On May 13, 2021 KemPharm, Inc. (NASDAQ: KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, reported its financial results for the first quarter ended March 31, 2021 (Press release, KemPharm, MAY 13, 2021, View Source [SID1234580042]).

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"The first quarter of 2021 was nothing short of transformational for KemPharm, highlighted by the FDA approval of AZSTARYS, the completion of our financial restructuring, and the re-listing of our shares on Nasdaq," said Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm. "As a result, we stand today as a company with a solid balance sheet and capital structure that is moving full force with our partners at GPC and Corium to soon launch AZSTARYS as a once-daily product for the treatment of attention deficit hyperactivity disorder (ADHD) in patients age six years and older."

Dr. Mickle continued, "Following the close of the quarter, KemPharm further strengthened its position by agreeing to amend the License Agreement with an affiliate of GPC. We are now eligible to receive up to $590 million in future regulatory and sales milestone payments for AZSTARYS, as well as tiered royalty payments on a product-by-product basis for net sales. This is a significant increase from the original License Agreement and also provides an opportunity to adjust the economics of the License Agreement to optimize investment in the commercial launch of AZSTARYS. Ultimately, we believe this arrangement has the opportunity to produce significant shareholder value based on the market outlook for AZSTARYS."

Dr. Mickle continued, "As we have noted in prior communications, KemPharm believes that the product label for AZSTARYS is potentially best-in-class, with several elements in the label providing clear points of differentiation from other commercially available methylphenidate ADHD products. We were pleased with the recent determination that serdexmethylphenidate (SDX), was classified as a Schedule IV controlled substance by the Drug Enforcement Administration (DEA) following a thorough review by the U.S. Department of Health and Human Services (HHS). SDX comprises 70% of the active pharmaceutical ingredient (API) in AZSTARYS, which is classified as a Schedule II controlled substance. In short, the agencies determined that SDX has a generally low potential for abuse and a lower potential for abuse when compared to d-MPH, a Schedule II controlled substance. Having SDX designated as a Schedule IV controlled substance, we believe, potentially increases AZSTARYS’ appeal among prescribers and patients."

Dr. Mickle concluded, "Further, the Schedule IV classification of SDX is a significant development for our lead clinical product candidate, KP879, an extended-duration, agonist replacement therapy for the treatment of Stimulant Use Disorder (SUD), as SDX is the only API in KP879. We now look forward to initiating the clinical program for KP879 in 2021 after receiving FDA clearance for the Investigational New Drug (IND) application. If approved, KP879 could be a Schedule IV product, and physicians who are treating patients seeking to overcome addictions to cocaine, methamphetamine or other stimulants may be able to prescribe KP879 with the knowledge that the product candidate could have a significantly lower potential for abuse."

Q1 2021 Financial Results:

For Q1 2021, KemPharm reported revenue of $12.1 million, which was primarily derived from a $10 million milestone payment earned upon the AZSTARYS NDA approval and service fee revenue of $2.1 million, as compared to Q4 2020 revenue of $2.4 million, which was derived primarily from service fee revenue. Current consulting arrangements contractually continue through March 2022.

KemPharm’s net loss for Q1 2021 was $10.3 million, or $0.54 per basic share and diluted share, compared to net loss of $5.8 million, or $1.92 per basic and diluted share for the same period in 2020. Net loss for Q1 2021 was driven primarily by a non-cash loss on extinguishment of debt of $16.9 million and net interest expense and other items of $0.4 million, partially offset by operating income of $7.0 million. The net operating income of $7.0 million for Q1 2021 was a change of $10.7 million compared to net operating loss of $3.8 million in the same period in 2020, which was primarily due to an increase in revenue of $10.0 million related to the milestone payment and a net decrease in operating expenses of $0.7 million period over period. The net decrease in operating expenses was primarily due to a decrease in severance expense of $0.8 million and a decrease in general and administrative expenses of $0.4 million, partially offset by an increase in royalty and direct contract acquisition costs of $0.3 million and an increase is research and development expenses of $0.1 million.

As of March 31, 2021, total cash, cash equivalents and restricted cash was $76.0 million, which was an increase of $71.7 million compared to December 31, 2020. The increase was driven by the Company’s multi-phase financial restructure process which was completed during the quarter.

As of March 31, 2021, total shares of common stock outstanding was 28,480,156 shares, and fully diluted common shares outstanding was 38,379,718 shares, which included 9,544,693 shares issuable upon exercise of warrants. In addition, no preferred stock is outstanding as of March 31, 2021.

"KemPharm has emerged from Q1 2021 as a Nasdaq-listed company with no debt and significant cash holdings on the balance sheet," said LaDuane Clifton, KemPharm’s Chief Financial Officer. "We have the resources needed to continue the development of KP879, and we have begun evaluating how to efficiently deploy capital to generate additional value streams for shareholders. There are many opportunities to explore, both internally and externally, and creating long-term value is top of mind."

Conference Call Information:

Interested participants and investors may access the conference call by dialing either:

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 May 13, 2021.

About AZSTARYS:

AZSTARYS is an FDA-approved, once-daily product for the treatment of attention deficit hyperactivity disorder (ADHD) in patients age six years or older. AZSTARYS consists of SDX, KemPharm’s prodrug of d-methylphenidate (d-MPH), co-formulated with immediate release d-MPH.

On May 13, 2021 Savara Inc. (Nasdaq: SVRA), a clinical stage biopharmaceutical company focused on rare respiratory diseases, reported financial results for the first quarter ending March 31, 2021 and provided a business update (Press release, Savara, MAY 13, 2021, View Source [SID1234580041]).

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"We made considerable progress in the first quarter of the year," said Matt Pauls, Chair and Chief Executive Officer, Savara. "This includes the strategic decision to wind down two pipeline programs, enabling us to focus solely on advancing our lead asset, molgramostim in aPAP. Additionally, we started the onboarding process of a global, full-service contract research organization that is partnering with us to execute the IMPALA-2 trial. Lastly, we strengthened our cash position through a $130M equity raise. With a balance sheet of approximately $193M, we believe our runway extends through 2025 – well beyond the expected top line results of IMPALA-2 in 2Q 2024."

"We are pleased to announce that the IMPALA-2 site activation process has begun," said Badrul Chowdhury, Chief Medical Officer, Savara. "Sites will be activated on a rolling basis, with approximately 10-15 expected to be active by the end of June. As sites launch, patients are evaluated, and those who are eligible will begin a six-week screening period. This means patients could be randomized and dosed by the end of 2Q or early 3Q 2021. We also recently strengthened our management team with the hire of Dr. Dhaval Desai as Head of Clinical Development and Brian Maurer as Head of Clinical Operations. Both are seasoned professionals with deep expertise in running global clinical trials."

First Quarter Financial Results (Unaudited)

Savara’s net loss attributable to common stockholders for the three months ended March 31, 2021 was $10.2 million, or $(0.13) per share, compared with a net loss attributable to common stockholders of $15.4 million, or $(0.27) per share, for the three months ended March 31, 2020.

Research and development expenses decreased by $5.6 million, or 42.5%, to $7.6 million for the three months ended March 31, 2021 from $13.2 million for the three months ended March 31, 2020. The decrease is largely attributable to $5.4 million of costs for the acquisition of inhaled liposomal ciprofloxacin (Apulmiq) in March 2020. There were no research and development costs incurred related to Apulmiq during the three months ended March 31, 2021.

General and administrative expenses decreased by approximately $0.2 million, or 6.8%, to $2.8 million for the three months ended March 31, 2021 from $3.0 million for the three months ended March 31, 2020. This was primarily due to a decrease in noncash stock-based compensation and personnel costs for the three months ended March 31, 2021.

As of March 31, 2021, Savara had cash, cash equivalents, and short-term investments of approximately $192.7 million and debt of approximately $25 million.

On May 13, 2021 Biogen Inc. (Nasdaq: BIIB) and Envisagenics reported a new collaboration to advance ribonucleic acid (RNA) splicing research within central nervous system (CNS) diseases (Press release, Biogen, MAY 13, 2021, View Source [SID1234580040]). As part of the collaboration, Biogen will leverage Envisagenics’ proprietary artificial intelligence (AI)-driven RNA splicing platform, SpliceCore, to define and understand the regulation of different RNA isoforms in CNS cell types.

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Genetic information encoded in the human chromosome is converted into RNA molecules which is then used as the template to make proteins. RNA splicing is the process that trims out extra information embedded in the intermediate RNA molecules, and this trimmed RNA is what is then used to produce functional proteins.

"Since Biogen’s earliest days, RNA splicing has been an integral part of our history and mission dating back to co-founder Phillip Sharp’s discovery of the process in 1977," said Alfred Sandrock, Jr., M.D., Ph.D., head of research and development at Biogen. "By combining Envisagenics’ SpliceCore platform with our deep expertise in this scientific approach, we believe that Biogen will be able to advance our understanding of RNA splicing and potentially identify new drug targets for CNS diseases."

"Envisagenics is thrilled to work with Biogen because we share a commitment to identifying potential treatments for CNS diseases through innovative AI technology like the SpliceCore platform. Envisagenics and Biogen recognize the power of RNA splicing to aid in the discovery of potential therapeutics," said Maria Luisa Pineda, Ph.D., chief executive officer of Envisagenics. Envisagenics’ Chief Technology Officer, Martin Akerman, Ph.D., added, "scientists have only recently been able to uncover disease-causing novel isoforms at scale, thanks to improvements in the speed and sensitivity of bioinformatics software like SpliceCore."

Traditionally, the process of detecting, cataloging and interpreting RNA splicing errors has been laborious, slow and costly. However, by tapping into Envisagenics’ machine learning algorithms and high-performance computing, Biogen may now be able to identify, test and validate splicing errors at scale. Through this collaboration, Biogen will gain access to SpliceCore’s database of approximately seven million potential RNA splicing errors, which is the largest database of splicing errors in the world. This will provide Biogen with a broader lens to evaluate splicing events that may be targeted for therapeutic gain. In addition, collaboration aligns to Biogen’s broader objective of identifying and validating genetic targets of disease to increase the probability of success in CNS drug discovery.

On May 13, 2021 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of precision genetic medicines, reported its financial results for the three- and six-month periods ended March 31, 2021 (Press release, NeuBase Therapeutics, MAY 13, 2021, View Source [SID1234580039]).

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"We continue to expand and scale our unique precision genetic medicine platform that we believe can turn genes on, off, or edit them in vivo, and thus address most mechanisms that cause diseases in a single industry-unifying solution," said Dietrich A. Stephan, Ph.D., Founder, CEO and Chairman of NeuBase. "Our recent financing led by top-tier healthcare investors enables us to advance our lead program into the clinic next year and expand our pipeline to address historically undruggable oncogenic driver mutations. We look forward to hosting our R&D day on June 8th, during which we will present an update on our current pipeline programs, as well as introduce an oncology program targeting a genetic driver mutation in a high value indication."

Second Quarter of Fiscal Year 2021 and Recent Operating Highlights

Strengthened the balance sheet with a public equity offering generating $42.6 million in net proceeds, which will enable the Company to advance its lead program into the clinic and expand the pipeline of therapies for rare and common diseases
Expanded platform capabilities and consolidated PNA intellectual property through the acquisition of new scaffold technologies from Vera Therapeutics, formerly known as TruCode Gene Repair, Inc., in late April 2021
Presented positive in vitro and in vivo preclinical data for the Company’s lead drug candidate for the treatment of DM1 at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference
Moved into a newly built state-of-the-art laboratory and office space in Pittsburgh, providing the infrastructure to support accelerating R&D activities
Appointed Gerald McDougall, industry veteran, to the Board of Directors, bringing large-scale partnership and strategy expertise to the Company
Financial Results for the Second Fiscal Quarter Ended March 31, 2021

As of March 31, 2021, the Company had cash and cash equivalents of approximately $24.2 million, compared with cash and cash equivalents of approximately $32.0 million as of September 30, 2020
Subsequent to the end of the fiscal quarter ended March 31, 2021, the Company completed a public equity offering generating net proceeds of approximately $42.6 million
NeuBase estimates its current cash and cash equivalents are sufficient to fund currently planned operating and capital expenditures into the first quarter of CY2023
For the three-month period ended March 31, 2021, the Company reported a net loss of approximately $5.5 million, or a net loss of $0.24 per share, compared with a net loss of approximately $4.4 million, or a net loss of $0.26 per share, for the three-month period ended March 31, 2020
For the three-month period ended March 31, 2021, total operating expenses were approximately $5.9 million, consisting of approximately $2.7 million in general and administrative expenses and $3.2 million of research and development expenses; compared with total operating expenses of $4.4 million for the three-month period ended March 31, 2020, which was comprised of approximately $2.7 million in general and administrative expenses and $1.6 million in research and development expenses
Financial Results for the Six-Month Period Ended March 31, 2021

For the six-month period ended March 31, 2021, the Company reported a net loss of approximately $9.6 million, or a net loss of $0.41 per share, compared with a net loss of approximately $8.9 million, or a net loss of $0.52 per share, for the same period last year
For the six-month period ended March 31, 2021, total operating expenses were approximately $10.6 million, consisting of approximately $5.4 million in general and administrative expenses and $5.2 million of research and development expenses. This compares with total operating expenses of $8.1 million for the same period last year, which was comprised of approximately $5.3 million in general and administrative expenses and $2.8 million in research and development expenses

On May 13, 2021 iTeos Therapeutics, Inc. (Nasdaq: ITOS), a clinical-stage biopharmaceutical company pioneering the discovery and development of a new generation of highly differentiated immuno-oncology therapeutics for patients, reported financial results for the first quarter ended March 31, 2021 and provided recent business highlights (Press release, iTeos Therapeutics, MAY 13, 2021, View Source [SID1234580038]).

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"Our current clinical strategy includes six studies for our two clinical programs in different indications and combinations that are expected to provide meaningful readouts before the end of 2022. In addition to our recent EOS-448 Phase 1 data presentation at AACR (Free AACR Whitepaper), we also continue to advance our adenosine A2A receptor antagonist, inupadenant, and we look forward to reporting updated data with evidence that expression of the A2A receptor in tumor biopsy samples is associated with anti-tumor benefit at the upcoming ASCO (Free ASCO Whitepaper) meeting in June," said Michel Detheux, PhD, president and chief executive officer of iTeos. "In addition to this progress across our two lead programs, we also remain committed to our ongoing discovery efforts to identify and advance new novel product candidates that could expand our pipeline and continue to serve our mission to improve the lives of people with cancer."

Program Highlights

EOS-448: EOS-448 is an IgG1 antibody with the ability to engage the Fc gamma receptor (FcγR) and to enhance the anti-tumor response through a multifaceted mechanism.

The company presented initial clinical and safety data from the monotherapy dose escalation part of the Phase 1 trial in adult patients with advanced solid tumors at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2021. These preliminary data showed the drug was well-tolerated across dose levels, caused depletion of TIGIT-expressing Treg cells in the blood, providing evidence of target and FcyR engagement, and had encouraging signs of anti-cancer activity.

The company plans to advance EOS-448 into combination trials in both checkpoint-naïve and resistant patients in mid-2021. These Phase 1b trials will assess the safety of EOS-448 in combination with pembrolizumab and with iTeos’ novel agent inupadenant in patients with solid tumors, and as a monotherapy and in combination with an Immunomodulatory Drug (IMiD) in patients with multiple myeloma.
Inupadenant (EOS-850): Designed as a highly selective small molecule antagonist of the adenosine A2A receptor. Inupadenant is currently in an open-label multi-arm Phase 1/2a clinical trial in adult patients with advanced solid tumors.

iTeos is currently enrolling patients in three distinct cohorts in its Phase 1/2a clinical trial as both as a single agent and in combination. The initial cohort is evaluating inupadenant as a monotherapy in prostate cancer, and the second cohort is evaluating the safety of inupadenant in combination with pembrolizumab in patients with solid tumors with planned expansions in prostate cancer and melanoma. The final cohort is evaluating inupadenant in combination with chemotherapy in patients with triple-negative breast cancer.

iTeos plans to report updated single-agent data, including results from tumor biopsy analyses as part of an e-poster at the upcoming 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting held virtually June 4-8, 2021. The abstract will be available on Wednesday, May 19th at 5:00 p.m. ET, and the e-poster will be available for on-demand viewing starting on Friday, June 4 at 9:00 a.m. ET.

Abstract Title: Phase 1 trial of the adenosine A2A receptor antagonist inupadenant (EOS-850): Update on tolerability, and antitumor activity potentially associated with the expression of the A2A receptor within the tumor.
Abstract Number: 2562
Preclinical programs: iTeos continues to progress research programs focused on additional targets that address additional pathways of immunosuppression and complement the mechanism of action of A2AR and TIGIT programs. iTeos expects to nominate an additional product candidate for Investigational New Drug-enabling studies before the end of 2021.

Upcoming Events

Corporate presentation at the Jefferies Healthcare Conference, June 1-4, 2021
Present on inupadenant in an e-poster at the ASCO (Free ASCO Whitepaper) Annual Meeting, June 4-8, 2021
Corporate presentation at the Citi European Healthcare Conference, June 15-16, 2021
First Quarter 2021 Financial Results

Cash Position: The Company had cash and cash equivalents of $321.4 million as of March 31, 2021, compared to $147.7 million as of March 31, 2020. This cash balance provides a runway into 2023.
Research and Development (R&D) Expenses: R&D expenses were $11.6 million for the quarter ended March 31, 2021, compared to $5.8 million for the same quarter of 2020. This increase was primarily due to an increase in activities related to clinical trials for inupadenant and EOS-448 and increased headcount.
General and Administrative (G&A) Expenses: G&A expenses were $7.0 million for the quarter ended March 31, 2021, compared to $2.4 million for the same quarter of 2020. The increase was primarily due to increased headcount and professional fees and other costs associated with becoming a publicly traded company.
Net Loss: Net loss attributable to common shareholders was $13.5 million, or a net loss of $0.39 per basic and diluted share, for the quarter ended March 31, 2021, as compared to $6.5 million, or a net loss of $25.53 per basic and diluted share, for the same quarter of 2020.
Conference Call Details:
iTeos Therapeutics will host a conference call and webcast today, Thursday, May 13th, at 4:30 p.m. ET. To access the live event, please dial the numbers and reference the conference ID listed below. A live audio webcast of the event will also be accessible from the Events page of the Company’s website at View Source archived webcast will be available approximately two hours after the completion of the event and for 30 days following the call.