On March 6, 2020 Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, reported financial results for the fourth quarter and year ended December 31, 2019 and provided a clinical and corporate update (Press release, Milestone Pharmaceuticals, MAR 6, 2020, View Source [SID1234555286]).

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"Our team is keenly focused on delivering the near-term topline readout of the pivotal Phase 3 efficacy and safety trial, NODE-301," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "Results from NODE-301, if favorable, have the potential to serve as the sole efficacy registration trial required for an NDA submission with the United States (U.S.) Food and Drug Administration (FDA), bringing us one step closer to providing paroxysmal supraventricular tachycardia (PSVT) patients with a much-needed novel therapeutic option. Supported by compelling results from the Phase 2 NODE-1 trial, we believe etripamil has the potential to alter the PSVT treatment paradigm as the first self-administered therapy for the rapid termination of supraventricular tachycardia (SVT) episodes wherever and whenever they occur."

Mr. Oliveto added, "As we focus on topline results from NODE-301, we remain diligent in executing on the balance of our Phase 3 program of etripamil for PSVT, including the NODE-302 and NODE-303 safety studies, preparing for the potential commercialization of etripamil, and building out our pipeline beyond PSVT. This includes the expected initiation of our first clinical trial of etripamil in patients with atrial fibrillation and rapid ventricular rate."

Recent Updates

Topline Data from NODE-301 Trial Expected This Month. Milestone expects to report topline data for the NODE-301 trial this month. The NODE-301 trial is a Phase 3, multicenter, randomized, double-blind, placebo-controlled trial of etripamil, the Company’s novel short-acting calcium channel blocker, designed to terminate SVT episodes in the at-home setting. The primary endpoint of the NODE-301 trial is time to conversion of PSVT to sinus rhythm after the administration of trial drug, as confirmed by a central independent adjudication committee. A statistically significant result for NODE-301 would support the trial’s ability to fulfill the U.S. FDA’s previously guided efficacy review requirement for etripamil.

Milestone’s pivotal Phase 3 program of etripamil in PSVT, which was designed in consultation with U.S. and European Union regulatory authorities, consists of three distinct trials: NODE-301, the sole efficacy trial; NODE-302, the ongoing open-label safety extension trial; and NODE-303, the ongoing global safety trial and the largest trial ever conducted in PSVT. Additional blinded data will be collected from randomized patients who have not yet experienced an event at the time the NODE-301 trial reaches its target number of adjudicated SVT events. These data will be analyzed separately as a secondary data set, referred to as NODE-301B, and may contribute further to sub-population analyses and pharmacoeconomic assessments of the NODE-301 trial.

Enrolled First Patient in NODE-303 Trial. In October 2019, Milestone announced enrollment of the first patient in the Company’s Phase 3 open-label, global safety trial of etripamil in patients with PSVT. The trial will primarily evaluate the safety of etripamil when self-administered without medical supervision during single or multiple SVT episodes. Important secondary measures include efficacy, patient quality of life and pharmacoeconomic assessments. The trial will enroll up to 3,000 patients in order to collect data on approximately 1,000 patients who did not participate in NODE-301 or its open-label safety extension trial, NODE-302.

Jeff Nelson Promoted to Chief Operating Officer. Milestone reported the recent promotion of Jeff Nelson to Chief Operating Officer. Mr. Nelson, who joined the Company in 2018 as Vice President of Program Management, brings to this new role over 15 years of experience in the pharmaceutical and biotech field, working primarily in project management, clinical operations, regulatory affairs, drug supply and distribution and public finance.

Richard C. Pasternak, M.D. Appointed to Board of Directors. In November 2019, Milestone announced the appointment of Richard C. Pasternak, M.D. to its Board of Directors. Dr. Pasternak brings to Milestone over 40 years of clinical, academic, and biopharmaceutical industry experience in the area of cardiology.
Fourth Quarter 2019 Financial Results

As of December 31, 2019, Milestone had cash, cash equivalents, and short-term investments of $119.8 million compared to $86.0 million as of December 31, 2018, and 24.5 million shares outstanding.
Research and development expense for the fourth quarter of 2019 was $14.1 million compared with $7.3 million for the prior year period. For the full year ended December 31, 2019, research and development expense was $42.0 million compared with $16.8 million for the prior year.
General and administrative expense for the fourth quarter of 2019 was $2.3 million compared with $1.2 million for the prior year period. For the full year ended December 31, 2019, general and administrative expense was $7.0 million compared with $3.1 million for the prior year.
Commercial expense for the fourth quarter of 2019 was $2.5 million compared with $1.6 million for the prior year period. For the full year ended December 31, 2019, commercial expense was $8.9 million compared with $3.9 million for the prior year.
For the fourth quarter of 2019, operating loss was $18.9 million compared to $10.1 million for the prior year period. For the full year ended December 31, 2019, Milestone’s operating loss was $57.9 million compared to $23.8 million for the prior year.
About Paroxysmal Supraventricular Tachycardia

Paroxysmal supraventricular tachycardia (PSVT) is a rapid heart rate condition characterized by intermittent episodes of supraventricular tachycardia (SVT) that start and stop suddenly and without warning. Episodes of SVT are often associated with symptoms including palpitations, sweating, chest pressure or pain, shortness of breath, sudden onset of fatigue, lightheadedness or dizziness, fainting, and anxiety. Certain calcium channel blockers have long been approved for the treatment of PSVT as well as other cardiac conditions; however, when calcium channel blockers are used for the termination of SVT episodes, they must be administered intravenously under medical supervision, usually in an emergency department or other acute care setting.

About Etripamil

Etripamil, the Company’s lead investigational product, is designed to be a rapid response therapy for episodic cardiovascular conditions. The novel calcium channel blocker is self-administered via a nasal spray which may shift the current treatment paradigm for many patients with PSVT from the emergency department to the at-home setting. Milestone is conducting a comprehensive development program for etripamil, with Phase 3 trials underway in PSVT, and plans to commence a Phase 2 proof-of-concept trial in atrial fibrillation patients with rapid ventricular rate, with subsequent studies expected in other conditions where calcium channel blockers are utilized.

On March 6, 2020 Ardelyx, Inc. (Nasdaq: ARDX), a specialized biopharmaceutical company focused on developing first-in-class medicines to improve treatment choices for people with cardiorenal diseases, reported business highlights and financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Ardelyx, MAR 6, 2020, View Source [SID1234555285]).

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"2019 was a year of significant progress at Ardelyx. We successfully hit all of our key milestones bringing us closer to submitting a New Drug Application to the FDA for tenapanor for the control of serum phosphorus in adult patients with CKD on dialysis in mid-2020 and potentially providing this first in class agent to patients in need," said Mike Raab, president and chief executive officer of Ardelyx. "We enter 2020 well-positioned with data from three successful Phase 3 trials for tenapanor in hyperphosphatemia, key ex-U.S. partnerships and two years of cash on hand to prepare for U.S. commercialization of our novel therapy."

Key Accomplishments in 2019

Published positive Phase 3 results of tenapanor for the treatment of hyperphosphatemia in the Journal of the American Society of Nephrology.
Appointed renowned nephrologist, Geoffery A. Block, M.D., to the company’s board of directors.
Began the process of building a highly talented and experienced cardiorenal commercial team.
Announced positive, statistically significant results from the Phase 3 AMPLIFY study evaluating tenapanor in dialysis patients who have uncontrolled hyperphosphatemia despite phosphate binder treatment.
Received FDA approval for IBSRELA (tenapanor). The company continues to seek a strategic partner to market IBSRELA in the United States.
Expanded collaborative partnership with Kyowa Kirin Co., Ltd (KKC) with a new research agreement and a $20.0 million equity investment in Ardelyx under a Stock Purchase Agreement.
Announced positive topline results from the PHREEDOM study evaluating tenapanor as a monotherapy for the control of serum phosphorus in patients with chronic kidney disease (CKD) on dialysis. The PHREEDOM study met its primary endpoint demonstrating a statistically significant difference in least square (LS) mean serum phosphorus change (-1.4 mg/dL, p<0.0001), as compared to placebo.
Raised approximately $135 million, net of underwriting discounts and commissions, following a successful underwritten public offering of 23,000,000 shares of common stock to support commercial launch preparation for tenapanor for the control of serum phosphorus in patients with CKD on dialysis. The capital raised in the fourth quarter of 2019 extends the company’s cash runway into early 2022, based on its current operating plan.
Initiated the Phase 4 NORMALIZE study and announced initial results demonstrating that a significant number of patients achieved normal serum phosphorus levels with tenapanor alone or with tenapanor and only one to three sevelamer tablets a day.
On-Track to Submit NDA for Tenapanor for the Control of Serum Phosphorus in mid-2020: Ardelyx is on-track to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for tenapanor for the control of serum phosphorus in mid-2020.

Full Year 2019 Financial Results

Cash Position: As of December 31, 2019, Ardelyx had total capital resources including cash, cash equivalents and short-term investments of $247.5 million compared to total capital resources including cash, cash equivalents and short-term investments of $168.1 million as of December 31, 2018.
Revenue and Cost of Revenue: Total revenues were $5.3 million for the year ended December 31, 2019 related to the company’s ex-U.S. collaboration partnerships, and cost of revenues was $0.6 million related to payments due to AstraZeneca in accordance with the company’s termination agreement entered into with AstraZeneca in June 2015 compared to total revenues of $2.6 million and cost of revenues of $0.5 million for the year ended December 31, 2018.
R&D Expenses: Research and development expenses were $71.7 million for the year ended December 31, 2019, an increase of $2.3 million, or 3%, compared to $69.4 million for the year ended December 31, 2018. The increase consisted of a $3.7 million increase in our internal program costs and a $1.4 million decrease in our external program costs. The increase in our internal costs of $3.7 million was primarily due to an increase in headcount and related personnel costs and an increase in stock-based compensation expenses. The decrease in our external program costs of $1.4 million included a $4.6 million decrease in expenses primarily related to manufacturing of tenapanor and regulatory expenses related to our IBS-C NDA in 2018, partially offset by $2.5 million increase in clinical development expenses related to our RDX013 program and a $0.7 million increase primarily related to our tenapanor clinical trial expenses that includes an out-of-period adjustment recorded during the second quarter of 2019 that reduced clinical trial expenses by $3.6 million related to our tenapanor clinical trials.
G&A Expenses: General and administrative expenses were $24.3 million for the year ended December 31, 2019, an increase of $0.6 million, or 2%, compared to $23.7 million for the year ended December 31, 2018.
Net Loss: Net loss for the year ended December 31, 2019, was $94.9 million compared to a net loss of $91.3 million for the year ended December 31, 2018.
Financial Guidance

Ardelyx maintains its expectation that its cash, cash equivalents and short-term investments will be sufficient to fund the company’s operations until early 2022 based on its current operating plans.

On March 6, 2020 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX) reported the rescheduling of its fourth quarter and annual 2019 financial results and company update from Wednesday, March 11, 2020 to Wednesday, March 18, 2020 after market close (Press release, Cumberland Pharmaceuticals, MAR 6, 2020, View Source [SID1234555284]). A conference call and live Internet webcast will be held on Wednesday, March 18, at 4:30 p.m. Eastern Time to discuss the results.

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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 9759981. The live webcast and rebroadcast can be accessed via Cumberland’s website at View Source

Cumberland Pharmaceuticals Inc. is a specialty pharmaceutical company focused on the delivery of high quality prescription brands to improve patient care. The Company develops, acquires and commercializes brands for the hospital acute care, gastroenterology and oncology market segments. These medical specialties are categorized by moderately concentrated prescriber bases that we believe can be penetrated effectively by targeted sales forces. The Company’s portfolio of FDA approved brands includes:

Acetadote (acetylcysteine) Injection, for the treatment of acetaminophen poisoning;
Caldolor (ibuprofen) Injection, for the treatment of pain and fever;
Kristalose (lactulose) for Oral Solution, a prescription laxative, for the treatment of chronic and acute constipation;
Omeclamox-Pak, (omeprazole, clarithromycin, amoxicillin) for the treatment of Helicobacter pylori (H. pylori) infection and related duodenal ulcer disease;
Vaprisol (conivaptan) Injection, to raise serum sodium levels in hospitalized patients with euvolemic and hypervolemic hyponatremia;
Vibativ (telavancin) Injection, for the treatment of certain serious bacterial infections including hospital-acquired and ventilator-associated bacterial pneumonia, as well as complicated skin and skin structure infections.
RediTrex (methotrexate) Injection, for the treatment of active rheumatoid, juvenile idiopathic and severe psoriatic arthritis, as well as disabling psoriasis.
For more information on Cumberland’s approved products, including full prescribing information, please visit the individual product websites, links to which can be found on the Company’s website www.cumberlandpharma.com.

The Company has Phase II clinical programs underway evaluating its ifetroban product candidates in patients with cardiomyopathy associated with Duchenne Muscular Dystrophy ("DMD"), Systemic Sclerosis ("SSc"), and Aspirin-Exacerbated Respiratory Disease ("AERD"), Hepatorenal Syndrome ("HRS") and Portal Hypertension ("PH").

On March 6, 2020 CTI BioPharma Corp. (Nasdaq: CTIC) reported the closing of its previously announced rights offering (the "Rights Offering") (Press release, CTI BioPharma, MAR 6, 2020, View Source [SID1234555283]). At the closing, CTI BioPharma sold and issued an aggregate of 15,698,995 shares of its common stock (the "Common Stock") and an aggregate of 4,429.2423 shares of its series X convertible preferred stock (the "Series X Preferred") pursuant to the exercise of subscription rights and Oversubscription Rights in the Rights Offering by existing holders of CTI Biopharma’s Common Stock and series O convertible preferred stock. The Rights Offering was fully backstopped by certain existing stockholders of CTI BioPharma who agreed to purchase any shares of Common Stock and/or Series X Preferred offered in the Rights Offering that were not subscribed for (the "Oversubscription Rights"). CTI BioPharma raised aggregate gross proceeds of approximately $60.0 million in the Rights Offering.

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Of the total shares of Common Stock and Series X Preferred sold and issued in the Rights Offering, certain affiliates of BVF Partners L.P. purchased 3,047 shares of Series X Preferred, Stonepine Capital, LP purchased 3,267,127 shares of Common Stock and 673.2873 shares of Series X Preferred, OrbiMed Private Investments VI, LP purchased 4,520,600 shares of Common Stock and 298 shares of Series X Preferred and New Enterprise Associates, Inc. purchased 3,390,450 shares of Common Stock and 410.955 shares of Series X Preferred, in each case, pursuant to the exercise of their subscription rights and Oversubscription Rights.

The Rights Offering was made pursuant to CTI BioPharma’s effective shelf registration statement on file with the Securities and Exchange Commission (the "SEC") and a prospectus supplement and accompanying prospectus filed with the SEC on February 14, 2020.

JMP Securities served as financial advisor to CTI BioPharma in connection with the Rights Offering.

On March 6, 2020 AmerisourceBergen reported that it is continuing its strategic relationship with the American Oncology Network, LLC (AON), a high-growth medical oncology provider with a focus on supporting the long-term viability of oncology treatment in community-based settings (Press release, AmerisourceBergen, MAR 6, 2020, View Source [SID1234555282]). AmerisourceBergen will support AON in its effort to accelerate key strategic priorities to scale its integrated value-based care delivery model nationwide.

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AON currently serves an expanding network of partner practices across 11 states, providing dedicated end-to-end administrative support, access to an extensive array of centralized ancillary services, and proven practice management expertise. Delivered through its local market, physician-led model, AON empowers community-based practices with the necessary tools, support and capital to effectively navigate today’s increasingly dynamic healthcare landscape and practice value-based care within the community at scale.

"Our partnership with AmerisourceBergen is an important part of our effort to grow our network, expand our solution suite, and deliver exceptional services to our partner practices and their patients," said AON CEO Brad Prechtl, MBA.

AON Board Member & Chairman Dr. Stephen Orman added, "AmerisourceBergen is an industry leader with a proven track record of achieving exceptional results, and we look forward to collaborating further to expand our national oncology network."

AmerisourceBergen’s ION Solutions supports AON with GPO contracting. Oncology Supply handles distribution of chemotherapy and supportive care products to AON practices. Additional integrated service offerings available to AON practices through ION and Oncology Supply include:

Dedicated account teams that actively consult on inventory and create operational efficiencies
Robust inventory of specialty and full-line oral, infusible and injectable products
Technology, analytics and informatics solutions to drive higher-quality and lower-costs
Inventory management solutions
Centralized library of precision medicine testing recommendations and resources through ION Solutions’ Precision Medicine Center
Ongoing educational conference series designed to facilitate peer-to-peer learning and networking and strengthen the community of independent oncologists
The largest, longest-tenured pharmacy program that assists community oncology practices in successfully optimizing a medically integrated dispensing program
"As a company that’s pharmaceutical-centered and community focused, we strive for partners like AON," said Brian Ansay, President Specialty Physician Group Purchasing, AmerisourceBergen. "AON’s physician-led model and patient-centric services are creating new opportunities in community oncology, and we are excited about their vision for the future."

AON is physician led and physician governed. For more information about AON, please visit: www.AONcology.com.