On March 9, 2021 Alphamab Oncology (stock code: 9966.HK) reported that U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug application (IND) to initiate an open label, multi-center phase II pivotal clinical study (clinical trial No.: KN046-205) in the United States to evaluate the efficacy, safety and tolerability of KN046 (PD-L1/CTLA-4 bispecific antibody) for the treatment of thymic carcinoma (Press release, Alphamab, MAR 9, 2021, View Source [SID1234576358]).

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Thymic carcinoma is a rare but highly aggressive thymic tumor, and the most aggressive subtype of thymic epithelial tumors, accounting for about 20%. The number of patients with thymic carcinoma is about 4,200 to 6,000 in China per year, and 1,400 to 2,000 in the United States per year. Inoperable or metastatic thymic carcinoma has a very poor prognosis, and there is currently no approved standard treatment for patients who have failed platinum-based chemotherapy. The median overall survival after late-line chemotherapy or targeted therapy is less than 12 months, and there is an urgent need for better options to improve the efficacy.

KN046 is a bispecific antibody targeting PD-L1 and CTLA-4 immune checkpoints. It can more effectively activate T-cells and enhance the T-cells cancer killing ability. In a phase I clinical study conducted in Australia, KN046 showed a 75% disease response rate and 100% disease control rate in patients with thymic epithelial tumors. The research data was presented at the 21st World Conference on Lung Cancer (WCLC 2020). In September 2020, KN046 was granted the orphan drug designation by the FDA for the treatment of thymic epithelial tumors. In January 2021, the Phase II pivotal clinical trial of KN046 for the treatment of thymic carcinoma (clinical trial No.: ENREACH-Thymic) completed enrollment of the first patient in China.

About KN046
KN046 is PD-L1/CTLA-4 bispecific antibody independently developed by Jiangsu Alphamab. Its innovative designs include: a different mechanism CTLA-4 fused with PD-L1 single domain antibody; engineered to target the tumor microenvironment with high PD-L1 expression, and Treg (suppress tumor immunity) clearing function.

There are about 20 clinical trials of KN046 in different stages covering more than 10 types of tumors including NSCLC, TNBC, ESCC, HCC and pancreatic cancer in Australia and China. The results of these clinical trials have shown an advantage in survival for patients. Alphamab has received FDA clearance to enter phase Ⅱ trial of KN046 based on the clinical results in China and Australia. Moreover, KN046 has obtained the U.S. FDA’s orphan drug designation for thymic epithelial tumor in September 2020. Two pivotal clinical trials are currently being conducted.

On March 9, 2021 Novartis reported the Phase III CANOPY-2 study evaluating canakinumab (ACZ885), an inhibitor of interleukin-1beta (IL-1β), in combination with the chemotherapy agent docetaxel, did not meet its primary endpoint of overall survival (OS)1 (Press release, Novartis, MAR 9, 2021, https://www.novartis.com/news/media-releases/novartis-provides-update-phase-iii-study-evaluating-canakinumab-acz885-second-or-third-line-treatment-combination-chemotherapy-non-small-cell-lung-cancer [SID1234576288]). The trial was conducted among 237 adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) whose disease progressed while on or after previous platinum-based chemotherapy and PD-(L)1 inhibitor immunotherapy4. Two Phase III CANOPY trials continue, evaluating canakinumab in first-line and adjuvant settings2,3. Novartis and CANOPY-2 investigators will analyze the study data and are expected to submit its findings for presentation at an upcoming medical meeting.

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"While results from the CANOPY-2 trial are not what we hoped for in patients with advanced or metastatic non-small cell lung cancer who have been treated with other lines of therapy, these data give us valuable insights into IL-1β inhibition," said John Tsai, MD, Head of Global Drug Development and Chief Medical Officer at Novartis. "Ongoing Phase III studies in non-small cell lung cancer continue, evaluating canakinumab in earlier treatment settings. We sincerely thank the patients and clinical investigators involved in the CANOPY-2 study for their partnership."

CANOPY-1, a Phase III study evaluating canakinumab in combination with immunotherapy and chemotherapy, is expected to report final results before the end of the year2. CANOPY-A, another Phase III study, is investigating canakinumab as an adjuvant therapy and has enrolled more than 950 patients to date and is expected to enroll a total of 1,500 patients3.

About canakinumab (ACZ885)
Canakinumab is a human monoclonal antibody that binds with high affinity and selectivity to human interleukin-1beta (IL-1β)6,7 and neutralizes IL-1β activity by blocking its interaction with its receptors8. By neutralizing IL-1β, preliminary evidence suggests that canakinumab inhibits pro-tumor inflammation (PTI) to 1) enhance anti-tumor immune response; 2) reduce tumor cell proliferation, survival and invasiveness; and 3) impair angiogenesis8. Pro-tumor inflammation enables tumor development by driving cancer-causing processes and by suppressing anti-tumor immune responses9,10. Canakinumab is a first-in-class interleukin-1beta (IL-1β) inhibitor of PTI in non-small cell lung cancer10.

About the CANOPY program
Novartis launched the CANOPY study program after observing significantly lower than expected rates of lung cancer mortality among patients in the Phase III cardiovascular CANTOS trial. The CANTOS trial evaluated canakinumab as a secondary prevention measure for cardiovascular events in patients following a heart attack (CRP≥2 mg/L)8,9. Patients in the CANTOS trial were also at high risk for inflammatory cancers, like lung cancer, due to advanced age, smoking history and other clinical risk factors8,9. Based on these findings, Novartis launched three, large-scale, randomized, Phase III clinical trials and a Phase II clinical trial to investigate canakinumab as a potential treatment option in non-small cell lung cancer (NSCLC).

CANOPY-1 (NCT03631199) is a Phase III trial evaluating canakinumab as a first-line treatment for locally advanced or metastatic NSCLC in combination with pembrolizumab and platinum-based doublet chemotherapy2
CANOPY-2 (NCT03626545) is a Phase III trial investigating the role of canakinumab in combination with the chemotherapy agent docetaxel in second- or third-line therapy versus docetaxel alone in NSCLC. Part 1 of the CANOPY-2 trial – a safety run-in study to determine the appropriate dosage, was previously presented at ASCO (Free ASCO Whitepaper) 2019. Part 2 of the trial, reported today, evaluated overall survival (OS)4
CANOPY-A (NCT03447769) is a Phase III trial studying canakinumab in the adjuvant setting, following surgical resection and cisplatin-based chemotherapy. The adjuvant study is designed to determine if treatment with canakinumab can prevent cancer relapse3
CANOPY-N (NCT03968419) is a non-registrational Phase II neoadjuvant trial evaluating canakinumab in combination with pembrolizumab among patients with resectable NSCLC prior to their planned surgery5

Novartis and Lung Cancer
Lung cancer is the most common cancer worldwide, accounting for more than 2 million new cases diagnosed each year11. There are two main types of lung cancer – small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC)12. NSCLC accounts for approximately 85% of lung cancer diagnoses, resulting in nearly 1.7 million new cases each year11,13. Currently, the five-year survival rate for lung cancer is less than 20%14, decreasing further when the disease is diagnosed at later stages15. The majority of people with NSCLC are diagnosed with advanced or Stage III or IV disease16, and treatment options are limited for people with lung cancer who experience cancer growth or progression while on standard of care treatments17-19. More people die of lung cancer every year than any other cancer type11. Novartis is committed to developing best-in-class treatments for lung cancer patients around the world. With a focus on both targeted, personalized medicine and the role of newer, immuno-oncology therapies, the lung cancer drug development program at Novartis is among the most robust in the industry. With research activities informed by long-term relationships with leading lung cancer thought leaders and patient advocates, Novartis is focused on reimagining the treatment of lung cancer.

On March 9, 2021 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company focused on hospital acute care, gastroenterology and rheumatology, reported fourth quarter and full year 2020 financial results (Press release, Cumberland Pharmaceuticals, MAR 9, 2021, View Source [SID1234576307]). Net Revenues for the fourth quarter were $10.3 million, up 10% over the prior year period. For the full year 2020, Net Revenues totaled $37.4 million, a 9% increase over 2019. The company also recorded an additional $3.2 million in revenue during the year associated with divested product rights for two brands it is no longer distributing.

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As of December 31, 2020, the total assets of the Company were $96.5 million, including $24.8 million in cash and investments. Total Liabilities were $49.6 million, and Total Shareholder’s Equity was $47 million. Cumberland also had approximately $44 million in tax net operating loss carryforwards, resulting from the prior exercise of stock options.

2020 Highlights:

Implemented national launch of a Next Generation Caldolor product featuring a ready-to-use formulation in a pre-mixed bag
Commenced several national initiatives during 2020 to help hospitals access Cumberland’s acute care brands during the COVID-19 pandemic
Announced a series of publications featuring important new data associated with the Company’s Caldolor (ibuprofen) Injection, for the treatment of pain and fever and Vibativ (telavancin) Injection, for the treatment of hospital-acquired and ventilator-associated bacterial pneumonia, as well as complicated skin and skin structure infections
Introduced an FDA-approved RediTrex line of injectable methotrexate products, featuring an innovative delivery system for patients with arthritis
"We are grateful that Cumberland was able to successfully manage through the pandemic during 2020, in spite of the significant market headwinds and other challenges we encountered," said A.J. Kazimi, Chief Executive Officer of Cumberland Pharmaceuticals. "Our facilities remained open, our operations continued, and our organization remained intact." He continued, "I would like to acknowledge and thank my colleagues for their continued dedication during such trying times for many. Together, we remain focused on our mission of advancing patient care through the delivery of high-quality medicines."

Caldolor

Early in 2020, Cumberland implemented a national launch of its Next Generation Caldolor product featuring a ready-to-use formulation, offering time and cost savings associated with its administration. Caldolor is a non-steroidal anti-inflammatory drug (NSAID) and may be used as the sole method of treatment for mild-moderate pain or as part of a multimodal treatment for severe pain. Cumberland’s new formulation of Caldolor comes in a pre-mixed bag that is ready for use. It is the first FDA-approved pre-mixed bag of ibuprofen.

Caldolor was also the subject of several clinical studies published during the year. These studies demonstrated that patients given Caldolor experienced less postoperative pain and had decreased opioid use. Due to these studies, Caldolor was recommended for consideration in Enhanced Recovery After Surgery protocols for the management of postoperative pain – including that of traumatic origin.

Vibativ

Cumberland launched several national initiatives during 2020 to help hospitals access the Company’s acute care brands during the healthcare emergency. Cumberland’s Vibativ product was used to help COVID-19 patients who developed secondary-bacterial infections in their lungs. Vibativ is a potent antibiotic, FDA approved to treat hospital and ventilator acquired pneumonia that can result from a variety of infectious agents. It addresses a range of Gram-positive bacterial pathogens, including those that are considered difficult-to-treat and multidrug-resistant.

Additionally, there were a series of clinical study manuscripts published during the year regarding Vibativ. One study suggested that Vibativ is a promising and viable option for patients with bacteremia or endocarditis, including those with MRSA or another S. aureus pathogen. Two other studies confirmed the continued in vitro potency of Vibativ and detailed the positive outcomes that resulted.

RediTrex

The Company implemented a soft launch of its newly FDA-approved RediTrex product line during the fourth quarter. Cumberland is now preparing for a full national launch during the second half of 2021, once product supplies are assured and market conditions return to normal. The Company believes that RediTrex will be a valuable addition to the portfolio and help further diversify and grow its business.

RediTrex is approved for patients with severe, active rheumatoid arthritis and polyarticular juvenile idiopathic arthritis who have difficulty tolerating or responding to orally delivered methotrexate. It is also approved for symptomatic control of severe, recalcitrant, disabling psoriasis in adults who are not adequately responsive to other forms of therapy.

Omeclamox-Pak

Cumberland closely monitored its supply chain during the 2020 pandemic. This included monitoring the facilities that supply the raw materials along with those that manufacture the Company’s products. Overall, the chain remained intact with batches of finished goods shipped to the warehouses that supply the country’s hospital and retail pharmacies. However, there was one exception, as the facility packaging Omeclamox-Pak encountered financial difficulties due to the pandemic and suspended operations. Subsequently, Cumberland learned that this facility is reorganizing, and the Company awaits the resumption of its operations.

Ifetroban Phase II Clinical Programs

Cumberland’s development pipeline includes a series of product candidates in Phase II development. Enrollment in the Company’s studies significantly slowed during the pandemic, due to trial suspensions and the decrease in eligible patient admissions to medical centers across the country. While enrollment of new patients was limited most of the year, the Company ensured that patients already entered into a trial continued to receive their study drug. Cumberland looks forward to an improvement in study enrollment as the pandemic subsides and centers begin to reopen.

The Company has Phase II clinical programs underway evaluating its ifetroban product candidates in patients with cardiomyopathy associated with Duchenne Muscular Dystrophy, Systemic Sclerosis, and Aspirin-Exacerbated Respiratory Disease.

FINANCIAL RESULTS:

Net Revenue: For the three months ended December 31, 2020, net revenues were $10.3 million, up 10% from $9.3 million for the prior year period. The company also recorded an additional $900,000 in revenue during the fourth quarter associated with divested product rights.

Net revenue by product for the three months ended December 31, 2020, included $5.2 million for Kristalose, $2.3 million for Vibativ, $1.7 million for Caldolor, and $0.9 million for the Company’s newest brand, RediTrex.

For the year ended December 31, 2020, net revenues were $37.4 million, an 8.9% increase compared to $34.4 million for the year ended December 31, 2019. Additionally, the company recorded a total of $3.2 million in revenue during 2020 associated with divested product rights.

Net revenue by product for the year ended December 31, 2020, included $15.6 million for Kristalose, $10.9 million for Vibativ, $5.3 million for Caldolor, $1.9 million for Acetadote (including the brand and Company’s Authorized Generic), $1.1 million for Vaprisol, $0.9 million for RediTrex, and $0.3 million for Omeclamox- Pak.

Operating Expenses: Total operating expenses for the three months ended December 31, 2020 were $12.0 million, down from $12.7 million for the prior year period. Total operating expenses for the year ended December 31, 2020 were $43.8 million, similar to $43.7 million for 2019.

Adjusted Earnings: Adjusted Earnings for the three months ended December 31, 2020 were $0.2 million, or $0.01 per share, compared to a loss of $(1.9) million, or $(0.12) per share for the prior year period. Adjusted Earnings for the full year ended December 31, 2020 were a loss of $(0.1) million, or $(0.01) per share, a significant turnaround over the loss of $(3.4) million, or $(0.22) per share in 2019.

This performance measure represents net income attributable to common shareholders with adjustments for the impact of income taxes, depreciation, amortization, share based compensation expenses and expenses that are non-core to the operating performance of the period. The definition and the reconciliation of Adjusted Earnings are provided in this release.

Cash Flow: Cash flow from operations for the year ended December 31, 2020 was $5.4 million, compared to $3.1 million for the prior year.

Balance Sheet: At December 31, 2020, Cumberland had $24.8 million in cash and cash equivalents. Total assets at December 31, 2020 were $96.5 million. Total Liabilities were $49.6 million, including $15.0 million outstanding on the Company’s revolving line of credit and $8.2 million related to contingent liabilities related to the Vibativ acquisition, resulting in Total Shareholder’s Equity of $47 million.

Conference Call and Webcast

A conference call and live Internet webcast will be held on Tuesday, March 9, 2021 at 4:30 p.m. Eastern Time to discuss the Company’s fourth quarter and annual 2020 financial results. To participate in the call, please dial 877-303-1298 (for U.S. callers) or 253-237-1032 (for international callers). A rebroadcast of the teleconference will be available for one week and can be accessed by dialing 855-859-2056 (for U.S. callers) or 404-537-3406 (for international callers). The Conference ID for the rebroadcast is 7005878. The live webcast and rebroadcast can be accessed via Cumberland’s website at View Source

On March 9, 2021 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the fourth quarter and year ended December 31, 2020 and provided a corporate update (Press release, Regulus, MAR 9, 2021, View Source [SID1234576323]).

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"Regulus finished the year on a high note", stated Jay Hagan, CEO of Regulus. "We advanced RGLS4326 into a Phase 1b study in ADPKD patients, achieved an enrollment milestone for RG-012 with Sanofi and its Phase 2 clinical study for the treatment of patients with Alport syndrome, and completed a private financing. With the recent progress, we believe we are well positioned to advance the ADPKD program through Phase 1b while also advancing our next generation ADPKD compound toward the clinic."

Program Updates

RGLS4326 for ADPKD: In February 2021, the Company completed enrollment in the first cohort of a Phase 1b clinical study for RGLS4326 in patients with ADPKD (The "Phase 1b"). The Phase 1b is an adaptive design, open-label, multiple dose study in up to three cohorts of patients with ADPKD. The study is designed to evaluate the safety, pharmacokinetics, and changes in levels of polycystin 1 ("PC1") and polycystin 2 ("PC2") in patients with ADPKD administered RGLS4326 every other week for a total of four doses. The dose level for the first cohort is 1 mg/kg of RGLS4326 and the dose level for the second cohort is 0.3 mg/kg. The third and final cohort will be dosed at a level to be determined based on the results of the first two cohorts. Concurrent with completion of enrollment in the first cohort and based on the review of the available interim safety data, the first patient of the second cohort has commenced dosing of RGLS4326. The Company anticipates availability of results from the first cohort in early Q2 2021.

RG-012 for Alport syndrome: In November of 2020, the Company announced that it has achieved the remaining $5 million milestone associated with interim enrollment under its Collaboration and License Agreement (the "Milestone") with Sanofi for its development of miR-21 programs. The Milestone was triggered upon achievement of an enrollment metric by Sanofi in its Phase 2 clinical study evaluating RG-012 for the treatment of patients with Alport syndrome. Results from a previously completed biopsy study demonstrated kidney tissue concentrations that would be predictive of therapeutic benefit based on animal disease models as well as engagement of the target, miR-21. Over the course of the one year open-label study, promising trends in disease markers were observed and RG-012 was generally well tolerated with no serious adverse events. RG-012 has received orphan designation in both the U.S. and Europe.

Corporate Highlights

Closed $19 Million Private Financing: In December 2020, the Company entered into a definitive securities purchase agreement with certain existing and new institutional investors. The Company received gross proceeds of approximately $19 million from the sale of 24,341,607 shares of the Company’s common stock ("Common Stock") and accompanying warrants to purchase up to an aggregate of 18,256,204 shares of Common Stock at a purchase price of $0.622 per share of Common Stock and $0.125 for each share of Common Stock underlying such warrants. In addition, the Company sold 272,970 shares of non-voting Class A-3 convertible preferred stock, in lieu of shares of Common Stock, at a price of $6.22 per share, and accompanying warrants to purchase an aggregate of 2,047,276 shares of Common Stock at a price of $0.125 for each share of Common Stock underlying these warrants. Each share of non-voting Class A-3 convertible preferred stock is convertible into 10 shares of Common Stock, subject to certain beneficial ownership conversion limitations. Investors in the private placement included existing institutional investors, New Enterprise Associates (NEA) and BVF Partners L.P., and members of the Company’s Board and management. The financing also included participation from several new institutional investors, including RS Investments, Point72 and Asymmetry Capital. The Company expects to use the net proceeds of approximately $18M from the transaction primarily to advance RGLS4326 for the treatment of ADPKD and for general corporate purposes, and the Company expects net proceeds, together with existing cash, will provide cash resources to fund planned activities through Q1 2022.

Expanded Board of Directors: In January 2021, Regulus announced the appointment of Dr. Alice Huang to the Company’s Board of Directors. Dr. Huang brings an extensive scientific background to the Board to help direct the company’s drug discovery and development efforts. Dr. Huang is currently Senior Faculty Associate of Biology and Biological Engineering at the California Institute of Technology having joined Caltech in July 1997. Previous to her tenure at Caltech she was Dean for Science and Professor of Biology at New York University, Professor of Microbiology and Molecular Genetics at Harvard Medical School and Director, Laboratories of Infectious Disease at Boston Children’s Hospital.

Fourth Quarter 2020 Financial Results

Cash Position: As of December 31, 2020, Regulus had $31.1 million in cash and cash equivalents.

Revenue: Revenue was $5.0 million and $10.0 million for the quarter and year ended December 31, 2020, respectively, compared to less than $0.1 million and $6.8 million for the same periods in 2019. Revenue recognized for the quarter and year ended December 31, 2020 was attributable to the achievement of the enrollment milestone and completion of transfer and verification of certain materials to Sanofi under the August 2020 amendment to our collaboration agreement with Sanofi related to RG-012, currently in Phase 2 for Alport syndrome. Revenue recognized for the same periods in 2019 was attributable to the upfront payments received under the 2018 Sanofi amendment related to the transfer of the RG-012 program to Sanofi.

Research and Development (R&D) Expenses: Research and development expenses were $4.0 million and $15.3 million for the quarter and year ended December 31, 2020, respectively, compared to $2.1 million and $12.3 million for the same periods in 2019. The aggregate increase for the quarter ended December 31, 2020, as compared to the quarter ended December 31, 2019, was driven by an increase in external development expenses, primarily attributable to start-up activities and initiation of enrollment in our RGLS4326 Phase 1b study in October 2020. The aggregate increase for the year ended December 31, 2020, as compared to the year ended December 31, 2019, was driven by an increase in external development expenses, attributable to RGLS4326 MAD and Phase 1b study activities, partially offset by a reduction in personnel and internal expenses.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.1 million and $8.8 million for the quarter and year ended December 31, 2020, compared to $2.4 million and $11.3 million for the same periods in 2019. These amounts reflect personnel-related and ongoing general business operating costs. The decreases are primarily attributable to continued cost reduction efforts.

Net Loss: Net loss was $1.3 million, or $0.03 per share (basic and diluted), and $15.7 million, or $0.45 per share (basic and diluted), for the quarter and year ended December 31, 2020, respectively, compared to $4.9 million, or $0.23 per share (basic and diluted), and $18.6 million, or $1.08 per share (basic and diluted), for the same periods in 2019.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of Pkd1 and Pkd2, reduction of cyst growth in human in vitro ADPKD models, and attenuation of cyst proliferation and improvement of kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by FDA until the second set of requirements outlined by the agency have been satisfactorily addressed. The Company will use information from the Phase 1 clinical studies, including the first cohort of the Phase 1b together with information from the recently completed additional nonclinical studies generated in 2020, in its plan to address the second set of requirements outlined in the Partial Clinical Hold letter to support studies of extended duration. Regulus plans to discuss its approach to addressing the remaining Partial Clinical Hold requirements with FDA in mid-2021. RGLS4326 received orphan drug designation from FDA in July 2020.

On March 9, 2021 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that it will present at the Oppenheimer 31st Annual Healthcare Conference at 4:30 p.m. Eastern Time on Tuesday Mar. 16, 2021 (Press release, Neurocrine Biosciences, MAR 9, 2021, View Source [SID1234576340]). Matt Abernethy, Chief Financial Officer, will present at the conference.

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The live presentation will be webcast and may be accessed on the Company’s website under Investors at www.neurocrine.com. A replay of the presentation will be available on the website approximately one hour after the conclusion of the events and will be archived for approximately one month.