromegaly. The Company will hold a conference call at 8:00 a.m. Eastern Time today to discuss these results. In addition, a Key Opinion Leader (KOL) call will be held on November 20th to discuss these results in the context of the current standard of care with clinical experts.

The prespecified primary endpoint in Edge was achieved, showing that once daily oral paltusotine maintained insulin-like growth factor-1  (IGF-1) levels at Week 13 in acromegaly patients who were switched from an injected somatostatin receptor ligand  (SRL) depot of either octreotide or lanreotide monotherapy  [change in IGF-1 = -0.034  (-0.107, 0.107), median (IQR)]. There were 25 patients enrolled in this prespecified primary analysis population (Group 1). During the four-week washout period after the 13-week treatment period, Group 1 patients showed a meaningful  (>20%) and prompt  (within two weeks) rise in IGF-1 levels from baseline, which characterized the magnitude of therapeutic activity of oral paltusotine in acromegaly patients. Edge also enrolled an additional 22 patients into four different exploratory populations  (Groups 2-5). 

Paltusotine was generally well tolerated among the 60 ACROBAT participants (including both Edge and Evolve), which is consistent with prior clinical findings in healthy volunteers. There were no discontinuations due to drug-related adverse events, no safety signals seen in clinical laboratory analyses, no treatment-related serious adverse events (SAEs), and no patients required rescue treatments with standard acromegaly medications during treatment. The most common treatment-emergent adverse events (>10%) included: headache, arthralgia, fatigue, peripheral swelling, paresthesia and hyperhidrosis.

“These results from Edge and Evolve support the potential to effectively switch acromegaly patients from their current depot injections to a once-daily oral treatment while maintaining hormonal control,” stated Alan Krasner M.D., Chief Medical Officer of Crinetics. “The heterogeneous nature of the ACROBAT patient population is representative of the real-world population, where acromegaly patients are prescribed a variety of treatments in an effort to control their IGF-1 levels, often unsuccessfully. The potential for once daily oral paltusotine to offer similar disease control provided by first-line injected depot SRLs gives us confidence to move into a Phase 3 program to further evaluate the efficacy and safety of paltusotine.”

Scott Struthers, Ph.D., founder and Chief Executive Officer of Crinetics, added, “Overall, we believe these findings support our thesis that once daily oral paltusotine has the potential to replace injected peptide depots for acromegaly therapy. We

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Nicox’s NCX 470 Receives Approval by Chinese Authorities for Local Start of Mont Blanc Phase 3 Trial

 

October 26, 2020 – release at 7:30 am
Sophia Antipolis, France

 

Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmology company, today announced that its partner, Ocumension Therapeutics, has received approval from China’s Center for Drug Evaluation of the National Medical Products Administration to carry out the Chinese part of the ongoing Mont Blanc trial, the first Phase 3 clinical trial on NCX 470 for the lowering of intraocular pressure (IOP) in patients with open angle glaucoma or ocular hypertension.

 

NCX 470, Nicox’s lead clinical product candidate, is a novel second generation nitric oxide (NO)-donating bimatoprost analog exclusively licensed to Ocumension Therapeutics for the Chinese, Korean and South East Asian markets.

 

Dr. José Boyer, VP and Interim Head of R&D at Nicox, said: “We are pleased with this second Chinese IND approval in our collaboration with Ocumension.  NCX 470 development remains on track, with first results from the Mont Blanc trial expected in Q4 2021.  Initiation of Chinese sites in this trial will be essential in preparing the way for Denali, the second Phase 3 trial with NCX 470, which will include a larger number of Chinese patients.”

 

The Press Release by Ocumension can be found here:

The NCX 470 Mont Blanc Phase 3 clinical trial is a 3-month trial to evaluate the safety and efficacy of NCX 470 ophthalmic solution, 0.1%, versus the current standard of care, latanoprost ophthalmic solution, 0.005%, for the lowering of IOP in patients with open-angle glaucoma or ocular hypertension.  The Mont Blanc trial is expected to randomize approximately 670 patients, at around 50 clinical sites in the U.S. and at a small number of clinical sites in China.  The Mont Blanc trial was initiated in the U.S. in June 2020 and top-line results are currently expected in Q4 2021. 

Nicox and Ocumension will jointly fund the second NCX 470 Phase 3 glaucoma trial, Denali, which is expected to start by end of 2020 and will also evaluate NCX 470 ophthalmic solution, 0.1%, versus latanoprost ophthalmic solution, 0.005%.  The Denali trial will include clinical sites in both the U.S. and China, with the large majority of the patients to be recruited in the U.S.  The Denali trial was designed to fulfill the regulatory requirements to support New Drug Application (NDA) filings in the U.S. and China.

 

NCX 470 is a novel, potential best-in-class, second generation nitric oxide (NO)-donating bimatoprost analog in development to reduce intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension.  Glaucoma is a group of ocular diseases in which the optic nerve is injured, leading to peripheral and, ultimately, central visual field loss and it can eventually lead to blindness if not treated. It is frequently linked to abnormally high IOP (~90% of patients) due to blockage or malfunction of the eye’s aqueous humor drainage system in the front of the eye.  In 2019, worldwide sales of treatments targeting glaucoma were over $6.0 billion out of a $21.9 billion worldwide market for ophthalmic drugs. 

NCX 470 is designed

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