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CNS Pharmaceuticals Announces Business Highlights and 2019 Fourth Quarter Financial Results

On March 12, 2020 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, reported business highlights and financial results for the three months ended December 31, 2019 and Fiscal Year 2019 (Press release, CNS Pharmaceuticals, MAR 12, 2020, View Source [SID1234555517]).

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Business highlights for the fourth quarter of 2019 and recent weeks include the following:

Selected a leading Polish institution for the Phase 1 pediatric trial with Berubicin in glioblastoma multiforme ("GBM"). In February 2020, CNS announced the selection of Children’s Memorial Health Institute, the largest pediatric hospital in Poland as the single site for this pediatric trial which is expected to commence in the second half of 2020.
Completed final Good Manufacturing Practice ("GMP") reprocessing and purity validation of the existing batch of its lead drug candidate Berubicin. In February 2020, the Company announced the final GMP reprocessing of the existing batch of Berubicin, reporting the GMP material met all specifications and analytical testing is now underway. The Company is continuing large-scale production of Berubicin and intends to utilize this supply to complete its planned Phase 2 clinical trial for patients with GBM.
Licensed a novel DNA-binding technology from The University of Texas MD Anderson Cancer Center to expand the clinical pipeline. In January 2020, CNS entered into a licensing agreement with MD Anderson, granting the Company rights to develop and commercialize WP1244, a new class of DNA-binding agent designed to cross the blood-brain barrier for the potential treatment of primary and metastatic brain cancers. WP1244 has been shown in preclinical studies to be 500-times more potent than the chemotherapeutic agent daunorubicin in inhibiting tumor cell proliferation.
Received positive feedback from the U.S. Food and Drug Administration ("FDA") for Pre-IND (Investigational New Drug) proposal. In its December 2019 positive response to the Company’s Pre-IND request, the FDA indicated that the proposal to use a previously manufactured and currently available supply of a lyophilized drug product (i.e., Berubicin) in the Phase 2 clinical trial appears reasonable. Furthermore, the FDA noted that the requested dosage regimen for the planned Phase 2 trial with Berubicin in GBM, which will be based on the Reata Phase 1 trial, was reasonable.
Closed initial public offering ("IPO") of common stock. The Company closed its IPO of 2,125,000 shares of common stock at an offering price of $4.00 per share on November 13, 2019 and an additional 318,750 shares pursuant to the exercise in full of the underwriters’ over-allotment option sold at the IPO price of $4.00 per share on November 20, 2019. Gross proceeds from the offering, including the exercise of the underwriters’ over-allotment option, were $9.8 million and will be used to fund CNS’ clinical trials and for working capital.
"Since completing our IPO we are very pleased with the progress we have made toward initiating our Phase 2 clinical study of Berubicin to treat GBM, which represents a significant unmet medical need," stated John M. Climaco, Chief Executive Officer of CNS Pharmaceuticals. "We believe Berubicin has the ability to cross the blood-brain barrier with positive responses in these types of tumors, as demonstrated in the Phase 1 study conducted by Reata. In the second half of this year, we look forward to initiating our Phase 2 clinical study in adults in the U.S., as well as two studies conducted in Poland by our sub-license partner, WPD Pharmaceuticals, including a Phase 2 study in adults which will mirror our U.S.-based study, and a first-ever Phase 1 study in children. In addition, we plan to perform further preclinical studies for our recently licensed WP1244 drug candidate, a novel and potent DNA binding agent with high potency to inhibit tumor cell proliferation."

Fourth Quarter Financial Results

General and administrative expense was $1.0 million for the fourth quarter of 2019, compared with $0.2 million for the prior-year period. The increase is largely attributable to the expanded corporate infrastructure implemented in order to advance the Company’s clinical development program following its IPO.

Research and development expense for the fourth quarter of 2019 was $1.5 million, compared with $0 for the fourth quarter of 2018. The expense in the quarter was largely related to the cost of reprocessing and validating the existing batch of Berubicin needed for the commencement of the Phase 2 clinical trial, as well as starting the production of a new batch of the drug necessary for the contemplated clinical trials, both in the U.S. and in Poland.

The net change in cash in the fourth quarter of 2019 was $6.3 million. As of December 31, 2019, CNS had cash and cash equivalents of $7.2 million, which includes $8.8 million in net proceeds from our IPO.

Conference Call

CNS senior management will provide a business update in a conference call and live audio webcast beginning at 4:30 p.m. Eastern time today, March 12, 2020. The conference call dial-in and webcast information is as follows:

DOMESTIC DIAL-IN:

(844) 535-4071

INTERNATIONAL DIAL-IN:

(706) 679-2458

PASSCODE:

1254059

WEBCAST:

CNS Business Update Conference Call.

For those unable to participate in the live conference call or webcast, a replay will be available beginning approximately two hours after the close of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 1254059. The replay can be accessed for a period of time on the CNS website at CNS Business Update Conference Call.

Regulus Reports Fourth Quarter and Year-End 2019 Financial Results and Recent Updates

On March 12, 2020 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, 2019 and provided a corporate update (Press release, Regulus, MAR 12, 2020, View Source [SID1234555516]).

"We have made significant progress over the past few months, including receiving notification from FDA of their decision to lift the partial clinical hold on our Phase 1 multiple ascending dose clinical study for RGLS4326, enabling the initiation of dosing of the second cohort of that study for the treatment of autosomal dominant polycystic kidney disease this past February," said Jay Hagan, CEO of Regulus. "This important milestone, coupled with the closing of the second tranche of financing, enables us to advance the program toward key data read-outs."

Corporate Highlights

Closed $26 Million Second Tranche of Private Financing: In December 2019, following the announcement of the Company’s plan to recommence the Phase 1 Multiple Ascending Dose ("MAD") clinical study of RGLS4326 for the treatment of autosomal dominant polycystic kidney disease ("ADPKD") in the first quarter of 2020, the Company completed a second and final closing under the May 2019 securities purchase agreement, pursuant to which the Company sold and issued 3,288,390 shares of non-voting Class A-2 convertible preferred stock, in lieu of shares of common stock, at a price of $6.66 per share, and accompanying warrants to purchase an aggregate of 32,883,900 shares of common stock at a price of $0.125 for each share of common stock underlying such warrants. Each share of the non-voting Class A-2 convertible preferred stock is convertible into 10 shares of common stock, subject to certain beneficial ownership conversion limitations. The warrants are exercisable for a period of five years following the date of issuance and have an exercise price of $0.666 per share, pursuant to proportional adjustments in the event of stock splits or combinations or similar events. Together with the first tranche, which closed in May 2019, the Company raised a total of $42.7 million from the private financing, which the Company expects will provide cash resources to fund planned activities into mid-2021.

Program Highlights

Initiated Dosing of the Second Cohort in RGLS4326 Phase 1 for ADPKD: In February 2020, the Company initiated dosing of the second cohort of the MAD clinical study of RGLS4326, a novel oligonucleotide designed to inhibit miR-17 for the treatment of ADPKD. The Company expects to complete this study in mid-2020 with topline results available thereafter. The Company is also planning to initiate a Phase 1b short-term dosing study in patients with ADPKD in the second half of 2020 to evaluate RGLS4326 for safety, pharmacokinetics, and biomarkers of pharmacodynamic activity. The re-initiation of the MAD study was allowed following a satisfactory complete response provided to FDA in November 2019 to address the partial clinical hold placed on the clinical program in July 2019. A partial clinical hold on studies of RGLS4326 dosed for extended durations remains in effect until the second set of requirements outlined by FDA have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration.

Financial Results

Cash Position: As of December 31, 2019, Regulus had $34.1 million in cash and cash equivalents.

Research and Development (R&D) Expenses: R&D expenses were $2.1 million and $12.3 million for the quarter and year ended December 31, 2019, respectively, compared to $5.3 million and $34.0 million for the same periods in 2018. The decreases were largely driven by decreases in external development expenses, primarily attributable to the pause of the RGLS4326 MAD study in the third quarter of 2018 and transfer of the RG-012 program to Sanofi beginning in the fourth quarter of 2018. Additionally, the decreases were driven by reductions in personnel and internal expenses, primarily attributable to a reduction in costs subsequent to our corporate restructuring in the third quarter of 2018.

General and Administrative (G&A) Expenses: G&A expenses were $2.4 million and $11.3 million for the quarter and year ended December 31, 2019, respectively, compared to $2.7 million and $12.9 million for the same periods in 2018. The decreases were mostly driven by a reduction in costs subsequent to our corporate restructuring in the third quarter of 2018.

Revenue: Revenue was less than $0.1 million and $6.8 million for the quarter and year ended December 31, 2019, respectively, compared to less than $0.1 million and $0.1 million for the same periods in 2018. The increase for the year ended December 31, 2019 was attributable to revenue recognition of the upfront payments received under the 2018 Sanofi Amendment related to the transfer of the RG-012 program to Sanofi.

Net Loss: Net loss was $4.9 million and $18.6 million for the quarter and year ended December 31, 2019, respectively, compared to a net loss of $8.6 million and $48.7 million for the same periods in 2018. Basic and diluted net loss per share was $0.23 and $1.08 for the quarter and year ended December 31, 2019, respectively, compared to $0.98 and $5.59 for the same periods in 2018.

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 in human ADPKD cyst cells, a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by the U.S. Food and Drug Administration.

China Biologic Reports Financial Results for the Fourth Quarter and Fiscal Year 2019

On March 12, 2020 China Biologic Products Holdings, Inc. (NASDAQ: CBPO, "China Biologic" or the "Company"), a leading fully integrated plasma-based biopharmaceutical company in China, reported its financial results for the fourth quarter and fiscal year of 2019 (Press release, China Biologic Products, MAR 12, 2020, View Source [SID1234555515]).

Fourth Quarter 2019 Financial Highlights

Total sales in the fourth quarter of 2019 decreased by 9.6% in RMB terms and 11.1% in USD terms to $102.2 million from $114.9 million in the same quarter of 2018.
Gross profit decreased by 17.2% to $64.0 million from $77.3 million in the same quarter of 2018. Gross margin decreased to 62.6% from 67.3% in the same quarter of 2018.
Income from operations decreased by 56.2% to $18.6 million from $42.5 million in the same quarter of 2018. Operating margin decreased to 18.2% from 37.0% in the same quarter of 2018.
Non-GAAP adjusted income from operations decreased by 28.9% in RMB terms and 30.2% in USD terms to $27.8 million from $39.8 million in the same quarter of 2018.
Net income attributable to the Company decreased by 64.1% to $12.5 million from $34.8 million in the same quarter of 2018. Diluted earnings per share decreased to $0.32 compared to $0.87 in the same quarter of 2018.
Non-GAAP adjusted net income attributable to the Company decreased by 31.2% in RMB terms and 32.6% in USD terms to $20.7 million from $30.7 million in the same quarter of 2018. Non-GAAP adjusted earnings per diluted share decreased to $0.53 from $0.76 in the same quarter of 2018.
Fiscal Year 2019 Financial Highlights

Total sales in 2019 increased by 12.3% in RMB terms and 7.9% in USD terms to $503.7 million from $466.9 million in 2018.
Gross profit increased by 2.8% to $329.1 million from $320.1 million in 2018. Gross margin decreased to 65.3% from 68.6% in 2018.
Income from operations increased by 11.9% to $163.6 million from $146.2 million in 2018. Operating margin increased to 32.5% from 31.3% in 2018.
Non-GAAP adjusted income from operations increased by 16.6% in RMB terms and 11.6% in USD terms to $198.3 million from $177.7 million in 2018.
Net income attributable to the Company increased by 8.4% to $138.8 million from $128.1 million in 2018. Diluted earnings per share remained stable at $3.53 in 2019 as compared to 2018.
Non-GAAP adjusted net income attributable to the Company increased by 20.5% in RMB terms and 15.4% in USD terms to $168.4 million from $145.9 million in 2018. Non-GAAP adjusted earnings per diluted share increased to $4.28 from $4.02 in 2018.
NOTE: Detailed financial statements and information are available through this link: View Source

"We are pleased to report that China Biologic surpassed our upwardly revised guidance for the full year 2019. As anticipated, however, we saw a year-over-year decline in the fourth quarter after exceptionally strong results for the first nine months of the year," said Joseph Chow, Chairman and CEO of China Biologic. "During the fourth quarter we continued our various initiatives to transform our business, including improving our earnings quality, distribution network, and credit policy management. We saw encouraging progress in several areas including distribution channel consolidation, plasma collection expansion and improving accounts receivable turnover. In 2020, we expect to face challenges including increasingly strict industry regulations, intensified market competition and disruptions to our operations as a result of the coronavirus outbreak. Despite these challenges, we are confident our newly-reorganized management team will successfully implement our strategies as we pursue our mission of raising standards in China’s plasma industry and improving lives through the application of groundbreaking medical science."

Financial Outlook

The COVID-19 outbreak has impacted various aspects of CBPO’s operations, including plasma collection, production of certain products, and sales and marketing activities. The Company is actively evaluating the overall impact on CBPO’s business, and has decided to postpone the release of financial guidance for the full year 2020. The Company will provide financial guidance to the market when it has better visibility.

Conference Call

The Company will host a conference call at 7:30 a.m. Eastern Time on Friday, March 13, 2020, which is 7:30 p.m. Beijing Time on March 13, 2020, to discuss its fourth quarter and fiscal year 2019 results and answer questions from investors. Listeners may access the call by dialing:

US:

1 888 346 8982

International:

1 412 902 4272

Hong Kong:

800 905 945

Mainland China:

400 120 1203

A telephone replay will be available one hour after the conclusion of the conference all through March 20, 2020. The dial-in details are:

US:

1 877 344 7529

International:

1 412 317 0088

Passcode:

10139946

A live and archived webcast of the conference call will be available through the Company’s investor relations website at View Source

Study Shows African American Men with Advanced Prostate Cancer Treated with PROVENGE® (sipuleucel-T) Live Longer than Caucasian Men

On March 12, 2020 Dendreon Pharmaceuticals, a commercial-stage biopharmaceutical company and pioneer in the development of immunotherapy, reported findings from a sub-analysis of data from its PROCEED registry comparing overall survival (OS) in African American (AA) and Caucasian (CAU) men with metastatic castrate-resistant prostate cancer (mCRPC) who were treated with PROVENGE (sipuleucel-T) in a real-world treatment setting (Press release, Dendreon, MAR 12, 2020, View Source [SID1234555513]).

When comparing PSA-matched AA men to CAU men with a baseline PSA less than or equal to the median (29.48 ng/mL), AA men in the PROCEED registry demonstrated a median OS of over 4.5 years (54.3 months) versus over 2.7 years (33.4 months) for CAU men – an improvement of 20.9 months and a 48% relative risk reduction in death.1,2

"Racial differences in the effectiveness of treatments in mCRPC are well documented," said Oliver Sartor, M.D., co-lead author of the publication, associate dean for oncology at Tulane University School of Medicine and medical director of the Tulane Cancer Center. "The high percentage of AA men enrolled in PROCEED compared to other trials provided a unique opportunity to evaluate the effectiveness of immunotherapy in extending survival in this patient population."

The PROCEED registry enrolled nearly 2,000 patients with mCRPC who received PROVENGE between 2011 and 2014 in everyday treatment settings, and followed them for three years. Approximately 12% of patients enrolled in PROCEED were AA. This analysis compared OS in a subset of AA patients (n=107) and CAU patients (n=222) matched by baseline PSA.3

"Black men tend to present with aggressive prostate cancer and have twice the mortality rate as compared to white men," said Andrew J. Armstrong, M.D., co-lead author of the publication, professor of medicine at Duke University School of Medicine and member of the Duke Cancer Institute. "However, in the context of immunotherapy for men with mCRPC, we observe that black men have significantly improved overall survival as compared to similar white men. While we do not yet understand the reasons for this improved survival by self-identified race, these data provide evidence to support the early use of sipuleucel-T immunotherapy regardless of race in order to improve long-term survival."

PROCEED evaluated the real-world use of PROVENGE in men with asymptomatic or minimally symptomatic mCRPC. These data were published online in Prostate Cancer and Prostatic Diseases (PCAN), a peer-reviewed Nature Research journal. The publication in PCAN is the first time these sub-group data have been published in a peer-reviewed journal.

"These findings add to the growing body of published clinical evidence that PROVENGE extends life in men with mCRPC and underscore its added effectiveness in African American men," said Bruce A. Brown, M.D., chief medical officer at Dendreon. "No other prostate cancer treatment has shown this level of added benefit in African American men with mCRPC, so these findings are exciting."

About the PROCEED Registry

PROCEED (NCT01306890) was a multicenter, open-label, observational registry conducted at urology and medical oncology clinics in private practice and academic sites. PROCEED enrolled 1,976 patients with mCRPC, of whom 1,902 received PROVENGE between 2011 and 2014 in everyday treatment settings. Of these, approximately 12% were African American. In the entire PROCEED population, patients were followed for a median of 46.6 months. Their median age was 72 years and their median baseline PSA was 15.0 ng/mL. The sub-analysis published in PCAN compared OS in a subset of African American patients (n=219) and Caucasian patients (n=438) matched by baseline PSA.

About Prostate Cancer in African American Men

Prostate cancer is the most frequently occurring non-cutaneous cancer among men in the United States and is second only to lung cancer among the leading causes of cancer-related deaths. In 2020, an estimated 191,930 new cases of prostate cancer will be diagnosed in the United States and 33,330 men will die from the disease.4

African American men have the highest prostate cancer incidence rate of any racial or ethnic group in the world.5 In the United States, the risk of prostate cancer is 74% higher in black men than non-Hispanic white men.6 The incidence of prostate cancer is about 60% higher in blacks than in whites for reasons that remain unclear.4 Prostate cancer death rates in blacks are more than double those of every other racial and ethnic group in the United States.4

About PROVENGE (sipuleucel-T)

PROVENGE is the only FDA-approved immunotherapy made from a patient’s own immune cells for the treatment of prostate cancer. More than 30,000 men have been prescribed PROVENGE, and it has been clinically proven to extend life for certain men in advanced stages of the disease.

INDICATION

PROVENGE is an autologous cellular immunotherapy indicated for the treatment of asymptomatic or minimally symptomatic metastatic castrate-resistant (hormone-refractory) prostate cancer.

IMPORTANT SAFETY INFORMATION

Acute Infusion Reactions: Acute infusion reactions (reported within 1 day of infusion) may occur and include nausea, vomiting, fatigue, fever, rigor or chills, respiratory events (dyspnea, hypoxia, and bronchospasm), syncope, hypotension, hypertension, and tachycardia.

Thromboembolic Events: Thromboembolic events, including deep venous thrombosis and pulmonary embolism, can occur following infusion of PROVENGE. The clinical significance and causal relationship are uncertain. Most patients had multiple risk factors for these events. PROVENGE should be used with caution in patients with risk factors for thromboembolic events.

Vascular Disorders: Cerebrovascular events (hemorrhagic/ischemic strokes and transient ischemic attacks) and cardiovascular disorders (myocardial infarctions) have been reported following infusion of PROVENGE. The clinical significance and causal relationship are uncertain. Most patients had multiple risk factors for these events.

Handling Precautions: PROVENGE is not tested for transmissible infectious diseases.

Concomitant Chemotherapy or Immunosuppressive Therapy: Chemotherapy or immunosuppressive agents (such as systemic corticosteroids) given concurrently with the leukapheresis procedure or PROVENGE has not been studied. Concurrent use of immune-suppressive agents may alter the efficacy and/or safety of PROVENGE.

Adverse Reactions: The most common adverse reactions reported in clinical trials (≥ 15% of patients receiving PROVENGE) were chills, fatigue, fever, back pain, nausea, joint ache, and headache.

McKesson Announces Final Results of Exchange Offer

On March 12, 2020 McKesson Corporation (NYSE:MCK) reported the final results of its offer to its stockholders to exchange their issued and outstanding shares of McKesson common stock for shares of PF2 SpinCo, Inc. ("SpinCo") common stock owned by McKesson as part of its split-off of its interest in Change Healthcare LLC ("Change Healthcare") and its agreement with Change Healthcare Inc. (NASDAQ:CHNG) ("Change") to merge SpinCo with and into Change (Press release, McKesson, MAR 12, 2020, View Source [SID1234555512]).

The exchange offer expired at 11:59 p.m., New York City time, on March 9, 2020. Under the terms of the offer, 11.4086 shares of SpinCo common stock were exchanged for each share of McKesson common stock accepted in the offer. McKesson accepted 15,426,537 of the tendered shares in exchange for 175,995,192 shares of SpinCo common stock, which were immediately converted into an equal number of whole shares of Change common stock (with cash in lieu of fractional shares) upon completion of the merger, which closed on March 10, 2020. The exchange offer and merger are generally expected to be tax-free to participating McKesson stockholders for U.S. federal income tax purposes except to the extent of any cash received in lieu of fractional shares of Change common stock.

Because the exchange offer was oversubscribed, McKesson accepted tendered shares of McKesson common stock on a pro rata basis in proportion to the total number of shares validly tendered and accepted for exchange. Stockholders who owned fewer than 100 shares of McKesson common stock, or an "odd lot" of such shares, and who validly tendered all of their shares, were not subject to proration in accordance with the terms of the exchange offer. The final proration factor of approximately 14.82% was applied to all other shares of McKesson common stock that were validly tendered and not validly withdrawn to determine the number of such shares that were accepted from each tendering stockholder.

Based on the final count by the exchange agent Equiniti Trust Company, the final results of the exchange offer are as follows:

Total number of shares of McKesson common stock validly tendered and not validly withdrawn:

97,324,143

Shares tendered and not validly withdrawn that were subject to proration:

96,151,322

"Odd-lot" shares tendered that were not subject to proration:

1,172,821

Total number of shares of McKesson common stock accepted:

15,426,537