On May 13, 2021 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported its financial results for the quarter ended March 31, 2021 and provided a business update (Press release, Phio Pharmaceuticals, MAY 13, 2021, View Source [SID1234579938]).

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"This year has started out with great momentum across our pipeline, as we continue to present a steady flow of positive data that further demonstrate the value of direct therapy with INTASYL compounds, as well as their application in improving the efficacy of cell-based immunotherapy products. Results from various animal studies, including data presented at the AACR (Free AACR Whitepaper) conference last month, show how INTASYL compounds directly delivered to the tumor can activate the immune response resulting in strong anti-tumor effects. We also presented additional compelling data showing improvements of T cell immunotherapy products by reprogramming them with INTASYL compounds, and their resulting increased in vivo efficacy. For example, at the ASGCT (Free ASGCT Whitepaper) conference this week, we announced data showing how PH-762 can unlock the potential of CAR-T cell therapy in solid tumors. These data provide proof-of-concept for the application of PH-762 mediated PD-1 checkpoint silencing in CAR-T cells prior to adoptive cell therapy to enhance the therapeutic efficacy of CAR-T cell therapy. This is tremendous data for our program, especially when considering a recently published clinical study showing that similar strategies using CRISPR-Cas9 mediated PD-1 disruption in T cells is much less efficient," said Dr. Gerrit Dispersyn, President and CEO of Phio.

"As a result of the overwhelmingly positive data we’ve collected across our pipeline, we have moved to initiate a clinical study using intra-tumoral administration of PH-762 in patients with advanced melanoma, which is expected to begin in the fourth quarter of 2021. The clinical trial will be conducted at the Gustave Roussy Institute, which is one of the leading anti-cancer centers in Europe, with Dr. Caroline Robert as our lead principal investigator. This is an exciting next step in the development of innovative therapeutics based on our INTASYL platform and we look forward to keeping you posted as we move closer towards initiation of the study," concluded Dr. Dispersyn.

Quarter in Review and Recent Corporate Updates

Presented positive data from a study assessing the potential of PH-762, a PD-1 targeting INTASYL compound, to enhance the therapeutic efficacy of HER2-targeted CAR-T cells (HER2CART) in solid tumors, namely a subcutaneous HER2-expressing SKOV3 model of human ovarian cancer in mice. Compared to untreated HER2CART cells, HER2CART cells treated with PH-762 showed a statistically significant and durable inhibition of in vivo tumor growth. These data were included in a poster presentation during the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper).
Announced new in vivo data showing intratumoral (IT) treatment with PD-1 targeting INTASYL (mPH-762) inhibits tumor growth in a dose dependent fashion in both PD-1 responsive and refractory models. These data were included in a poster presentation during the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021.
Further bolstered the balance sheet to a total of $32.7 million in cash, following the completion of two financings in Q1 2021 for gross proceeds of $21.7 million in additional capital.
Completed a $7.7 million registered direct offering of common stock priced at-the-market.
Completed a $14.0 million private placement of common stock and warrants priced at-the-market.
Upcoming Pipeline Milestones for 2021

Scheduled to present new in-vivo data regarding dual targeting INTASYL drug therapy (BRD4 and PD-1) at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
Initiate a first-in-human clinical study on the use of PH-762 in adoptive cell therapy with tumor infiltrating lymphocytes in patients with metastatic melanoma or squamous cell carcinoma of the head and neck (SSCHN).
Initiate a first-in-human clinical study on the use of PH-762 direct drug therapy (intratumoral injection) in patients with advanced melanoma.
Additional data publications on the Company’s pipeline programs.
Financial Results

Cash Position

At March 31, 2021, the Company had cash of $32.7 million as compared with $14.2 million at December 31, 2020. The Company expects its current cash will be sufficient to fund currently planned operations to the second quarter of 2023.

Research and Development Expenses

Research and development expenses were approximately $2.6 million for the quarter ended March 31, 2021, compared to approximately $1.2 million for the quarter ended March 31, 2020. The increase is primarily due to manufacturing costs and fees for the required preclinical studies in support of the Company’s planned clinical trials for PH-762 as compared to the same period in the prior year.

General and Administrative Expenses

General and administrative expenses were approximately $1.0 million for the quarter ended March 31, 2021, compared to approximately $1.1 million for the quarter ended March 31, 2020. The decrease is primarily due to a decrease in legal fees as compared to the same period in the prior year.

Net Loss

Net loss was $3.4 million, or $0.32 per share, for the quarter ended March 31, 2021, compared with $2.4 million, or $1.33 per share, for the quarter ended March 31, 2020. The increase in net loss was primarily attributable to the increase in research and development expenses, as described above. The change in net loss per share was primarily due to an increase in the number of shares outstanding as a result of the Company’s capital raise activities as compared to the prior year period.

On May 13, 2021 -Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the first quarter ended March 31, 2021 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAY 13, 2021, View Source [SID1234579955]).

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Company Updates and Upcoming Milestones

Apitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).

TOPAZ 12-Month Data Show Transformative Potential in SMA of Apitegromab as Add-on to SMN Upregulator Therapy. In April 2021, positive top-line data were announced from the TOPAZ Phase 2 trial (NCT03921528) in patients with Type 2 and Type 3 SMA. Both younger and older non-ambulatory patients treated with apitegromab and background SMN upregulator therapy over the 12-month period attained meaningful motor function improvements with 74% (23/31) of patients demonstrating a clinical improvement of ≥1-point increase in Hammersmith Functional Motor Scale Expanded (HFMSE).

In the younger non-ambulatory TOPAZ cohort (ages 2-6 years), treatment with apitegromab led to a mean increase from baseline in HFMSE of +6.2 points across both evaluated doses; a mean increase of +7.1 points for the 20 mg/kg dose and +5.3 points with the 2 mg/kg dose.

Increase in HFMSE from baseline across both apitegromab doses:

− 82% (14/17) of patients attained a >1-point increase
− 59% (10/17) of patients attained a >3-point increase
− 59% (10/17) of patients attained a >5-point increase
− 35% (6/17) of patients attained a >10-point increase

In the older non-ambulatory TOPAZ cohort (age 8-19 years), treatment with apitegromab 20 mg/kg led to a mean increase from baseline in HFMSE of +0.6 points by the primary intent-to-treat analysis and +1.2 points by the prespecified per protocol analysis.

Increase in HFMSE from baseline for this patient population:

− 64% (9/14) of patients attained a >1-point increase
− 29% (4/14) of patients attained a >3-point increase
Patients enrolled in TOPAZ were receiving chronic maintenance doses of nusinersen; both the younger and older non-ambulatory cohorts had received more than five mean maintenance doses of nusinersen (approximately two years of treatment) at baseline. Clinical data from the CHERISH and SHINE studies of nusinersen offer background insights into this patient population.1 These studies observed that nusinersen-treated patients primarily experience stabilization or only slight increases in HFMSE scores beyond the initial 15-month treatment period.2 In addition, even in the initial 15-month treatment period, older patients (age 5-12) on average experience declines in HFMSE and rarely attain a >3-point increase in HFMSE in a 12-month timeframe.3

The five most frequently reported treatment-emergent adverse events included headache, pyrexia, upper respiratory tract infection, cough, and nasopharyngitis​. All 57 patients who completed the 12-month treatment period elected to opt into the extension period.

Phase 3 Registrational Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 Patients Anticipated to Initiate by Year-End 2021. TOPAZ Phase 2 top-line efficacy and safety results support the continued evaluation of apitegromab in a Phase 3 trial. Subject to feedback from regulatory agencies, Scholar Rock intends to conduct a Phase 3 trial to evaluate apitegromab with background SMN upregulator therapy in non-ambulatory Type 2 and Type 3 SMA, the patient population where apitegromab demonstrated the largest increases in motor function (HFMSE scores) in TOPAZ. Patients with non-ambulatory Type 2 and Type 3 SMA represent approximately two-thirds of the overall prevalent SMA patient population.
Additional Data and Analyses from the TOPAZ Trial to be Presented at Upcoming Medical Congresses. Scholar Rock is conducting additional data analyses, including exploratory analyses using patient-level data, evaluating additional outcome measures, and reviewing additional safety data from the TOPAZ Phase 2 trial. The Company plans to present the TOPAZ top-line results as well as findings from these analyses at upcoming medical congresses.
Apitegromab Granted Priority Medicines (PRIME) Designation by the European Medicines Agency (EMA). In March 2021, the EMA granted PRIME designation to apitegromab, recognizing its potential to address unmet medical needs of patients with SMA. With the PRIME designation, Scholar Rock has greater access and enhanced dialogue with the regulatory agency to optimize development plans and evaluations.
Two U.S. Patents Issued Adding Further Protection for Inhibitors of Myostatin Activation and Related Methods, Including Apitegromab. In March 2021, the United States Patent Office (USPTO) issued U.S. Patent No. 10,946,036 with an expiry of June 2037, protecting both add-on and combination therapy with a myostatin inhibitor and a neuronal corrector therapy (such as SMN upregulator therapy) for the treatment of SMA. In April 2021, the USPTO issued U.S. Patent No. 10,981,981 with an expiry of May 2034, broadly covering methods for making inhibitors of myostatin (GDF8) activation based on Scholar Rock’s proprietary platform approach of targeting the precursor forms of growth factors.
Additional Potential Indications Identified for Apitegromab. Scholar Rock has identified multiple diseases for which the selective inhibition of the activation of myostatin may offer therapeutic benefit, including additional patient populations in SMA (such as Type 1 SMA and ambulatory Type 3 SMA) and potential indications outside of SMA, such as Becker Muscular Dystrophy (BMD).
SRK-181 is a selective inhibitor of latent TGFβ1 activation being developed with the aim of overcoming resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.

Published Preclinical Development Data for SRK-181 in International Journal of Toxicology. In March 2021, Scholar Rock announced the publication of "Nonclinical Development of SRK-181: An Anti-latent TGFβ1 Monoclonal Antibody for the Treatment of Locally Advanced or Metastatic Solid Tumors," in the peer-reviewed International Journal of Toxicology. This publication provided a comprehensive preclinical assessment of the pharmacology, pharmacokinetics, and safety of SRK-181, which provided support for the dose selection strategy for the ongoing DRAGON Phase 1 trial.
Initial Clinical Response and Safety Data from Part A of the DRAGON Phase 1 Trial Anticipated by Year-End 2021. SRK-181 is being evaluated in the two-part DRAGON trial (NCT04291079) in patients with locally advanced or metastatic solid tumors exhibiting primary resistance to anti-PD-(L)1 therapy. Dose escalation continues to progress in Part A of the trial, which evaluates the safety and pharmacokinetics of SRK-181, and the Company expects to advance to the Part B dose expansion portion of the trial in mid-2021. Part B will consist of multiple cohorts, including urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, and other solid tumors. Each cohort will enroll up to 40 patients who have demonstrated primary resistance to anti-PD-(L)1 therapy and will be treated with SRK-181 in combination with an approved anti-PD-(L)1 therapy.
"2021 is off to a great start as we are delighted with the positive 12-month top-line data from our TOPAZ study, which shows the transformative potential of apitegromab in SMA," said Tony Kingsley, President and CEO of Scholar Rock. "Based on these exciting results and the potential to bring apitegromab to the SMA community, we will be working with urgency on behalf of patients to initiate our Phase 3 registrational trial in patients with non-ambulatory Type 2 and 3 SMA by the end of the year."

First Quarter 2021 Financial Results

For the quarter ended March 31, 2021, net loss was $27.7 million or $0.76 per share compared to a net loss of $17.1 million or $0.58 per share for the quarter ended March 31, 2020.

Revenue was $4.7 million for the quarter ended March 31, 2021 compared to $5.0 million for the quarter ended March 31, 2020 and was related to the Gilead fibrosis-focused collaboration (the "Gilead Collaboration Agreement") that was executed in December 2018.
Research and development expense was $22.5 million for the quarter ended March 31, 2021 compared to $16.9 million for the quarter ended March 31, 2020. The increase year-over-year primarily reflects manufacturing costs associated with apitegromab and higher personnel and facility-related costs, partially offset by lower manufacturing costs associated with SRK-181.
General and administrative expense was $9.4 million for the quarter ended March 31, 2021 compared to $5.8 million for the quarter ended March 31, 2020. The increase year-over-year was primarily attributed to higher personnel and facility-related costs.
"We are focused on advancing our portfolio of clinical and preclinical programs with several near-term milestones, including the initiation of a Phase 3 trial evaluating apitegromab in SMA, completion of dose escalation in Part A and initiation of the Part B dose expansion portion of the DRAGON Phase 1 trial of SRK-181 in patients with solid tumors," said Ted Myles, CFO and Head of Business Operations of Scholar Rock. "We are well positioned to continue to execute on our plans as we ended the first quarter with approximately $315 million in cash, which can fund operations into 2023."

1This information from third-party studies is provided for background purposes only and is not intended to convey or imply a comparison to the TOPAZ clinical trial results.
2 Source: "Longer-term treatment with nusinersen: results in later-onset spinal muscular atrophy from the SHINE study" P.257, World Muscle Society Congress 2020
3 Source: Mercuri E, et.al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018;378:625-635.

On May 13, 2021 TCR2 Therapeutics Inc. (Nasdaq: TCRR), a clinical-stage cell therapy company with a pipeline of novel T cell therapies for patients suffering from cancer, reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, TCR2 Therapeutics, MAY 13, 2021, View Source [SID1234580026]).

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"We remain on track to identify the recommended Phase 2 dose for gavo-cel in 2021 and advance into the expansion portion of the Phase 1/2 trial where our primary focus will be efficacy as well as continuing to monitor for safety. The clinical benefit we have observed with our cancer patients gave us confidence to expand our manufacturing capabilities by establishing a commercial-scale cGMP facility in Rockville, Maryland, which we expect to come online in 2023," said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics. "In the last quarter we were also able to showcase our early-stage programs in our rich pipeline and reflect on the versatility of our TRuC-T cell platform. This included presenting our CD70-targeted autologous TRuC-T cell and our mesothelin-targeted allogeneic TRuC-T cell at AACR (Free AACR Whitepaper). As for our additional solid tumor milestones in 2021, we expect to present more complete data from the dose escalation Phase 1 portion of the gavo-cel trial, file an IND for TC-510, a mesothelin-targeted TRuC-T cell enhanced with a PD-1:CD28 switch receptor, and present preclinical data on our IL-15 enhancement, which we believe will drive even longer persistence and expression of a memory phenotype, characteristics which could be beneficial across our portfolio."

Recent Developments

gavo-cel:

TCR2 presented clinical and translational data from the dose escalation portion of the Company’s Phase 1/2 clinical trial of gavo-cel in patients with treatment refractory mesothelin-expressing solid tumors at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting. The data reported were from eight treatment-refractory mesothelin-expressing solid tumor patients and highlighted translational markers consistent with the clinical profile, including transduction efficiency, CD4:CD8 ratio, memory phenotype, expansion and persistence and cytokines.
Early-Stage Pipeline

TCR2 presented new preclinical data from its allogeneic "off the shelf" TRuC-T cell targeting mesothelin highlighting a lack of alloreactivity, reduced risk of host rejection, upregulation of activation markers, secretion of cytokines and clearance of tumors comparable to gavo-cel at the AACR (Free AACR Whitepaper) Virtual Annual Meeting.
TCR2 presented new preclinical data from its TRuC-T cell targeting CD70 highlighting T cell expansion, improved memory phenotype, significant anti-tumor efficacy in multiple xenograft mouse models with no evidence of in vivo fratricide at the AACR (Free AACR Whitepaper) Virtual Annual Meeting.
Corporate:

TCR2 announced that it signed a long-term, full-building lease with Alexandria Real Estate Equities, Inc. (NYSE: ARE) for an existing 85,000 square foot cell therapy manufacturing facility in Rockville, Maryland which is ready for Current Good Manufacturing Practice (cGMP) build-out. The site will support clinical and commercial production of gavo-cel with a capacity that is projected to treat several thousand cancer patients annually. The facility is expected to accelerate the Company’s commercial-scale manufacturing timelines with production anticipated in 2023.
TCR2 announced the appointment of Aaron Vernon as Vice President of Technical Operations. Previously, Mr. Vernon held senior positions including Vice President of Global Technical Operations and Vice President of Engineering & Supply Chain at Autolus Therapeutics, where he led manufacturing capacity expansion and overseeing internal and external manufacturing of plasmid, vector and cell therapy products.
Anticipated Milestones

TCR2 to present additional safety, efficacy and translational data from the Phase 1 portion of the gavo-cel Phase 1/2 clinical trial for patients with mesothelin-expressing solid tumors throughout 2021.
TCR2 to present an interim update from the Phase 1 portion of the TC-110 Phase 1/2 clinical trial for patients with CD19+ non-Hodgkin lymphoma or adult acute lymphoblastic leukemia in the second half of 2021.
TCR2 plans to file an IND for TC-510, the first enhanced TRuC-T cell targeting mesothelin with a PD-1:CD28 switch, in the second half of 2021.
TCR2 plans to select a development candidate for its allogeneic program in the second half of 2021.
TCR2 to present preclinical data on its IL-15 enhancements program in the second half of 2021.
TCR2 anticipates production of clinical trial material from ElevateBio LLC and its manufacturing facility in Stevenage, UK, both in anticipation of demand from the Phase 2 expansion trial of gavo-cel, in 2022.
Financial Highlights

Cash Position: TCR2 ended the first quarter of 2021 with $333.3 million in cash, cash equivalents, and investments compared to $228.0 million as of December 31, 2020. Net cash used in operations was $19.9 million for the first quarter of 2021 compared to $16.4 million for the first quarter of 2020. TCR2 projects net cash use of $100-110 million for 2021, which includes tenant improvements to the Rockville facility. We expect cash on hand to support operations through 2023.

R&D Expenses: Research and development expenses were $15.9 million for the first quarter of 2021 compared to $12.0 million for the first quarter of 2020. The increase in R&D expenses is primarily related to ongoing clinical trials, increase in headcount and additional lab facilities.

G&A Expenses: General and administrative expenses were $5.7 million for the first quarter of 2021 compared to $4.3 million for the first quarter of 2020. The increase in general and administrative expenses was primarily due to an increase in personnel costs.

Net Loss: Net loss was $21.5 million for the first quarter of 2021 compared to $15.5 million for the first quarter of 2020, driven predominantly by increased personnel expenses.
Rockville Facility

In March 2021, we entered into a lease for a new manufacturing facility. The landlord built an approximately 85,000 square foot rental building in Rockville, Maryland and leased the facility to the Company as a manufacturing facility for an initial term of 15 years through June 2036.

Because we are involved in the construction project and are responsible for paying a significant portion of the costs of normal finish work and structural elements of the facility, the Company is deemed for accounting purposes to be the owner of the building during the construction period under build to suit lease accounting guidance under ASC 840, Leases. Therefore, the Company recorded a construction-in-progress asset and a related construction financing obligation on our consolidated balance sheets as a component of property and equipment and lease financing obligations, respectively. We anticipate lease payments of $1.4 million for the remainder of 2021.

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 EpiVax Therapeutics, Inc. ("EVT") reported the publication "Multi‑step screening of neoantigens’ HLA and TCR‑interfaces improves prediction of survival" in Scientific Reports (Press release, EpiVax, MAY 13, 2021, View Source [SID1234579907]). This study demonstrates a superior method of survival analysis for cancer patients using EVT’s Ancer platform. EVT spun-off from EpiVax, Inc. in 2017 to apply EpiVax tools to clinical research.

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Built on validated in silico tools EpiMatrix and JanusMatrix, Ancer analyzes individual cancer mutanomes to define the presence of T cell epitopes likely to be immunogenic. The concept behind Ancer is that the presence of a mutation alone is not sufficient to generate a protective immune response, but that the differences present between the natural (human genome) sequence and the mutated sequence at the face of the epitope that is identifiable to T cells (the TCR-face) should be accounted for when creating precision cancer vaccines and predicting survival outcomes for cancer patients. This is because some mutations create self-like epitopes and can be tolerated by the immune system.

Researchers compared three approaches to analyzing cancer mutanomes and evaluated their ability to predict patient outcomes. Ancer, the best performing method, improved the differentiation of patients that did poorly from those who did well, accurately predicting survival outcome nearly 6 years longer as compared to other approaches, which only predicted as far as 3 years. This study has important consequences in our ability to identify patients with a high risk of tumor progression and early mortality based on their tumor genome.

On study implications, Dr. Randy Sweis of the University of Chicago stated, "These results suggest that defining the number of true neoepitopes using Ancer may represent a novel prognostic or predictive biomarker for cancer patients." Dr. Annie De Groot, EVT co-founder, suggested "In addition to biomarker identification, using Ancer novel precision vaccines for individual cancer patients offers major advantages compared to other approaches, since Ancer prioritizes truly immunogenic CD8 and CD4 neoepitopes, while removing self-like or inhibitory neoepitopes."