On August 12, 2020 Neoleukin Therapeutics, Inc., "Neoleukin" (NASDAQ:NLTX), a biopharmaceutical company utilizing sophisticated computational methods to design de novo protein therapeutics, reported financial results for the second quarter ending June 30, 2020 and a midyear corporate update (Press release, Neoleukin Therapeutics, AUG 12, 2020, View Source [SID1234563921]).

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"All of the preclinical activities for NL-201 are substantially complete, and we are excited to be moving towards our first clinical trial in patients with advanced solid tumors," said Jonathan Drachman, M.D., Chief Executive Officer of Neoleukin. "To our knowledge, this will be the first fully de novo protein to enter clinical testing and has the potential to improve outcomes for patients with a wide variety of cancers. Additionally, our research group continues to advance future pipeline programs and to broaden the de novo protein design platform, some of which was highlighted during the recent AACR (Free AACR Whitepaper)-II Conference."

Recent Updates

NL-201 Development

In June, Neoleukin announced the presentation of preclinical data on its lead immunotherapy candidate NL-201, an IL-2 and IL-15 agonist, and applications of its de novo protein design platform at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II.

Key findings include the ability of NL-201 to potently stimulate and expand key cancer-killing cells and induce robust and durable antitumor activity across many tumor types as well as encouraging immunogenicity data in non-human primates. New data highlights the ability of NL-201 to stimulate and expand CD8+ and NK cells at very low doses with minimal impact on immunosuppressive regulatory T cells. Additionally, low incidence of immunogenicity was observed following five weekly doses of NL-201 in non-human primates.

Neoleukin remains focused on its efforts to support an Investigational New Drug (IND) Application for its lead therapeutic, NL-201, anticipated during the fourth quarter of 2020, and is working with key vendors to finalize all activities and study reports. At this time, Neoleukin does not expect a delay due to COVID-19 but acknowledges the potential exists for this timing to be impacted.

Other Applications of De Novo Protein Design Presented at AACR (Free AACR Whitepaper) Virtual Meeting

Research presented highlights a conditional activation approach – de novo split technology – demonstrating that de novo proteins can be divided into two inactive pieces that regain the ability to bind receptors when co-located in the tumor microenvironment. This split approach to conditional activation may increase the therapeutic index and offers the potential to create next-generation de novo therapeutics.

Additional research demonstrated that NL-201 and targeted variants may improve outcomes when combined with CAR-T cells in vivo in lymphoma and solid tumor models.

De Novo Protein Design for Coronavirus

In the past several months, Neoleukin scientists developed NL-CVX1, a fully de novo ACE2 decoy that binds to the SARS-CoV2 spike protein and neutralizes viral infection of mammalian cells in vitro. This protein is highly stable, was designed to be resilient to viral mutation, and has the potential for local administration to treat and/or prevent infection. The early results of this discovery research were recently posted on the public access website bioRxiv.org.

Board Appointment

In June, Neoleukin announced the appointment of Erin Lavelle to the Company’s Board of Directors. Ms. Lavelle has more than 20 years of strategic and operational leadership experience in the biopharmaceutical industry. Ms. Lavelle started her career in Merrill Lynch’s Investment Banking group, spent 15 years at Amgen, and most recently served as Chief Operating Officer for Alder Biopharmaceuticals, Inc.

Follow-On Offering

In July, Neoleukin closed a public offering of approximately 3.2 million shares of common stock and pre-funded warrants to purchase 1.7 million shares of common stock with an exercise price of $0.000001. The aggregate net proceeds received by the Company from the offering, after deducting offering fees and expenses, were $71.4 million. This is a subsequent event to the quarter. As such, Neoleukin’s cash balance as of June 30, 2020 does not include these proceeds.

Summary of Financial Results

Cash Position: Cash and cash equivalents totaled $129.6 million as of June 30, 2020 compared to $143.1 million as of December 31, 2019. The decrease in cash was primarily the result of cash used in operations as well as expenditures on property and equipment, offset partially by proceeds from the exercise of options.

Based upon our current operating plan, and following the completion of the July follow-on offering, Neoleukin believes that its cash-on-hand will be sufficient to fund operations into 2023.

R&D Expenses: Research and development expenses for the second quarter of 2020 increased to $4.8 million from ($2.0) million for the second quarter of 2019. This increase was primarily due to IND-enabling activities related to Neoleukin’s lead product candidate, NL-201, and advancement of other Neoleukin technologies, compared to a credit in expenses during Q2 2019 as a result of the suspension of all research and development activities from the former Aquinox operations in June 2018, as well as reductions to accrued research and development expenses given final costs were less than contracted.

G&A Expenses: General and administrative expenses for the second quarter of 2020 increased to $4.9 million from $2.4 million for the second quarter of 2019. The increase is primarily due to investments in G&A after the merger, completed in August 2019, compared to lower personnel and overhead costs as a result of Aquinox’s restructuring in the prior year.

Net Loss: Net loss for the second quarter of 2020 was $9.7 million compared to a net loss of $0.0 million in the second quarter of 2019 primarily due to increased costs related to Neoleukin’s lead candidate, NL-201, and other Neoleukin technologies in 2020, as well as the reductions to accrued research and development expenses during the second quarter of 2019.

Conference Call Information

Neoleukin will host a conference call today to provide a second quarter corporate update and review financials. Details are as follows:

Date: August 12, 2020
Time: 1:30 p.m. Pacific / 4:30 p.m. Eastern
Toll-free: (866) 357-7878
International: (315) 625-3088
Conference ID: 5862564
Webcast URL: View Source

The archived audio webcast with slides will be available on the Investor Relations section of the Neoleukin website approximately two hours after the event and will be available for replay for at least 30 days after the event.

On August 12, 2020 The University of Texas MD Anderson Cancer Center reported that a combination regimen of venetoclax and azacitidine was safe and improved overall survival (OS) over azacitidine alone in certain patients with acute myeloid leukemia (AML), according to the Phase III VIALE-A trial (Press release, MD Anderson, AUG 12, 2020, View Source [SID1234563693]).

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The results were presented in the virtual 25th European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress and were published today in the New England Journal of Medicine.

The addition of venetoclax, an inhibitor of the BCL-2, to azacitidine resulted in a median OS of 14.7 months compared to 9.6 months in patients receiving azacitidine alone. Additionally, 66.4% of patients receiving the combination therapy achieved complete remission, while azacitidine alone achieved a 28.3% complete remission rate.

The responses to treatment were both rapid and durable: 43% of patients in the combination therapy group exhibited a response to treatment during the first cycle, and the observed median duration of remission was 17.5 months.

Treating a subgroup of AML patients without effective therapeutic options

Although there is not yet a reliable standard treatment regimen for AML, many patients receive chemotherapy and/or a stem cell transplant. However, not all patients are eligible for these therapies.

"A large portion of patients with AML, including those older than 75 or those who have medical comorbidities, cannot tolerate existing treatment strategies, and the patients with AML who are ineligible for intensive chemotherapy often experience poor prognoses," said Courtney D. DiNardo, M.D., lead investigator and associate professor of Leukemia. "We launched the VIALE-A trial to evaluate whether we could safely use a combination therapy to treat this critical patient population."

In this multi-institution trial, 431 patients were randomized in a 2:1 ratio to receive either the combination of venetoclax and azacitidine or azacitidine plus placebo. The primary objective was to evaluate whether the combination improved OS compared to azacitidine, with additional goals to examine the safety of the combination therapy.

Combination treatment shows positive safety results

These results demonstrate that the combination of venetoclax and azacitidine has a safety profile similar to that of both drugs separately. The most common adverse events in both the experimental and placebo treatment groups were hematologic and gastrointestinal. In general, rates of adverse events were consistent between the two treatment groups, although a higher frequency of neutropenia (42% vs. 29%) and febrile neutropenia (42% vs.19%) was observed with the combination therapy comparted to azacitidine and placebo.

"The primary adverse events seen with azacitidine and venetoclax are related to increased cytopenias, including neutropenia and neutropenia-related infections," said DiNardo. "Key management guidelines include dosing interruptions between cycles to allow for count recovery in the setting of a leukemia-free marrow, and the use of granulocyte colony-stimulating factor as an adjunct to improve neutrophil count once a patient is in remission."

New research provides options for patients

This research is likely to be practice-changing for the treatment of some groups of patients with AML. Additional research is needed to evaluate how new therapies, including this combination therapy, can improve outcomes for all patients with AML.

"While this combination represents a key advance in AML therapy, improving both remission and survival rates in newly diagnosed patients with AML, many unfortunately will still relapse," said DiNardo. "Our next steps include an evaluation of azacitidine and venetoclax as a backbone to which additional novel therapeutics are being evaluated in particularly high risk populations."

This trial (NCT02993523) was supported by Abbvie and Genentech. A full list of co-authors and their disclosures is included in the paper.

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.