On August 14, 2020 Myriad Genetics, Inc. (NASDAQ: MYGN, "Myriad" or the "Company"), a global leader in molecular diagnostics and precision medicine, reported that in connection with the previously announced appointment of Paul J. Diaz as President and Chief Executive Officer of the Company, effective yesterday, August 13, 2020, the Company’s Board of Directors authorized the grant to Mr. Diaz of (i) a restricted stock unit award for 298,954 shares of the Company’s common stock, effective as of the first day of his employment (the "RSUs"); (ii) a performance stock unit award for 298,954 shares of the Company’s common stock, to be effective within a reasonable period of time following his first day of employment (the "PSUs"); (iii) a time-based non-qualified stock option for the purchase of 342,040 shares of the Company’s common stock, effective as of the first day of his employment (the "Time-Based Options"); and (iv) a performance-based non-qualified stock option for the purchase of 339,088 shares of the Company’s common stock, effective as of the first day of his employment (the "Performance-Based Options" and together with the RSUs, PSUs and Time-Based Options, the "Inducement Awards") (Press release, Myriad Genetics, AUG 14, 2020, View Source [SID1234563655]). The Inducement Awards are inducements material to Mr. Diaz’s entering into employment with the Company in accordance with Nasdaq listing Rule 5635(c)(4).

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The Time-Based Options and Performance-Based Options have an exercise price of $13.38 per share, the fair market value of the Company’s common stock on the date of grant. The Time-Based Options will vest as to 25% of the shares on each of the first four anniversaries of Mr. Diaz’s employment. The shares subject to the Performance-Based Options become eligible to vest upon achievement of certain stock price targets and shall vest as to one-fifth (1/5) of the shares subject to the Performance-Based Options upon achievement of each of five stock price targets, provided that no portion of the Performance-Based Options may vest earlier than the first anniversary of Mr. Diaz’s employment. The Time-Based Options and Performance-Based Options each have seven-year terms and are subject to the terms and conditions of their respective stock option agreements.

The RSUs will vest as to 50% of the shares on the first anniversary of Mr. Diaz’s employment with the remainder vesting in equal instalments on each of the second, third and fourth anniversaries of Mr. Diaz’s employment. The shares subject to the PSUs become eligible to vest upon achievement of certain performance targets for the fiscal year ending June 30, 2021. To the extent that the performance targets have been met, the PSUs will vest as to 25% of the shares on the date the performance targets are determined to have been met, with the remainder vesting in equal instalments on each of the second, third and fourth anniversaries of Mr. Diaz’s employment. The RSUs and PSUs are subject to the terms and conditions of their respective restricted stock unit agreements.

On August 14, 2020 Seneca Biopharma, Inc. (Nasdaq: SNCA), a biopharmaceutical company focused on developing novel treatments for diseases of high unmet medical need, reported its financial results for the quarter ended June 30, 2020 (Press release, Neuralstem, AUG 14, 2020, View Source [SID1234563654]).

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Business Highlights for 2020 to date.

During the First Half of 2020, the Company achieved the following business milestones:

Completed offerings resulting in net proceeds of over $14.8 million.
Continued progress on both the Company’s out-licensing effort to partner NSI-566 and NSI-189 programs and initiative to in-license or acquire novel therapeutics.
Appointed of Matthew W. Kalnik, Ph.D. as President and Chief Operating Officer and Dane R. Saglio as Chief Financial Officer.
Affirmed guidance that data readout from the Company’s non-GCP Phase II trial evaluating NSI-566, for the treatment of chronic ischemic stroke, is expected during the second half of 2020.
Announced that as a result of feedback received from the FDA, Seneca believes that the existing Phase 1 and 2 trial results support moving into a Phase 3 clinical study for ALS.
Completion of the Company’s stem cell manufacturing facility in Suzhou, China which will be used to manufacture NSI-566 for clinical trials within China.
Financial Results for the Quarter Ended June 30, 2020

Cash Position and Liquidity: At June 30, 2020, cash was approximately $15.8 million as compared to approximately $10 million at March 31, 2020. The increase in cash is attributed to the May 2020 warrant exercises and registered direct offering.

Operating Loss: Operating loss for the quarter ended June 30, 2020 was $1.9 million compared to a loss of $1.9 million for the comparable 2019 period. For the six-month period ended June 30, 2020, the operating loss was $3.9 million versus $4.4 million for the six months ended June 30, 2019. The decrease in operating loss for 2020 was primarily due to a decrease in R&D expenses as we continue to wind down the clinical programs. This decrease was partially offset by an increase in G&A expenses which reflects an enhanced management structure to support corporate objectives as compared to the same period of 2019.

Net Loss: Net loss for the quarter ended June 30, 2020 was $2.0 million, or $0.15 per share, compared to a loss of $1.4 million, or $1.45 per share on a post-reverse stock-split basis, for the same period in 2019. For the 2020 six-month period the net loss was $9.5 million, or $0.92 per share versus a net loss of $4.6 million, or $4.78 per share for the same period in 2019. The 2020 increase in net loss was primarily attributed to a non-cash expense of $5.6 million related to the January 2020 warrant inducement transaction.

On August 14, 2020 CureVac B.V. ("CureVac" or the "Company"), a clinical-stage biopharmaceutical company developing a new class of transformative medicines based on messenger ribonucleic acid ("mRNA"), reported the pricing of its initial public offering of 13,333,333 common shares at an initial public offering price of $16.00 per common share, for total gross proceeds of approximately $213.3 million (Press release, CureVac, AUG 14, 2020, View Source [SID1234563653]). In addition, the Company has granted the underwriters a 30-day option to purchase up to an additional 1,999,999 common shares at the public offering price, less underwriting discounts and commissions. All of the common shares are being offered by CureVac. The shares are scheduled to begin trading on the Nasdaq Global Market today, August 14, 2020, under the ticker symbol "CVAC." The offering is expected to close on August 18, 2020, subject to customary closing conditions.

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BofA Securities, Jefferies and Credit Suisse are acting as joint book-running managers for the proposed offering, with Berenberg and Kempen & Co acting as passive book-running managers.

A registration statement relating to these securities was declared effective by the Securities and Exchange Commission ("SEC") on August 13, 2020. The offering is being made only by means of a prospectus. Copies of the final prospectus relating to the offering may be obtained, when available, for free by visiting EDGAR on the SEC website at www.sec.gov. Alternatively, copies of the final prospectus, when available, may be obtained from BofA Securities, Attention: Prospectus Department, NC1-004-03-43, 200 North College Street, 3rd Floor, Charlotte, NC 28255-0001, by telephone at (800) 299-1322 or by email at [email protected]; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at (877) 821-7388 or by email at [email protected]; and Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, 6933 Louis Stephens Drive, Morrisville, North Carolina 27560, by telephone at (800) 221-1037 or by email at [email protected].

On August 14, 2020 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported financial results for the three and six months ended June 30, 2020, and provided an update on clinical development programs with GEN-1, its DNA-mediated IL-12 immunotherapy currently in Phase II development for the treatment of advanced stage ovarian cancer, and ThermoDox, its proprietary heat-activated liposomal encapsulation of doxorubicin currently in Phase III development for the treatment of hepatocellular carcinoma, or primary liver cancer (Press release, Celsion, AUG 14, 2020, View Source [SID1234563640]).

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"GEN-1, our oncology-focused immunotherapy, continues to show encouraging results at the 100 mg/m² dose cohort in the OVATION 2 Study, which is consistent with the results reported from our earlier Phase Ib trial (the OVATION 1 Study) in advanced-stage ovarian cancer. In June 2020, the Data Safety Monitoring Board (DSMB) for the OVATION 2 Study recommended that the Phase II portion of the OVATION Study proceed with the dose of 100 mg/m2," reported Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "These findings were reinforced by strong progression-free survival (PFS) when comparing study patients to a statistically validated synthetic control arm (SCA) of matched patients from prior studies. In July 2020, we announced the randomization of the first two patients in the Phase II OVATION 2 Study. This milestone was achieved approximately five months ahead of our previously announced schedule. We have a very aggressive recruitment program and anticipate completing enrollment of 105 patients in the second quarter of 2021. Importantly, as an open-label study, clinical updates will be provided throughout the course of treatment including response rates and surgical resection scores," Mr. Tardugno added.

Continuing his comments, Mr. Tardugno noted, "In early July, Celsion received a wholly unexpected recommendation from the independent Data Monitoring Committee (DMC) to consider stopping the global Phase III OPTIMA Study. This recommendation was made following the DMC’s second pre-planned interim safety and efficacy analysis of the OPTIMA Study on July 9, 2020. The DMC’s analysis found that the pre-specified boundary for stopping the trial for futility of 0.900 was crossed with an actual value of 0.903. However, the p-value of 0.524 for this analysis provides a high level of uncertainty as to the actual hazard ratio value, therefore, the DMC left the final decision of whether to stop the OPTIMA Study to the Company.

Mr. Tardugno further stated, "This development had never been anticipated by the Company or our advisors, nor would it have been forecasted by the first pre-planned efficacy analysis. Further, blinded data available to the Company appeared to be tracking well against the sub-group analysis of the Company’s earlier HEAT Study, upon which the OPTIMA Study is based."

In early August, after conducting additional analyses of the unblinded data from the second pre-planned interim analysis, the Company announced plans to continue following patients for overall survival (OS), noting that the unexpected and marginally crossed futility boundary, suggested by the Kaplan-Meier analysis at the second interim analysis, may be associated with a data maturity issue. Additionally, Celsion reported that it is sending all clinical trial data, including Chemistry, Manufacturing and Controls (CMC) data, to the National Institutes of Health (NIH) for independent analysis, including computed tomography (CT) scans for NIH’s evaluation of PFS. Depending on the trends noted during the OS follow-up period, Celsion may choose to discontinue the Study at any time. The Company also notes that the vast majority of expenses related to the OPTIMA Study already have been incurred.

Recent Developments

GEN-1 Immunotherapy

Initiation of Phase II OVATION 2 Study in Advanced Ovarian Cancer. In July 2020, the Company announced the randomization of the first two patients in the Phase II portion of the OVATION 2 Study with GEN-1 in advanced ovarian cancer. The Company anticipates completing enrollment of up to 118 patients in the second quarter of 2021. Because this is an open-label study, clinical updates will be provided throughout the course of treatment including response rates and surgical resection scores.

The OVATION 2 Study combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve OS. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in PFS (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).

DSMB Recommends GEN-1 to Proceed to Phase II of the OVATION 2 Study in Advanced Ovarian Cancer. In May 2020, the Company announced the final recommendations of the DSMB following completion of the Phase I dose-finding and tolerance portion of the OVATION 2 Study with GEN-1 in advanced (Stage III/IV) ovarian cancer. Based on favorable safety data from 15 randomized patients, the DSMB recommended that the Phase II portion of the OVATION Study proceed with the dose of 100 mg/m2. The DSMB also determined that safety is satisfactory with an acceptable risk/benefit, and that patients tolerate up to 17 doses of GEN-1 during a course of treatment that lasts up to six months. No dose limiting toxicities were reported.

In March 2020, the Company announced the following clinical development achievements for GEN-1:

●Highly encouraging initial clinical data from the first 15 patients enrolled in the ongoing Phase I/II OVATION 2 Study for patients newly diagnosed with Stage III and IV ovarian cancer. GEN-1 plus standard NACT produced positive dose-dependent efficacy results, with no dose-limiting toxicities, which correlates well with successful surgical outcomes as summarized below:
oOf the 15 patients treated in the Phase I portion of the OVATION 2 Study, nine were treated with GEN-1 at a dose of 100 mg/m² plus NACT and six were treated with NACT only. All 15 had successful resections of their tumors, with seven out of nine patients (78%) in the GEN-1 treatment arm having an R0 resection, which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed. Only three out of six patients (50%) in the NACT only treatment arm had an R0 resection
oWhen combining these results with the surgical resection rates observed in the Company’s prior Phase Ib dose-escalation trial (the OVATION 1 Study), a population of patients with inclusion criteria identical to the OVATION 2 Study, the data reflect the strong dose-dependent efficacy of adding GEN-1 to the current standard of care NACT:

Medidata-matched patient data from a SCA compared with results from the Phase Ib dose-escalating OVATION 1 Study with GEN-1 in Stage III/IV ovarian cancer patients showed positive results in PFS. The HR was 0.53 in the intent-to-treat group, showing strong signals of efficacy. Medidata is a globally recognized leader in clinical data management. GEN-1’s strong and encouraging treatment effect, evidenced by the SCA, suggests a potentially remarkable improvement in PFS, an FDA-recognized surrogate for OS, and appears to confirm the science behind IL-12’s ability to recruit the innate and adaptive elements of the immune system to fight malignancies. The strong PFS trend is supported by previously published translational data that clearly demonstrate the pro-immune changes in the tumor microenvironment associated with loco-regional GEN-1 therapy. PFS data generated from this analysis comparing GEN-1 with SCA showed the following:

● The European Medicines Agency (EMA) Committee for Orphan Medicinal Products recommended that GEN-1 be designated as an orphan medicinal product for the treatment of ovarian cancer. As established by the EMA, this designation provides for scientific advice and certain regulatory assistance during the product development phase, direct access to centralized marketing authorization and certain financial incentives for companies developing new therapies intended for the treatment of a life-threatening or chronically debilitating condition that affects no more than five in 10,000 people in the European Union. GEN-1 previously received orphan drug designation from the FDA.
ThermoDox

Patients in Phase III OPTIMA Study Will Continue to be Followed for Overall Survival. In August 2020, the Company provided an update on its ongoing review of unblinded data from the second pre-planned interim analysis of the global Phase III OPTIMA Study. The Company announced it will continue following patients for OS, noting that the unexpected and marginally crossed futility boundary suggested by the Kaplan-Meier analysis at the second interim analysis on July 9, 2020 may be associated with a data maturity issue. The Company further notes that 26 consecutive patient deaths represented exclusively in the second analysis behave far differently from the balance of the patients who have died as of that date. Removing the 26 consecutive patient deaths, which occurred between September 2019 and March 2020, from the pre-planned interim analysis suggests that the OPTIMA Study OS pattern is similar to the prospective HEAT Study subgroup upon which the OPTIMA Study is based, at the approximate comparable point in time. In addition, subsequent to the second interim analysis there were eight patient deaths in a 3:1 ratio of control arm to treatment arm patients, which further supports a concern for data maturity.

It was further noted that OPTIMA Study sites in China and Vietnam, which enrolled over 37% of the subjects, joined the Study approximately 12 and 18 months, respectively, after the trial was initiated. The Kaplan-Meier curves for both geographies demonstrate a potential data maturity issue when compared with the behavior of the HEAT Study subgroup and other OPTIMA Study testing site regions. The China sites, in particular, show a negative Kaplan-Meier curve, yet with a 56% improvement in the treatment arm in the median time to death. The Vietnam sites show a marginal Kaplan-Meier benefit, yet with a 45% improvement in the treatment arm in the median time to death. The Company believes that this dichotomy must be reconciled, most probably with longer follow up, before it can determine the Study’s direction.

Recommendation from the Independent DMC to Consider Stopping the Phase III OPTIMA Study of ThermoDox in Primary Liver Cancer. In July 2020, the Company announced that it received a recommendation from the independent DMC to consider stopping the global Phase III OPTIMA Study. The recommendation was made following the second pre-planned interim safety and efficacy analysis by the DMC on July 9, 2020. The DMC analysis found that the pre-specified boundary for stopping the trial for futility of 0.900 was crossed with an actual value of 0.903. However, the p-value of 0.524 for this analysis provides uncertainty; subsequently, the DMC left the final decision of whether or not to stop the OPTIMA Study to Celsion. There were no safety concerns noted during the interim analysis.

The statistical plan for the OPTIMA Study included two interim efficacy analyses by the DMC. The first interim analysis was announced in November 2019 following data lock in August 2019 after the prescribed minimum number of 128 patient events (deaths) was reached, and the second interim analysis was conducted on July 9, 2020 following data lock in April 2020 after the prescribed minimum number of 158 events was reached.

Corporate Developments

Strengthened Balance Sheet Through a $10 Million Underwritten Offering of Common Stock. In June 2020, the Company entered into an underwriting agreement relating to the sale of 2,666,667 shares of its common stock at an offering price of $3.75 per share. The net proceeds from the offering were $9.3 million, after deducting underwriting discounts and commissions, but before expenses payable by the Company. The shares of common stock were sold to both existing and new institutional investors of the Company. Oppenheimer & Co. Inc. acted as the sole underwriter for the offering.

Received $1.8 Million in Non-Dilutive Funding from the Sale of New Jersey State Net Operating Losses. In April 2020, the Company announced it received $1.8 million of net cash proceeds from the sale of approximately $1.9 million of its unused New Jersey net operating losses (NOLs). The NOL sales cover the tax years 2017 and 2018 and are administered through the New Jersey Economic Development Authority’s (NJEDA) Technology Business Tax Certificate Transfer (NOL) Program. An additional sale of $2.0 million of unused New Jersey NOLs anticipated in the second half of 2020 will further increase Celsion’s cash reserves on a non-dilutive basis.

Second Quarter Financial Results

For the quarter ended June 30, 2020, Celsion reported a net loss of $5.3 million ($0.18 per share), compared with $5.9 million ($0.29 per share) in the same period of 2019. Operating expenses were $4.9 million in the second quarter of 2020, which represented a $0.8 million (14%) decrease from $5.7 million in the same period of 2019.

The Company ended the second quarter of 2020 with $25.5 million in cash, investment securities and accrued interest receivable. With $25.5 million in cash as of June 30, 2020 coupled with future sales of the Company’s New Jersey NOL’s, the Company believes it has sufficient capital resources to fund its operations into the fourth quarter of 2021.

Research and development expenses decreased $0.6 million to $3.0 million in the second quarter of 2020, compared with $3.6 million in the second quarter of 2019. Clinical development costs for the Phase III OPTIMA Study decreased $0.6 million to $0.6 million in the second quarter of 2020, compared with $1.2 million in the second quarter of 2019, due to the completion of enrollment in this 556-patient trial in August 2018. Costs associated with the OVATION 2 Study increased to $0.2 million in the second quarter of 2020 compared with $0.1 million in the same period of 2019. Other costs related to clinical supplies and regulatory support for the ThermoDox and GEN-1 clinical development programs increased to $2.3 million in the current quarter from $2.2 million in the second quarter of 2019 largely driven by higher manufacturing costs for GEN-1 clinical supplies for the Phase II portion of the OVATION 2 Study.

General and administrative expenses were $1.9 million in the second quarter of 2020, compared with $2.1 million in the same period of 2019. The 11% decrease was primarily attributable to lower professional fees incurred during the second quarter of 2020.

In connection with the Company’s venture debt facility with Horizon entered in late June 2018, the Company incurred interest expense of $0.3 million during the second quarter of 2020. This compares with interest expense of $0.4 million in the comparable prior-year period.

Six Month Financial Results

For the six months ended June 30, 2020, the Company reported a net loss of $10.4 million ($0.37 per share), compared with $8.3 million ($0.42 per share) in the same period of 2019. Operating expenses were $9.8 million during the first six months of 2020, which represented a $0.9 million (8%) decrease from $10.7 million in the same period of 2019.

Net cash used for operating activities was $7.9 million in the first six months of 2020, compared with $10.2 million in the same period in 2019. This was in line with the Company’s projected cash utilization for 2020 of approximately $15 million, or an average of approximately $3.75 million per quarter. Cash provided by financing activities was $18.6 million during the first six months of 2020 resulting from equity offerings in March 2020 and June 2020, and proceeds from the exercise of stock options.

Research and development expenses decreased $0.3 million to $6.0 million in the first half of 2020 from $6.3 million in the first half of 2019. Clinical development costs for the Phase III OPTIMA Study decreased by $0.8 million to 1.3 million in the first half of 2020, compared with $2.1 million in the first half of 2019, due to the completion of enrollment in this 556-patient trial in August 2018. Costs associated with the OVATION 2 Study increased to $0.5 million in the first half of 2020, compared with $0.2 million in the comparable six-month period in 2019. Other costs related to clinical supplies and regulatory support for the ThermoDox and GEN-1 clinical development programs increased by $0.4 million in the first half of 2020, compared with the same prior-year period due to higher manufacturing costs for GEN-1 clinical supplies for the Phase II portion of the OVATION 2 Study.

Other expenses during the first half of 2020 included a non-cash charge of $0.3 million for the change in valuation of the earn-out milestone liability for the GEN-1 ovarian product candidate, compared with a non-cash gain of $2.7 million, net of charge of $0.4 million for the 200,000 warrant issuance related to an amendment for the potential milestone payments for the GEN-1 ovarian product candidate during the comparable prior-year period. The Company realized $0.1 million of interest income during the first half of 2020 and $0.3 million in the comparable prior-year period. In connection with the Company’s venture debt facility with Horizon entered in late June 2018, the Company incurred interest expense of $0.7 million during the first six months of 2020 and 2019.

Second Quarter Conference Call

The Company will host a conference call to provide a business update and discuss its second quarter 2020 financial results at 11:00 a.m. EDT today. To participate in the call, interested parties may dial 1-800-353-6461 (Toll-Free/North America) or 1-334-323-0501 (International/Toll) 10 minutes before the call is scheduled to begin, and ask for the Celsion Corporation Second Quarter 2020 Earnings Call (Conference Code: 4777957). The call will also be broadcast live on the internet at www.celsion.com. The call will be archived for replay through August 28, 2020. The replay can be accessed at 1-719-457-0820 or 1-888-203-1112 using Conference ID: 4777957. An audio replay of the call will also be available on the Company’s website, www.celsion.com, for 90 days after 2:00 p.m. EDT Friday, August 14, 2020.

On August 14, 2020 BioXcel Therapeutics, Inc. ("BTI" or "Company") (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence to identify improved therapies in neuroscience and immuno-oncology, reported its quarterly results for the second quarter ended June 30, 2020 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, AUG 14, 2020, View Source [SID1234563639]).

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"Since inception, this is one of the most exciting times in the Company’s history," stated Vimal Mehta, Chief Executive Officer of BTI. "First and foremost, we reported positive results from our two pivotal SERENITY trials last month that showed a robust treatment effect in reducing acute agitation in schizophrenia and bipolar disorder patients. We look forward to submitting an NDA for BXCL501 to the FDA in the first quarter of 2021 and, in preparation for a potential approval, we have made two key hires to lead commercialization and medical affairs. At the same time, we are advancing the TRANQUILITY and RELEASE trials in dementia and opioid withdrawal patients, respectively, and plan to initiate a trial in agitation associated with delirium patients this year, highlighting this candidate’s expected versatility across a wide range of diagnoses. We also continue to explore alternative indications associated with agitation, including alcohol withdrawal, post-traumatic stress disorder, traumatic brain injury and phobias. Finally, the successful completion of our recent follow-on equity offering puts us in a strong cash position to support BXCL501’s commercial launch, if approved, and our aggressive indication expansion strategy."

Dr. Mehta continued, "In addition, our immuno-oncology program is advancing. The Phase 2 efficacy trial of BXCL701 and KEYTRUDA for treatment emergent Neuroendocrine Prostate Cancer continues to progress on track, and we have expanded this study to include a separate cohort of men with CRPC. Our MD Anderson-led Phase 2 basket trial in advanced solid tumors is also making great strides, now having met the efficacy bar in both arms necessary for the study to proceed to completion. We believe BXCL701 has the potential to bridge innate and adaptive immunity, as demonstrated by the increase in IL-18 from baseline, and we look forward to reporting initial efficacy data from both trials later this year."

Second Quarter 2020 and Recent Highlights

BXCL501-Neuroscience Program

BXCL501 is an investigational sublingual thin film of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist, designed for the treatment of acute agitation. The Company believes BXCL501 may directly target a causal agitation mechanism.

In July 2020, BTI reported that the pivotal Phase 3 SERENITY trials for the acute treatment of agitation in patients with schizophrenia and bipolar disorder met the primary, key secondary and exploratory endpoints. BXCL501 was well tolerated, with rapid and durable reductions in agitation. The Company has a pre-NDA meeting with the FDA this October and plans to submit an NDA for both indications in the first quarter of 2021.
The Company is initiating a third dose cohort, 90 mcg, in the TRANQUILITY study, a Phase 1b/2 trial of BXCL501 for the acute treatment of agitation associated with dementia. The adaptive trial is designed to identify the most effective and tolerable dose in this elderly patient population. BTI has successfully completed two lower-dose cohorts (30 mcg and 60 mcg) in a total of 30 patients. Based on findings from the 90 mcg cohort, the Company expects to report topline results in the fourth quarter of 2020, or, if needed, proceed to an additional dose cohort.
In June 2020, the first patient was enrolled in the RELEASE study, a Phase 1b/2 trial of BXCL501 for the acute treatment of opioid withdrawal symptoms, with the third cohort currently enrolling (90 mcg twice a day, 12 hours apart). The Company expects to report topline results from the study in the first quarter of 2021.
The Company expects to initiate a Phase 2 trial of BXCL501 in patients with agitation associated with delirium later this year. The planned study population will include ICU patients with or without COVID-19. This potential indication may offer synergy with the commercial infrastructure being developed to support our first NDA.
The Company received a Notice of Allowance from the U.S. Patent and Trademark office for patient application No. 16/453,679 related to BXCL501. The patent is expected to cover film formulations containing Dex and methods of treating agitation using such film formulations. The patent, which is anticipated to be issued in the third quarter of 2020, is expected to extend IP protection until 2039.
BXCL701-Immuno-Oncology Program

BXCL701 is an orally-delivered small molecule, innate immunity activator designed to inhibit dipeptidyl peptidase (DPP) 8/9 and block immune evasion by targeting Fibroblast Activation Protein (FAP). It has shown single agent activity in melanoma and safety has been evaluated in more than 700 healthy subjects and cancer patients.

The Phase 2 portion of the Phase 1b/2 trial of BXCL701 in combination with pembrolizumab (KEYTRUDA) for treatment emergent Neuroendocrine Prostate Cancer (tNEPC) is advancing on track. Recently, BTI expanded the trial to include a separate cohort for CRPC (adenocarcinoma) patients who have failed taxane-based chemotherapy and up to two lines of second generation androgen pathway blockers, based on preliminary evidence of activity in this patient population in the safety cohort. The CRPC cohort is expected to run concurrently with the tNEPC cohort. Initial efficacy data from this trial are expected to be reported later this year.
The open label Phase 2 basket trial evaluating the combination of BXCL701 and KEYTRUDA in patients with advanced solid cancers is proceeding, with early signs of clinical activity in several difficult-to-treat cancers. This study, which is being conducted at the MD Anderson Cancer Center, consists of two arms: checkpoint naïve patients and patients who are refractory to checkpoint therapy. In June, the safety portion of the trial was completed and in August, the efficacy bar was met for both arms of the trial, allowing the study to proceed to completion. Initial efficacy data are expected to be presented at a scientific conference later this year.
The BXCL701 phase of the triple combination study of BXCL701, bempegaldesleukin (NKTR-214, Nektar Therapeutics, Inc.) and BAVENCIO (avelumab, Merck KGaA, Darmstadt, Germany and Pfizer) in second line pancreatic cancer is expected to begin following Nektar and Pfizer’s Phase 1b safety trial of a double combination of bempegaldesleukin and avelumab, pending the outcome of that trial.
Corporate Highlights

In July 2020, the Company raised gross proceeds of approximately $200 million in connection with its common stock offering. BTI believes that the proceeds from this offering, together with current reserves, provide cash runway well into 2022 to fund key clinical, regulatory, operational, and commercial activities.
In June 2020, William Kane was appointed as Executive Vice President and Chief Commercial Officer of BTI. Mr. Kane brings over three decades of product commercialization experience in the pharmaceutical industry, with a proven track record in bringing neuropsychiatric drugs to market.
In June 2020, Reina Benabou, M.D., Ph.D. was appointed as Senior Vice President and Chief Development Officer of BTI. Dr. Benabou has over 20 years of experience in directing drug development programs and implementing medical affairs strategies for product commercialization in neurology and psychiatry.
COVID-19

During the second quarter of 2020, the Company remained committed to ensuring the health and safety of its patients, investigators and employees. BTI continued to assess the impact of the COVID-19 pandemic to best mitigate risk, while continuing business operations. Beginning at the end of the second quarter, BTI began to slowly bring a limited number of staff back to the Company’s office. This return to work is scheduled to be completed in September 2020. To date, the Company’s business and operations has only been minimally impacted and have not experienced any significant delays to our ongoing or planned clinical trials, except for challenges in accessing elderly care facilities; however, this could rapidly change.

Second Quarter 2020 Financial Results

BTI reported a net loss of $21.4 million for the second quarter of 2020, compared to a net loss of $8.5 million for the same period in 2019. The second quarter 2020 results include approximately $2.0 million in non-cash stock-based compensation, compared to $1.0 million for the same period in 2019.

Research and development expenses were $17.9 million for the second quarter of 2020, compared to $6.5 million for the same period in 2019. The increase was primarily due to increases in clinical trial activity.

General and administrative expenses were $3.5 million for the second quarter of 2020, as compared to $2.1 million for the same period in 2019. The increase was primarily due to salaries, non-cash compensation costs and professional fees.

Total operating expenses for the second quarter of 2020 were approximately $21.4 million, compared to total operating expenses of approximately $8.6 million for the same period in 2019.

As of June 30, 2020, cash and cash equivalents totaled approximately $65.5 million. This does not include the $187.5 million in net proceeds generated from our equity offering that closed on July 31, 2020.

Conference Call:

BTI will host a conference call and webcast today at 8:30 a.m. ET. To access the call, please dial 877-407-2985 (domestic) and 201-378-4915 (international). A live webcast of the call will be available on the Investors sections of the BTI website at www.bioxceltherapeutics.com. The replay will be available through at least August 28, 2020.