On August 12, 2020 KemPharm, Inc. (OTCQB: KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, reported its financial results for the second quarter ended June 30, 2020 (Press release, KemPharm, AUG 12, 2020, View Source [SID1234563603]).

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"The second quarter was a period of continued advancement at KemPharm, highlighted by the FDA’s acceptance of the New Drug Application (NDA) for KP415 and, following that, receipt of the ‘Day-74 Letter’ from the FDA," said Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm. "Contained within the Day-74 Letter were two important updates from the FDA. The FDA informed us that the action date (PDUFA) for KP415 is March 2, 2021, and that an advisory committee is not required at this time."

Dr. Mickle continued, "Dovetailing with these important regulatory advancements, KemPharm also reported two key events in our collaboration with Gurnet Point Capital (GPC). Upon the FDA’s acceptance of the KP415 NDA, KemPharm received the $5 million milestone payment as provided by the definitive collaboration and license agreement. Additionally, Corium, Inc., a GPC portfolio company, will lead all commercialization activities for KP415, providing what we believe is the most well-suited and experienced commercial organization to launch and maximize the potential market for KP415. In addition, that opportunity has since been strengthened by the recent issuance of two U.S. patents governing KP415 and KP484. Both patents have an expiration date of December 9, 2037, which is an extension of approximately five years beyond prior serdexmethylphenidate (SDX) patents."

Dr. Mickle concluded, "Based on these recent events and accomplishments, we are very optimistic about KemPharm’s short- and long-term prospects. While certain headwinds remain, momentum continues to build behind the value potential of KP415, and we are excited to be working with the Corium team to develop the marketing and commercialization strategy for KP415, which we look forward to unveiling as soon as we are in a position to do so."

Q2 2020 Financial Results:

For Q2 2020, KemPharm reported revenue of $6.9 million which is comprised of a $5.0 million milestone payment received upon the FDA’s acceptance of the NDA for KP415 and $1.9 million from research and development services under the definitive collaboration and license agreement (KP415 License Agreement) and other consulting arrangements, as compared to Q1 2020 revenue of $2.1 million. KemPharm had no revenue in Q2 2019. This is KemPharm’s fourth sequential quarter reporting revenue, and the Company expects to continue earning revenue as the Company provides services under the KP415 License Agreement and other consulting arrangements.

KemPharm’s net income for Q2 2020 was $0.9 million, or $0.01 per basic share and diluted share, compared to a net loss of $9.3 million, or $0.33 per basic and diluted share for the same period in 2019. Net income for Q2 2020 was driven primarily by operating income of $2.6 million, partially offset by net interest expense and other items of $1.7 million. The net operating income of $2.6 million for Q2 2020 was an increase of $10.4 million compared to a net operating loss of $7.8 million in the same period in 2019, which was primarily due to revenue of $6.9 million, a decrease in research and development expenses of $2.8 million and a decrease in general and administrative expenses of $1.3 million, partially offset by royalty and direct contract acquisition costs of $0.6 million.

As of June 30, 2020, total cash, cash equivalents and restricted cash was $6.6 million, which was an increase of $4.0 million compared to March 31, 2020. Based on the Company’s current operating forecast, the Company believes that its expected revenues and existing resources are sufficient to continue operations past the potential March 2, 2021 PDUFA date for the KP415 NDA and up to the debt maturity date of March 31, 2021.

Conference Call Information:

KemPharm will host a conference call and live audio webcast with slide presentation on Wednesday, August 12, 2020, at 4:30 p.m. ET, to discuss its corporate and financial results for the second quarter 2020. Interested participants and investors may access the conference call by dialing either:

(866) 395-2480 (U.S.)
(678) 509-7538 (international)
Conference ID: 5176398
An audio webcast with slide presentation will be accessible via the Investor Relations section of the Company’s website, View Source An archive of the webcast and presentation will be available for 90 days beginning at approximately 5:30 p.m. ET, on August 12, 2020.

On August 12, 2020 Epigenomics AG (FSE: ECX, OTCQX: EPGNY, the "Company") reported operational highlights and financial results for the second quarter and first half of 2020 (Press release, Epigenomics, AUG 12, 2020, View Source [SID1234563555]).

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Operational highlights

In late February 2020, the Centers for Medicare and Medicaid Services (CMS) initiated the National Coverage Determination (NCD) review process on Epi proColon. This milestone will result in a proposed NCD by August 28 and a final reimbursement decision by the end of November. The public response during the initial 30-day public comment period in March was overwhelmingly positive. Epigenomics’ management is confident that the public support combined with the results of the microsimulation model and the major FDA approval studies will lead to a positive reimbursement decision.
Moreover, the National Comprehensive Cancer Network (NCCN) – a major medical society in the U.S.A. – included Epi proColon in April 2020 in its colorectal cancer (CRC) guidelines. The inclusion of Epi proColon in accordance with FDA-approved indications underlines the blood test’s potential to significantly increase CRC screening rates in the U.S.A.
A study published last week in the Journal of the National Cancer Institute (JNCI) concluded that Epi proColon(R) is the test of choice for the millions of individuals not willing to participate in FIT or colonoscopy screening. Annual mSEPT9 screening resulted in more Quality-adjusted-life-years gained (QALYG), CRC cases averted and CRC deaths averted than both annual FIT screening and Cologuard(R) every three years, albeit at a higher colonoscopy referral rate.
As a result of the capital increase successfully completed at the end of March and the cost-cutting measures taken in connection with the ongoing Covid-19 pandemic – including reduction in Executive Board and Supervisory Board compensation – the Company had sufficient cash and cash equivalents at the end of June 2020 to finance the Company well into the first quarter of 2021.
Greg Hamilton, CEO of Epigenomics AG commented: "With the recent JNCI study publication adding the robust body of evidence supporting Epi proColon we are eagerly anticipating the upcoming NCD publication within the next two weeks. CMS has indicated they are on track to issue the decisions on time despite the Covid 19 pandemic. With the upcoming reimbursement proposal at the end of August and the final decision by the end of November 2020, we are closer than ever to our major goal of CMS reimbursing Epi proColon. A positive reimbursement decision would be the breakthrough for our Company and we are confident that CMS will recognize the contribution that Epi proColon can make to the fight against colorectal cancer and thus will decide in the best interest of patients."

6M 2020 financial results

Total revenue in the first six months of 2020 decreased by 53% to EUR 322 thousand (6M 2019: EUR 679 thousand) compared to the same period of the previous year, due to the effects of Covid-19 in the second quarter. Epigenomics experienced significant decline in April and May but has seen a recovery in June and July. Product revenue fell from EUR 660 thousand in the first half of 2019 to EUR 293 thousand in the reporting period.
Research & development costs declined from EUR 3,867 thousand in the prior year to EUR 2,754 thousand. The decrease was mainly driven by the Covid-19 interruption of almost all clinical studies in the U.S.A., specifically the post-approval study for Epi proColon.
Selling, general and administrative costs also decreased by EUR 958 thousand to EUR 3,901 thousand (6M 2019: EUR 4,859 thousand) in the reporting period, due to the reduction in sales and marketing activities, as virtually all relevant events such as conferences and trade fairs were cancelled due to the pandemic.
Overall, operating costs fell from EUR 9.4 million to EUR 7.4 million compared to the first half of the previous year due to the impact of Covid-19 and the Company’s subsequent cost reduction measures.
Adjusted EBITDA (before share-based payment expenses) for the first six months of 2020 was EUR -5.7 million (6M 2019: EUR -7.2 million).
The net loss for the period improved to EUR 6.4 million (6M 2019: EUR 7.4 million); the loss per share was EUR 0.14 (6M 2019: EUR 0.21). This was also impacted by the increased number of shares resulting from the capital increases executed in the second half of 2019 and in March 2020.
The cash outflow from operating activities fell significantly in the first half of 2020 by EUR 2.3 million to EUR 5.5 million (6M 2019: 7.8 million). This is due to both the improved operating result (EBIT) and changes in current assets.
As of June 30, 2020, the Company had cash and cash equivalents of EUR 8.7 million (including marketable securities) compared to EUR 11.0 million at the end of 2019.
Outlook 2020:
Revenue / EBITDA / Cash consumption

Due to the continued uncertainty surrounding the effects of Covid-19, and like many publicly traded companies, Epigenomics AG is pulling its revenue guidance for 2020, however, the Company maintains its previous, adjusted EBITDA (before share-based payment expenses) guidance of between EUR -10.5 million to EUR -12.5 million and cash consumption guidance in a range of EUR 10.5 million to EUR 12.5 million.
Further Information

The financial report for the first six months of 2020 is available on the Epigenomics website: View Source." target="_blank" title="View Source." rel="nofollow">View Source

Conference call for analysts and investors

Epigenomics AG will host a conference call for analysts and investors today at 4.00 pm (CET) / 10.00 am (EDT). The presentation can be accessed on the Company’s website: View Source

Participants are kindly asked to dial in 10 minutes prior to the start of the call.

An audio replay of the conference call will be provided on the Company’s website subsequently.

On August 12, 2020 Dune Medical Devices reported IMPACT Lourdes, the women’s philanthropy group that supports the Mercy Health Foundation Lourdes, has awarded its annual grant to benefit Mercy Health — Lourdes Hospital (Press release, Dune Medical Devices, AUG 12, 2020, View Source [SID1234563552]).

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IMPACT Lourdes members voted to award $30,000 to purchase the MarginProbe system, equipment that assists in detecting cancerous breast tissue.
With MarginProbe, surgeons can assess breast tissue in the operating room to give them greater confidence that they successfully removed all the cancer in the first lumpectomy surgery.

"Thank you to all the members of IMPACT Lourdes for supporting our MarginProbe project," said surgeon Dr. Daniel Howard. "We already had one of the best breast cancer operations in the community and region, and thanks to IMPACT it’s now even better. Our breast cancer re-operation rate with this equipment is less than two% while the national average is 16%. We anticipate our MarginProbe equipment will benefit more than 200 lives each year thanks to the generosity of IMPACT Lourdes." Dr. Howard is a general surgeon with Mercy Health who specializes in breast cancer surgery.

On August 12, 2020 MANA RBM and CUSP reported the randomization of the first subject in ESCALATE, A Phase III Randomized Study Comparing Enzalutamide or Darolutamide with Radium-223 vs Enzalutamide or Darolutamide with Placebo and the Effect upon Symptomatic Skeletal Event-Free Survival for mCRPC Patients, clinicaltrials.gov #NCT04237584] (Press release, MANA RBM, AUG 12, 2020, View Source [SID1234563545]).

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Enrolment is planned for patients with metastatic prostate cancer which progressed with initial androgen deprivation therapy (mCRPC). Patients will be randomized to either darolutamide or enzalutamide, focusing upon detailed safety and tolerability analyses, in addition to clinical outcome assessments, with subsequent addition of radium-223 dichloride or placebo. This phase 3 trial plans to accrue approximately 500 subjects.

Dr. Neal Shore of Carolina Urologic Research Center, the Principal Investigator for the trial, designed the treatment paradigm in collaboration with Dr. Oliver Sartor, Professor at Tulane University School of Medicine. Dr. Shore stated, "The eventual outcome analytics may potentially enhance treatment options for mCRPC as patients as well as augment our understanding of advanced prostate cancer sequencing strategies. We are honored to have enrolled and randomized the first subject and are very appreciative of our trial site organization’s ongoing commitment."

The CUSP Group is a Clinical Research Consortium composed of 24 large tertiary uro-oncology community research practices. As a trial management organization, CUSP supported ESCALATE protocol development, constituted the study network and conducted preliminary protocol education as well as rapid budget and CTA harmonization. As a research advocacy group, CUSP endeavors to bring scientifically interesting and operationally well-crafted trials to its members. These CUSP consortium exclusive studies allow for the early adoption and rapid diffusion of new technologies, medicines, and procedures into the affiliated clinical practices. Additionally, as part of this CUSP advocacy mission, Thomas Paivanas, Executive Director of CUSP, said, "We are excited to be testing a complete digital research ePlatform developed by our colleagues at MANA RBM to conduct overall trial management and remote oversite. These endeavors promise to provide an integrated, potentially paperless system that works well for our research sites, saving them time and enhancing quality."

MANA RBM, the study sponsor, is a clinical trials institute and research laboratory. It is a leader in designing, testing, publishing, and pioneering enhanced methods for remote trial management and quality risk- based monitoring oversight for clinical trials. "For nearly a decade, we have continually enhanced our systems for remote trial management. We are excited to work with Drs. Shore and Sartor in collaboration with the CUSP Clinical Research Consortium to conduct this critical study and to enhance the way sites are able to conduct clinical research," said Penelope Manasco, M.D., CEO of MANA RBM. "We are also excited to announce another unique aspect of this Trial – the launch of REACHERTM. It is the first clinical trial quality management system, based on patent pending software, designed to automatically identify critical, protocol specific errors and systematic errors in near real time across all of the myriad of clnical trial technology systems."

On August 12, 2020 Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, reported financial results for the second quarter ended June 30, 2020 and provided a clinical and corporate update (Press release, Milestone Pharmaceuticals, AUG 12, 2020, View Source [SID1234563544]).

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"With the benefit of having completed our first Phase 3 clinical trial and a clear and efficient registration path defined for etripamil in paroxysmal supraventricular tachycardia (PSVT), we remain fully committed to bringing this therapy to PSVT patients highly in need of better treatment options," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "We are on track to reopen the RAPID study later this year and believe the updated study design will allow us to best maximize the potential clinical utility of etripamil, including in patients with more persistent supraventricular tachycardia (SVT) events. With our recent $25 million private placement, we believe we are well positioned to execute on the remainder of the PSVT program and look forward to providing updates on our progress in the coming months."

Recent Updates

Announced Regulatory Guidance and Updated Clinical Development Plan for Etripamil in PSVT. In July 2020, Milestone announced a clinical and regulatory update for its pivotal program with etripamil in PSVT following interactions with the U.S. Food and Drug Administration (FDA).

The FDA indicated that two studies, the second part of NODE-301, which the Company has renamed the RAPID study, and the completed NODE-301 study could potentially fulfill the efficacy requirement for Milestone’s New Drug Application for etripamil in patients with PSVT. In addition, the FDA agreed to the following program changes:

An updated statistical analysis plan (SAP) for both the RAPID and NODE-301 studies under which the primary efficacy endpoint will be defined as time to conversion over the first 30 minutes, with a target p-value of less than 0.05 for each study. When employing this updated SAP, results from the NODE-301 study show that 54% of etripamil patients vs. 35% of placebo patients converted within 30 minutes (HR 1.87, p=0.02).
In an effort to maximize the potential treatment effect of etripamil, the RAPID study will allow for an optional repeat administration of study drug (either 70 mg of etripamil or placebo) in patients who have not experienced symptom relief within 10 minutes of the first study drug administration.
Milestone expects to reopen enrollment in the RAPID study later this year, with data anticipated in late 2021 or early 2022.

Raised $25 Million Through Private Placement. In July 2020, Milestone entered into a securities purchase agreement with affiliates of an existing shareholder, RTW Investments, LP, whereby Milestone sold pre-funded warrants to acquire an aggregate 6,655,131 common shares at a purchase price of $3.7465 per pre-funded warrant. Each pre-funded warrant is exercisable for one common share at an exercise price of $0.01 per share, has no expiration date, and is immediately exercisable, subject to certain beneficial ownership limitations. Aggregate proceeds received by the Company were $25 million.
Second Quarter 2020 Financial Results

As of June 30, 2020, Milestone had cash, cash equivalents, and short-term investments of $85.4 million and 24.7 million shares outstanding.
Research and development expenses for the second quarter of 2020 were $8.6 million compared with $10.5 million for the prior year period. For the six months ended June 30, 2020, research and development expenses were $20.5 million compared with $18.3 million for the prior year period. The increase in the first half of 2020 reflects the increased clinical development costs and manufacturing and formulation activities supporting Milestone’s Phase 3 clinical trial and the efforts in developing a clinical pathway for etripamil.
General and administrative expenses for the second quarter of 2020 were $3.0 million compared with $1.6 million for the prior year period. For the six months ended June 30, 2020, general and administrative expenses were $5.7 million compared with $2.6 million for the prior year period. The increase reflects additional increasing insurance costs, professional fees and administrative headcount in the first half of 2020 compared to the same period in 2019 primarily to support the compliance requirements of being a public company.
Commercial expenses for the second quarter of 2020 were $1.5 million compared with $2.2 million for the prior year period. For the six months ended June 30, 2020, commercial expense was $3.7 million compared with $4.4 million for the prior year period. The decrease in the three and six months ended June 30, 2020 reflects the efforts in reducing operating expenses affecting primarily pre-commercialization activities as Milestone focuses its efforts on an optimized clinical development pathway for etripamil.
For the second quarter of 2020, operating loss was $13.1 million compared to $14.3 million in 2019. For the six months ended June 30, 2020, Milestone’s operating loss was $29.9 million compared to $25.3 million in the prior year period.
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 patients with atrial fibrillation with a rapid ventricular rate, with subsequent studies expected in other conditions where calcium channel blockers are used.