On March 11, 2021 BioXcel Therapeutics, Inc. ("BioXcel" or the "Company") (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, reported its quarterly results for the fourth quarter and full year ended December 31, 2020 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, MAR 11, 2021, View Source [SID1234576503]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Over the last 12 months, we have made significant progress with our leading neuroscience program, BXCL501, including reporting successful data readouts, submitting our first NDA, as well as advancing other potential indications," stated Vimal Mehta, Chief Executive Officer of BioXcel. "In addition to submitting our NDA for the acute treatment of schizophrenia and bipolar disorder related agitation, we also announced positive data from the TRANQUILITY Phase 1b/2 trial in dementia related agitation and will be discussing our plans for a pivotal Phase 3 program with the FDA in the second quarter of 2021. In parallel, we continue to evaluate BXCL501’s potential in a wide range of neuropsychiatric conditions with ongoing trials in opioid withdrawal symptoms and delirium related agitation. As we develop the commercial infrastructure needed to bring this potential candidate to market, we continue to execute on our clinical and corporate milestones, as well as leverage our unique AI platform to reach new patient populations where there are large unmet medical needs."

Dr. Mehta continued, "Moreover, our immuno-oncology candidate, BXCL701, has demonstrated encouraging signals of activity in difficult-to-treat tumors across both combination trials. We expect to report topline data from the ongoing trials in mid-2021, in hopes of further demonstrating this candidate’s potential at enhancing an individual’s innate immunity."

Fourth Quarter and Full Year 2020 and Recent Highlights

BXCL501-Neuroscience Program

BXCL501 is an investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist, designed for the treatment of agitation and opioid withdrawal symptoms. The Company believes BXCL501 may directly target a causal agitation mechanism.

BioXcel recently completed the rolling submission of its NDA to the FDA for BXCL501 for the acute treatment of schizophrenia and bipolar disorder related agitation. Leveraging the Company’s AI-based platform, BioXcel was able to develop this program from the first-in-human trial to NDA submission in just over 2 years.
The Company met the primary and secondary endpoints of the TRANQUILITY trial, a Phase 1b/2 trial of BXCL501 for the acute treatment of dementia related agitation. Topline results showed that BXCL501 was generally well tolerated, with statistically significant and clinically meaningful reductions in agitation achieved with both the 30 mcg and 60 mcg doses at 2 hours as measured by the PEC, PAS and Mod-CMAI scales. The end of Phase 2 meeting with the FDA is planned for Q2 2021, with the goal of initiating a registrational program in the H2 2021.
BioXcel has initiated a supplemental cohort investigating a 40 mcg dose of BXCL501 in 46 patients (1:1 randomization) as an expansion of TRANQUILITY to further inform its clinical development strategy.
The RELEASE study, a Phase 1b/2 trial of BXCL501 for the treatment of opioid withdrawal symptoms, is progressing on track, with the topline readout expected this month.
BioXcel has initiated the PLACIDITY trial, a Phase 2 trial of BXCL501 for the treatment of hospitalized patients with delirium related agitation. The Company expects to report topline results from this trial in Q1 2022.
BioXcel and its collaborators, the VA Connecticut Healthcare System and the Yale University Medical School, were awarded a grant by the U.S. Department of Defense’s ("DOD") Congressionally Directed Medical Research Programs ("CDMRP") to evaluate BXCL501 in patients suffering from post-traumatic stress disorder ("PTSD") related to alcohol and substance abuse disorder ("ASUD"). This will be the first time the Company is investigating BXCL501 as a potential chronic treatment.
The Company was issued U.S. patent No. 10,792,24 on October 6, 2020, which covers film formulations containing Dex and methods of treating agitation using such film formulations. The patent is expected to extend intellectual property ("IP") protection until 2039.
Commercial Highlights

Readiness initiatives for the potential launch of BXCL501 in schizophrenia and bipolar disorder related agitation continue to progress, including the infrastructure needed to support commercialization, the design of our sales force structure and size, as well as market research with both healthcare professionals and payers to inform launch strategy, communications and the BXCL501 value proposition.
The launch of our educational campaign on schizophrenia and bipolar disorder related agitation is planned for May 2021, in sync with Mental Health Awareness Month.
Medical Affairs Initiatives

Our Medical Science Liaison ("MSL") and Medical Manage Care ("MMC") teams are completing their training and will be deployed this month to begin scientific medical to medical exchange with healthcare professionals and payers, respectively.
Two abstracts on SERENITY I and II have been accepted for presentation at the American Psychiatric Association ("APA") Annual Meeting in May 2021.
BXCL701-Immuno-Oncology Program

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

The Phase 2 efficacy portion of the Phase 1b/2 trial of BXCL701 in combination with pembrolizumab (KEYTRUDA) for treatment emergent Neuroendocrine Prostate Cancer ("tNEPC") and castrate-resistant prostate cancer ("CRPC") is advancing, with topline results expected in mid-2021. In November 2020, interim data from this trial was presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 35th Anniversary Annual Meeting ("SITC"), with an efficacy update presented last month at the 2021 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium.
Preliminary efficacy data from the MD Anderson-led Phase 2 open label basket trial evaluating the combination of BXCL701 and KEYTRUDA in patients with advanced solid tumors was presented at SITC (Free SITC Whitepaper). Topline results from the trial are expected in mid-2021.
In January, the FDA granted orphan drug designation to BXCL701 for the treatment of soft tissue sarcoma.
Corporate Highlights

BioXcel continues to integrate and evolve its neuroscience AI machine learning and drug development platform, focusing on symptoms resulting from stress-related behaviors. This platform led to the identification and rapid development of BXCL501, leading to the submission of its first NDA. The Company continues to leverage the platform to identify and develop new neuroscience programs.
In March 2021, June Bray was appointed to the Company’s Board of Directors. Ms. Bray brings over forty years of extensive U.S. and global regulatory experience across all therapeutics areas, including psychiatry and neurology.
In February 2021, Javier Rodriguez was appointed as Chief Legal Officer and Corporate Secretary of BioXcel. Mr. Rodriguez joins the Company with over 20 years of extensive strategic and legal experience within the biopharmaceutical industry.
Fourth Quarter and Full Year 2020 Financial Results

BioXcel reported a net loss of $21.1 million for the fourth quarter of 2020, compared to a net loss of $8.3 million for the same period in 2019. For the full year, BioXcel reported a net loss of $82.2 million, compared to a net loss of $33.0 million for the same period in 2019.

For the fourth quarter of 2020, research and development expenses were $11.4 million as compared to $6.5 million for the same period in 2019. The increase was primarily attributable to increased personnel, clinical trial and professional research costs primarily related to our BXCL501 studies.

Research and development expenses were $58.0 million for the full year 2020, as compared to $25.8 million for the same period in 2019. The increase for the year ended December 31, 2020 was generally attributable to increased clinical trial costs, compensation costs and professional research costs associated with BXCL501, as the Company continues to expand its clinical programs.

General and administrative expenses were $9.7 million for the fourth quarter of 2020, as compared to $1.9 million for the same period in 2019. The increase was primarily due to an increase in compensation costs related to the growth of BioXcel’s operations. Professional fees also increased primarily related to costs associated with preparation for the potential commercial launch of BXCL501.

General and administrative expenses were $24.3 million for full year 2020, as compared to $7.8 million for the same period in 2019. The increase was primarily due to the growth of BioXcel’s operations. Professional and consulting fees also increased due to the expansion of our corporate activities.

The fourth quarter 2020 results include approximately $6.6 million in non-cash stock-based compensation costs. For the full year, non-cash stock-based compensation costs totaled $14.6 million.

As of December 31, 2020, cash and cash equivalents totaled approximately $213.1 million.

Conference Call:

BioXcel 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 BioXcel website at www.bioxceltherapeutics.com. The replay will be available through at least March 25, 2021.

On March 11, 2021 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported financial and operating results for the fourth quarter and full year 2020 (Press release, BioTime, MAR 11, 2021, View Source [SID1234576501]). Lineage management will host a conference call and webcast today at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its fourth quarter and full year 2020 financial and operating results and to provide a business update.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Our goal is to build Lineage into the preeminent allogeneic cell transplant company, and we hit our stride in 2020, reaching significant clinical, manufacturing, and business milestones and creating substantial value for our shareholders. We also positioned ourselves for success in 2021 and beyond"

Tweet this
"Our goal is to build Lineage into the preeminent allogeneic cell transplant company, and we hit our stride in 2020, reaching significant clinical, manufacturing, and business milestones and creating substantial value for our shareholders. We also positioned ourselves for success in 2021 and beyond," stated Brian M. Culley, Lineage CEO. "We know of no other company that possesses a comparable combination of cell therapy patent breadth, in-house manufacturing capabilities, and encouraging clinical evidence in three distinct disease areas, each with large unmet needs and billion-dollar commercial opportunities. We believe the field of cell therapy is poised for explosive growth in the months and years ahead. Our objective is to be positioned for that growth by continuing to provide evidence that allogeneic approaches can generate safety and efficacy data which leads to commercial and clinical advantages over alternate approaches. Importantly, Lineage also recently added $35.9 million in new capital through the timely sales of our equity and marketable securities. We believe this new capital will ensure we are funded to deliver additional significant milestones from our novel cell therapy pipeline and provide us with optionality in ongoing partnership discussions."

Some of the significant milestones we achieved during 2020 include:

– Completion of enrollment in a 24 patient Phase 1/2a clinical study of OpRegen for the treatment of dry age-related macular degeneration (AMD) with geographic atrophy (GA) with encouraging preliminary signs of tolerability and efficacy;

– Announcing the first known finding of retinal tissue restoration in a patient who received a retinal pigment epithelium (RPE) cell transplant which had persisted to 23 months with further improvements to visual acuity;

– Making major manufacturing improvements to our OPC1 acute spinal cord injury (SCI) program, including to the process, purity, and scale, and to the development of a "ready-to-inject" formulation, enabling use at a much larger number of treatment centers;

– The early exercise of our option with Cancer Research UK to bring the VAC immuno-oncology platform in-house;

– Reporting encouraging preliminary Phase 1 clinical study results with VAC2 for the treatment of non-small cell lung cancer with evidence of high levels of antigen-specific immunogenicity observed in all patients treated to date;

– Receiving a new research & development grant from the Israel Innovation Authority under their bio-convergence initiative, for the development of a novel bio-retinal patch for the treatment of retinal diseases in partnership with Precise Bio Ltd.;

– Announcing the extension of an OpRegen development grant from the Israel Innovation Authority;

– Receiving a $24.6 million payment from Juvenescence Ltd. for principal and interest due under a convertible promissory note issued as partial payment for the sale of common stock of AgeX Therapeutics, Inc.; and

– Successfully monetizing portions of Lineage’s non-core patent portfolio.

Some of the events and milestones that our shareholders can look forward to in 2021 include:

– Present new and accumulated OpRegen data from the ongoing Phase 1/2a clinical study on two occasions during the first and second quarters of 2021;

– Plan to meet with the FDA to discuss further clinical development of the OpRegen program, anticipated in the third quarter of 2021;

– Complete VAC2 patient enrollment in the ongoing Phase 1 clinical study for the treatment of non-small cell lung cancer, anticipated in the second quarter of 2021;

– Evaluate the Neurgain Parenchymal Spinal Delivery (PSD) system for OPC1, currently ongoing and throughout 2021;

– Plan to meet with the FDA to discuss plans to evaluate the Neurgain Parenchymal Spinal Delivery (PSD) system for OPC1, anticipated in the second quarter of 2021;

– Complete OPC1 process development to support a late-stage clinical trial, currently ongoing and throughout 2021;

– Introduce manufacturing enhancements to the VAC2 program, anticipated throughout 2021;

– Report results from the ongoing Phase 1 clinical study of VAC2 for the treatment of non-small cell lung cancer, anticipated in the fourth quarter of 2021;

– Plan to meet with the FDA to discuss further development of the OPC1 program and manufacturing improvements, including a late-stage clinical study, anticipated in the fourth quarter of 2021;

– Evaluate opportunities for new VAC product candidates based on newly discovered tumor antigens/neoantigens, throughout 2021; and

– Evaluate partnership opportunities and expansion of existing external collaborations and identification of new collaborations for OpRegen, OPC1 and VAC2, currently ongoing and throughout 2021.

Balance Sheet Highlights

Cash, cash equivalents, and marketable securities totaled $41.6 million as of December 31, 2020. Marketable securities as of December 31, 2020 include our remaining ownership of unrestricted securities in OncoCyte, and Hadasit Bio-Holdings Ltd (Hadasit).

During 2020, we funded our operations primarily by receiving payment in full for a total of $24.6 million on the Juvenescence promissory note, and by selling a portion of our marketable securities, resulting in net proceeds of approximately $13.1 million.

Additionally, during 2020, we selectively sold 3,094,322 of our common shares under the ATM offering for gross proceeds of approximately $5.1 million (which excludes $0.3 million of cash in transit related to 2020 sales that settled in 2021).

2021 fundraising activities

From January 1, 2021 through March 5, 2021, we sold an additional portion of our marketable securities, resulting in net proceeds of approximately $10.1 million and an additional $19.9 million in gross proceeds through sales of 7,941,122 of our common shares under the ATM offering (which includes $0.3 million in cash in transit related to 2020 sales that settled in 2021). As of March 5, 2021, the value of the Company’s cash and cash equivalents were in excess of $57 million.

As of March 5, 2021, we hold 1,122,401 shares of OncoCyte stock valued at approximately $4.2 million and 169,167 shares of Hadasit stock valued at approximately $330,000, in each case based on the closing prices of those shares on March 5, 2021.

The Company anticipates that net operational spend for 2021 will be approximately $20.0 to $22.0 million, which is similar to 2020 spending levels. The Company believes that it is well funded into 2023 as a result of sustained cost savings initiatives in 2020 and recent fundraising activities.

Fourth Quarter Operating Results

Revenues: Lineage’s revenue is generated primarily from research grants, royalties, and licensing fees. Total revenues for the three months ended December 31, 2020 were approximately $0.4 million, a decrease of $0.8 million as compared to $1.2 million for the same period in 2019. The decrease was primarily related to an approximate $0.6 million decrease in royalties and licensing fees, which was primarily driven by the absence of a $0.6 million upfront payment from a new license agreement in 2019, and a $0.2 million decrease in grant revenues due to the timing of grant related activities.

Operating Expenses: Operating expenses are comprised of research and development (R&D) expenses and general and administrative (G&A) expenses. Total operating expenses for the three months ended December 31, 2020 were $6.1 million, a decrease of $1.9 million as compared to $8.0 million for the same period in 2019.

R&D Expenses: R&D expenses for the three months ended December 31, 2020 were $2.6 million, a decrease of $0.9 million as compared to $3.5 million for the same period in 2019. The overall decrease was primarily related to decreases of $1.5 million in OpRegen and other ophthalmic application expenses, attributable primarily to a decrease in manufacturing activities in 2020 as compared to 2019, and $0.1 million in Renevia and HyStem related expenses, offset by an increase of $0.5 million in OPC1 expenses, attributable primarily to an increase in manufacturing activities, and a $0.3 million increase in VAC program expenses.

G&A Expenses: G&A expenses for the three months ended December 31, 2020 were $3.5 million, a decrease of $1.0 million as compared to $4.5 million for the same period in 2019. The decrease was primarily attributable to decreases of $0.4 million in legal and patent expenses, $0.3 million in rent expense, $0.3 million in expenses related to our merger with Asterias Biotherapeutics, Inc. (Asterias Merger) and $0.2 million in accounting and consulting expenses, offset by a $0.2 million increase related to the cessation of shared services reimbursements.

Loss from Operations: Loss from operations for the three months ended December 31, 2020 was $5.9 million, a decrease of $1.0 million as compared to $6.9 million for the same period in 2019.

Other Income/(Expenses), Net: Other income/(expenses), net for the three months ended December 31, 2020 reflected other income, net of $6.9 million, compared to other income, net of $1.5 million for the same period in 2019. The variance was primarily related to the gain on sale of marketable securities and changes in the value of marketable equity securities for the applicable periods, as well as foreign currency translation adjustments related to Lineage’s international subsidiaries.

Net Income/(Loss) attributable to Lineage: The net income attributable to Lineage for the three months ended December 31, 2020 was $2.0 million, or $0.01 per share (basic and diluted), compared to a net loss attributable to Lineage of ($4.5) million, or ($0.03) per share (basic and diluted), for the same period in 2019.

Full Year Operating Results

Revenues: Lineage’s revenue is generated primarily from research grants, royalties, and licensing fees. Total revenues for the year ended December 31, 2020 were $1.8 million, a decrease of $1.7 million as compared to $3.5 million for the same period in 2019. The decrease was primarily related to a $1.0 million decrease in grant revenue due to less grant-related activities, $0.4 million decrease in royalties from product sales and license fees, and a $0.3 million decrease in the sale of research products and services due to the cessation of such sales.

Operating Expenses: Operating expenses are comprised of R&D expenses and G&A expenses. Total operating expenses for the year ended December 31, 2020 were $27.9 million, a decrease of $14.1 million as compared to $42.0 million for the same period in 2019.

R&D Expenses: R&D expenses for the year ended December 31, 2020 were $12.3 million, a decrease of $5.6 million as compared to $17.9 million for the same period in 2019. The overall decrease was primarily related to a decrease of $6.5 million in OpRegen and other ophthalmic application expenses, attributable primarily to a decrease in manufacturing activities in 2020 as compared to 2019, a decrease of $0.8 million in Renevia and other related expense as we are spending less and actively looking for a commercialization partner, and a $0.5 million decrease in OPC1-related expenses, primarily driven by a return of unspent project funds of approximately $0.8 million from a former Asterias service provider, offset by a $2.2 million increase in VAC program expenses, primarily related to the accrual of the signature fee of £1.25 million ($1.6 million) to Cancer Research UK.

G&A Expenses: G&A expenses for the year ended December 31, 2020 were $15.6 million, a decrease of approximately $8.4 million as compared to $24.0 million for the same period in 2019. The decrease was primarily attributable to a $5.5 million decrease in Asterias Merger related expenses, a $2.1 million reduction in compensation costs as a result of headcount reductions in 2019, a $0.9 million reduction in accounting expenses, a $0.5 million reduction in rent and utilities, a $0.3 million reduction in travel expenses, a $0.3 million reduction in office and information technology related expenses and a $0.2 million reduction in consulting expenses, offset by a $0.9 million increase related to the cessation of shared services reimbursements and a $0.5 million increase in legal and patent expenses.

Loss from Operations: Loss from operations for the year ended December 31, 2020 was $26.4 million, a decrease of $12.5 million as compared to $38.9 million for the same period in 2019.

Other Income, Net: Other income, net for the year ended December 31, 2020 reflected other income, net of $4.5 million, compared to other income, net of $19.6 million for the same period in 2019. The variance was primarily related to the changes in the value of equity method investments and marketable equity securities for the applicable periods, gain on sale of marketable securities for the applicable periods, as well as foreign currency translation adjustments related to Lineage’s international subsidiaries.

Net loss attributable to Lineage: The net loss attributable to Lineage for the year ended December 31, 2020 was $20.6 million, or $0.14 per share (basic and diluted), compared to a net loss attributable to Lineage of $11.7 million, or $0.08 per share (basic and diluted), for 2019.

Conference Call and Webcast

Lineage will host a conference call and webcast today, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its fourth quarter and full year 2020 financial results and to provide a business update. Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through March 19, 2021, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 4176568.

On March 11, 2021 Bicycle Therapeutics plc (NASDAQ: BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported financial results for the fourth quarter and full year ended December 31, 2020 and discussed recent corporate updates (Press release, Bicycle Therapeutics, MAR 11, 2021, View Source [SID1234576500]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Bicycle has overcome the unprecedented challenges caused by the COVID-19 pandemic this past year, executing on our 2020 goals, including advancing multiple Bicycle Toxin Conjugates (BTCs) in the clinic and preparing our first tumor targeted immune cell agonist (TICA) for an expected clinical start later this year, while also thoughtfully strengthening our balance sheet," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "We are encouraged by the progress of our pipeline and are looking forward to continuing the momentum in 2021. With our clinical data presented to date, we believe we are closer to actualizing a world where Bicycles offer a much-needed new treatment paradigm for people living with cancer and other serious diseases."

Fourth Quarter 2020 and Recent Highlights

Announced Publication of Preclinical Data of Tumor-targeted Immune Cell Agonizing Molecules (TICAs) in the Journal for ImmunoTherapy of Cancer. The article, titled "Anticancer immunity induced by a synthetic tumor-targeted CD137 agonist," outlines the work Bicycle is undertaking to unlock a new method of cancer immunotherapy via small molecule agonism of TNF superfamily receptors.
Provided Oncology Pipeline Progress Update. In January 2021, Bicycle announced progress updates for its wholly owned oncology pipeline:
BT1718: The Phase IIa portion of the Phase I/IIa trial of BT1718, a potential first-in-class BTC targeting a key tumor antigen MT1-MMP, is actively enrolling patients in the Phase I/IIa trial sponsored by Cancer Research UK’s Center for Drug Development. Enrollment in the Phase IIa portion of the trial is ongoing at four clinical sites, with additional sites expected to begin enrolling patients during the first half of 2021. Patients are currently being enrolled into two solid tumor cohorts, one in squamous non-small cell lung cancer (NSCLC) and the other in an all-comers "basket" cohort. Following receipt of results from these first two cohorts, Cancer Research UK may initiate up to two additional cohorts.
BT5528: Enrollment in the company-sponsored Phase I/II trial of BT5528, a second-generation BTC targeting EphA2, a target for which prior antibody-based approaches have been unsuccessful, remains on track with additional sites expected to begin enrolling patients in 2021. BT5528 has been dosed up to 8.5mg/m2 weekly, which Bicycle believes, based on pre-clinical studies, is toward the top of the therapeutic range, with transient neutropenia observed at that dose. The dose-escalation of the Phase I/II trial remains ongoing. Bicycle observed preliminary signs of anti-tumor activity. Since implementation of an EphA2 immunohistochemistry (IHC) assay in 2020, as of January 14, 2021, two EphA2 selected patients have enrolled in the trial, one of whom was response evaluable: in a urothelial patient currently receiving 6.5mg/m2 of BT5528 every other week, whose prior lines of therapies included both a PD-1 inhibitor and enfortumab vedotin, a 43% reduction in target lesions was observed at the first radiologic response assessment, constituting a partial response under RECIST version 1.1 criteria. Reductions in non-target lesions were also observed, and the patient remains enrolled in the trial.
BT8009: Early clinical data for BT8009, a second-generation Nectin-4-targeting BTC with a potentially differentiated profile as compared to a Nectin-4 targeting antibody-drug conjugate (ADC), supports a pharmacokinetic profile that is consistent with both preclinical predications and data as of January 14, 2021 from Bicycle’s ongoing Phase I trial of BT5528, a BTC that utilizes the same linker and toxin payload. Patient enrollment in the Phase I portion of the Phase I/II clinical trial of BT8009 remains ongoing. The first clinical trial site outside of the United States opened in the first quarter of 2021.
BT7480: Bicycle continues to advance its novel TICAs and expects BT7480 to enter the clinic in the second half of this year. Bicycle has identified additional TICA candidates targeting natural killer (NK) cells through early immuno-oncology (I-O) discovery efforts and is moving these TICA candidates into lead optimization.
Presented Posters at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 35th Anniversary Annual Meeting. In November 2020, Bicycle presented new data that demonstrated the potential generalizability of its TICA platform, with a Nectin-4/CD137 TICA and an EphA2/CD137 TICA exhibiting similar anti-tumor activity and immune modulation.
Continued to Strengthen the Balance Sheet in 2021. In March 2021, the Company drew an additional $15.0 million available under its debt facility with Hercules Capital, Inc. and amended the loan and security agreement to extend the interest-only payment period until the second half of 2023, with the potential to further extend it into 2024, contingent on the satisfaction of performance milestones. In addition, gross proceeds from Bicycle’s at-the-market (ATM) offering program during the first quarter of 2021 totaled $50.2 million as of March 10, 2021. Cash at December 31, 2020 does not include additional borrowings under the debt facility or gross proceeds from the ATM offering program received in the first quarter of 2021.
Financial Results

Cash was $136.0 million as of December 31, 2020, compared to $92.1 million as of December 31, 2019. The increase in cash at December 31, 2020 was primarily due to net proceeds of $48.1 million received from the ATM offering program during 2020, $31.0 million received from Genentech under the strategic collaboration agreement, as well as $14.4 million in net proceeds received from the debt facility with Hercules Capital, Inc., offset by cash used in operating activities. Cash of $136.0 million at December 31, 2020 is expected to provide financial runway through multiple clinical milestones.
Research and development expenses were $10.1 million for the three months ended December 31, 2020 and $33.1 million for the year ended December 31, 2020, compared to $6.6 million for the three months ended December 31, 2019 and $25.5 million for the year ended December 31, 2019. The increase in expense of $3.5 million for the three months ended December 31, 2020 as compared to the same period in the prior year was primarily due to increased clinical program expenses for BT5528 and BT8009, increased other unallocated discovery and platform related expenses due to the timing of development activities, and increased personnel-related expenses, including $0.3 million of incremental non-cash share-based compensation expense. The increase in expense of $7.6 million for the year ended December 31, 2020 as compared to the same period in the prior year was primarily due to increased clinical program expenses for BT5528 and BT8009, as well as increased TICA program development expenses and increased personnel-related expenses, including $1.3 million of incremental non-cash share-based compensation expense.
General and administrative expenses were $10.9 million for the three months ended December 31, 2020 and $29.2 million for the year ended December 31, 2020, compared to $3.4 million for the three months ended December 31, 2019 and $14.6 million for the year ended December 31, 2019. The increase of $7.5 million for the three months ended December 31, 2020 and $14.6 million for the year ended December 31, 2020 as compared to the same period in the prior year was primarily due to an increase in professional fees and costs including costs due to operations as a public company, the settlement and license agreement with Pepscan entered into in November 2020, an unfavorable effect of foreign exchange rates and an increase in personnel-related costs, including $0.2 million and $2.1 million of incremental non-cash share-based compensation expense for the three months ended December 31, 2020 and for the year ended December 31, 2020, respectively.
Net loss was $17.4 million, or $(0.83) basic and diluted net loss per share, for the three months ended December 31, 2020, and net loss was $51.0 million, or $(2.66) basic and diluted net loss per share for the year ended December 31, 2020, compared to net loss of $4.4 million, or $(0.25) basic and diluted net loss per share for three months ended December 31, 2019, and net loss of $30.6 million, or $(2.77) basic and diluted net loss per share for the year ended December 31, 2019.

On March 11, 2021 Bicycle Therapeutics plc (NASDAQ: BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle ) technology, reported that management will participate in a fireside chat at the Oppenheimer 31st Annual Healthcare Conference on March 16, 2021 at 8:40 a.m. ET (Press release, Bicycle Therapeutics, MAR 11, 2021, View Source [SID1234576499]). The conference will be held in a virtual meeting format.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of the fireside chat will be accessible in the Investors & Media section of Bicycle’s website at www.bicycletherapeutics.com. An archived replay of the webcast will be available for 90 days following the presentation date.

On March 11, 2021 Alpine Immune Sciences, Inc. (NASDAQ:ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune/inflammatory diseases, reported the company will release fourth quarter and full year 2020 financial results on Thursday, March 18, 2021 after the close of market (Press release, Alpine Immune Sciences, MAR 11, 2021, View Source [SID1234576498]). Alpine will host a corresponding conference call and live webcast at 4:30 p.m. ET/1:30 p.m. PT on the same day to discuss the results and provide a corporate update.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Conference Call and Webcast Details

To access the live call by phone, dial (800) 816-3005 (domestic) or (857) 770-0069 (international) and reference conference ID: 2539338. A live webcast of the presentation will be available online in the investor relations section of the company’s website at View Source A replay of the presentation will be available on the company website for 90 days following the webcast.