On March 4, 2021 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, reported financial results for the fourth quarter and full year ended December 31, 2020 (Press release, X4 Pharmaceuticals, MAR 4, 2021, View Source [SID1234576072]). The company also provided an update on its lead product candidate, mavorixafor, a novel small molecule currently being evaluated in a Phase 3 clinical trial (4WHIM) for patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome and in two Phase 1b trials for patients with Waldenström’s macroglobulinemia (Waldenström’s) and Severe Congenital Neutropenia (SCN), respectively.

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!

"As many companies did, we encountered a number of unexpected challenges in 2020, most notably the COVID-19 pandemic. However, I could not be prouder of our accomplishments, as we remained steadfast in our commitment to advance our lead candidate, mavorixafor, in a number of rare disease indications," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "We believe that we have much to look forward to this year, as we have overcome these obstacles, have made significant progress in advancing our clinical and research programs, and assembled an exceptional team to execute on our 2021 goals and beyond.

"On the clinical front, we continue to expect top-line data from our Phase 3 WHIM trial in 2022 and, as our Phase 1b Waldenström’s trial is progressing well, we continue to expect initial data from that study in the first half of 2021, with a more fulsome dataset expected towards the end of 2021. We also anticipate initial data from our SCN Phase 1b trial in 2021."

Recent Highlights

Received Fast Track and Rare Pediatric Designations (RPD) from the FDA for Mavorixafor for the treatment of WHIM Syndrome: These FDA designations further recognize WHIM as a serious condition with a clear unmet need in both adult and pediatric populations.
Through the Fast Track program, X4 will be eligible for more frequent meetings with the FDA to discuss the drug’s development plan, protocols and clinical data that would support mavorixafor’s potential approval for WHIM.
Under the RPD program, a sponsor who receives an approval for a drug for a "rare pediatric disease" and a Fast Track designation may qualify for a voucher that can be redeemed to receive a priority review by the FDA for any subsequent marketing application for a different product. Such a voucher is transferable and may be sold.
Mavorixafor was previously granted Breakthrough Therapy Designation by the FDA, as well as Orphan Drug status by the FDA and the European Commission for the treatment of WHIM syndrome.
Strengthened Leadership Team: During the Fourth Quarter of 2020:
Diego Cadavid, M.D., joined as Chief Medical Officer, bringing more than 20 years of industry experience and having led multiple programs through all phases of clinical development, including small molecules and biologics for the treatment of rare and immunological diseases.
Art Taveras, Ph.D., joined as Chief Scientific Officer with more than 30 years of experience leading small molecule research and development programs focused on cancer, dysregulated immune disorders, neurodegeneration, and metabolic diseases.
Alison Lawton was appointed to the company’s Board of Directors, bringing more than 30 years of experience across a full spectrum of drug development and commercial roles and having previously served on X4’s corporate advisory board and as consulting Chief Operating Officer.
Fourth Quarter 2020 Financial Results

Cash, Cash Equivalents & Restricted Cash: X4 had $80.7 million in cash, cash equivalents and restricted cash as of December 31, 2020. X4 continues to expect that its cash and cash equivalents will fund company operations into 2022.
Research and Development Expenses were $12.3 million and $41.9 million for the fourth quarter and full year ended December 31, 2020, respectively, as compared to $7.1 million and $30.2 million for the comparable periods in 2019, respectively. R&D expenses include $0.6 million and $2.3 million of certain non-cash expenses for the fourth quarter and fully year ended December 31, 2020, respectively.
General and Administrative Expenses were $5.4 million and $20.9 million for the fourth quarter and full year ended December 31, 2020, respectively, as compared to $3.9 million and $17.6 million for the comparable periods in 2019, respectively. G&A expenses include $0.8 million and $3.1 million of certain non-cash expenses for the fourth quarter and full year ended December 31, 2020, respectively.
Net Loss: X4 reported net losses of $18.4 million and $62.1 million for the fourth quarter and full year ended December 31, 2020 as compared to net losses of $10.8 million and $52.8 million for the comparable periods in 2019, respectively. Net losses include $1.4 million and $5.4 million of certain non-cash expenses for the fourth quarter and full year ended December 31, 2020, respectively. Net losses in the full year of 2019 included a $3.9 million loss on the sale of non-financial assets.
Conference Call and Webcast
The Company will host a conference call and webcast today at 8:30 a.m. ET to discuss these financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 1053189. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.

On March 4, 2021 NuCana plc (NASDAQ: NCNA) reported its financial results for the fourth quarter and year ended December 31, 2020 and provided an update on its broad clinical program with its transformative ProTide therapeutics (Press release, Nucana BioPharmaceuticals, MAR 4, 2021, View Source [SID1234576088]).

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!

As of December 31, 2020, NuCana had cash and cash equivalents of £87.4 million compared to £100.7 million at September 30, 2020 and £52.0 million at December 31, 2019. NuCana continues to advance its various clinical programs and reported a net loss of £12.3 million for the quarter ended December 31, 2020, as compared to £7.7 million for the quarter ended December 31, 2019. Net loss for the year ended December 31, 2020 was £30.7 million, compared to a net loss of £21.4 million for the year ended December 31, 2019. Basic and diluted loss per share was £0.24 for the quarter and £0.81 for the year ended December 31, 2020, as compared to £0.24 per share for the comparable quarter and £0.66 for the year ended December 31, 2019.

"2020 was a very successful year for NuCana," said Hugh S. Griffith, NuCana’s Founder and Chief Executive Officer. "Against the challenging backdrop of the COVID-19 pandemic, we continued to advance all of our clinical programs and generate encouraging clinical and non-clinical data. Throughout 2020, we made good progress in the Phase III NuTide:121 biliary tract cancer study and expect to enroll sufficient patients in 2021 to complete the first interim analysis in 2022, which may support an accelerated approval filing. In September, we provided an update on two of our lead programs at ESMO (Free ESMO Whitepaper) that further validate the use of our proprietary ProTide technology. NUC-3373 continued to demonstrate its potential to offer enhanced efficacy, an improved safety profile and a more convenient dosing regimen as compared to 5-FU in patients with colorectal cancer. We also announced the first-ever clinical data for NUC-7738, which showed promising indications of anti-cancer activity and a favorable tolerability profile."

Mr. Griffith continued: "During 2020, we completed a successful $80 million follow-on offering that significantly strengthens our balance sheet. We also appointed a new non-executive Chairman, Andrew Kay and a new non-executive director, Bali Muralidhar. Both individuals bring a wealth of experience and we look forward to their contributions as we advance NuCana’s programs."

Mr. Griffith concluded: "We were excited to begin 2021 with the announcement of additional interim data for NUC-3373 in patients with colorectal cancer that continued to demonstrate encouraging efficacy and safety at ASCO (Free ASCO Whitepaper) GI. We look forward to continuing to drive recruitment across all of our ongoing studies, announce additional clinical data, and initiate new studies in 2021. We remain focused on our mission of significantly improving treatment outcomes for patients with cancer."

Anticipated 2021 Milestones

Acelarin is a ProTide transformation of gemcitabine. In 2021, NuCana expects to:

Complete recruitment sufficient to enable the first interim analysis in 2022 of the Phase III study of Acelarin combined with cisplatin as a first-line treatment for patients with advanced biliary tract cancer.

NUC-3373 is a ProTide transformation of the active anti-cancer metabolite of 5-FU. In 2021, NuCana expects to:

Report data from the Phase Ib study (NuTide:302) of NUC-3373 in combination with other agents with which 5-FU is typically combined, such as leucovorin, oxaliplatin and irinotecan in patients with advanced colorectal cancer.
Initiate and report data from a Phase Ib expansion / Phase II study of NUC-3373 in combination with other agents for patients with colorectal cancer.
Initiate a Phase III study of NUC-3373 in combination with other agents for patients with colorectal cancer.
Report data from the Phase I study (NuTide:301) of NUC-3373 in patients with advanced solid tumors.
NUC-7738 is a ProTide transformation of a novel nucleoside analog, 3’-deoxyadenosine. In 2021, NuCana expects to:

Report data from the Phase I study (NuTide:701) of NUC-7738 in patients with advanced solid tumors.
Initiate a Phase II study of NUC-7738 in patients with solid tumors.

On March 4, 2021 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that Alan H. Auerbach, Chairman, Chief Executive Officer, President and Founder of Puma, will provide an overview of the Company at the Barclays Global Healthcare Conference, which will be held virtually from March 9-11, 2021 (Press release, Puma Biotechnology, MAR 4, 2021, View Source [SID1234576120]). Puma’s presentation will take place at 1:50 p.m. EST (10:50 a.m. PST) on Thursday, March 11, 2021.

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 presentation will be available on the Company’s website at www.pumabiotechnology.com. The presentation will be archived on the website and available for 30 days.

On March 4, 2021 Exscientia, a clinical stage pharmatech pioneering the use of artificial intelligence (AI) to design new drugs, reported that funds managed by BlackRock joined the Company’s Series C investment round (Press release, Exscientia, MAR 4, 2021, View Source [SID1234576073]). Including existing Series C investors, Novo Holdings, Evotec, Bristol Myers Squibb, and GT Healthcare Capital, the round totalled $100 million in funding.

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!

This new capital will be used to support Exscientia’s platform development towards autonomous drug design. In addition, the company will extend its proprietary pipeline into clinical trials and expand existing capabilities in biological analytics that support target selection and portfolio development.

With the company’s end-to-end AI-first drug discovery platform, CentaurAI, Exscientia has been able to generate novel drugs and overcome conventional drug discovery limitations several years faster than industry benchmarks. Exscientia has demonstrated the platform’s capabilities by creating the first fully AI-designed drug to enter clinical trials and advancing multiple drug candidates into preclinical testing. In addition to its growing proprietary pipeline, the company has conducted drug discovery partnerships with Bristol-Myers Squibb, Sanofi, Bayer and Dainippon Sumitomo as well as several biotech companies.

"Exscientia is breaking ground in small molecule drug design, with a platform that radically improves drug discovery" says William Abecassis, Head of BlackRock’s Innovation Capital who will join Exscientia’s Board of Directors as an observer. "We are thrilled to be investing in this world-class team, who are already delivering results with AI-designed drugs now entering clinical trials."

Andrew Hopkins, CEO of Exscientia added, "We are delighted that BlackRock shares our vision for revolutionising how drugs are discovered. It is also recognition of the ingenuity and hard work of our employees to turn the promise of AI into reality today, where we are bringing the world’s first AI designed drugs into the clinic. BlackRock’s investment is an important step in our vision that all drugs will be designed by AI. I believe that our company’s reimagined approach to drug discovery will become the new de facto standard."

Exscientia has doubled in size over the past year and expects to double again in 2021, including a recent US expansion into Miami and Boston. The company employs over 100 people from over 20 different nations, with equal balance of technologists and drug discovery scientists, of which 60% are PhDs. Exscientia is named the fastest growing private biotech or pharmaceutical company in the UK in the 2021 Alantra Pharma Fast 50, as well as one of the fastest growing healthcare companies in the Financial Times FT1000.

On March 4, 2021 NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class immunomedicines to treat cancer and other immune-related diseases, reported fourth quarter and full year 2020 financial results and provided a business update (Press release, NextCure, MAR 4, 2021, View Source [SID1234576089]).

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 team has worked diligently throughout 2020 to advance our programs despite the difficult environment created by the COVID-19 pandemic. We are thrilled that Dr. Han Myint has recently joined NextCure as our Chief Medical Officer and we are poised to harness his expertise in oncology product development to further advance our product pipeline. As we enter 2021, we are excited to announce the expansion of our pipeline. We remain encouraged with the potential of NC318 given the evidence of clinical activity seen to date and are now reexploring the treatment of lung cancer through an investigator-initiated trial at Yale University," said Michael Richman, NextCure’s president and chief executive officer. "In addition, the NC410 Phase 1 trial is advancing and we look forward to reporting initial data in the second half of the year and we are very excited to introduce NC762, a B7-H4 antibody expected to enter the clinic next quarter."

Business Highlights

NC318
The two previously announced objective responder patients in the Phase 1 portion of the company’s ongoing Phase 1/2 clinical trial are continuing to receive drug and remain on study at over 118 weeks and over 92 weeks.
The trial continues in head and neck squamous cell carcinoma (HNSCC) and triple-negative breast cancer (TNBC) patients.
The previously announced confirmed partial response (PR) seen in the HNSCC cohort is off study after 40 weeks.
A confirmed PR has been observed in the TNBC cohort and the patient remains on study at over 21 weeks.
Given these responses in the HNSCC and TNBC cohorts, these cohorts met the criteria to advance into the stage 2 portion of the Simon 2-stage trial.
The NC318 Phase 2 monotherapy protocol will be revised to select S15+ patients for enrollment starting in the second quarter of this year.
Yale University plans to initiate in the second quarter of 2021, an investigator-initiated Phase 2 trial of NC318 as a monotherapy and in combination with pembrolizumab in patients with advanced non-small cell lung cancer.
NC410
Enrollment is on track and the company expects to announce initial clinical data from the Phase 1 portion of the trial in the second half of 2021.
NC762
An investigational new drug (IND) application has been filed with the U.S. Food and Drug Administration (FDA) for NC762, a humanized monoclonal antibody targeting B7-H4, which is upregulated in multiple solid tumor types, and which we believe is differentiated from other B7-H4-targeted antibodies.
Announced the appointment of Dr. Han Myint as Chief Medical Officer.
Expected Upcoming Milestones

Initiate in the second quarter of 2021, the Yale University Phase 2 investigator-initiated trial of NC318 in NSCLC with anticipated initial data first half of 2022.
Report NC318 Phase 2 monotherapy data in the fourth quarter of 2021.
Initiate the NC762 Phase 1 trial in the second quarter of 2021 and report initial data in mid-2022.
Report NC410 initial Phase 1 data in the second half of 2021.
Financial Guidance
Based on its current research and development plans, NextCure expects its existing cash, cash equivalents and marketable securities will enable it to fund operating expenses and capital expenditure requirements into the second half of 2023.

Financial Results for Fourth Quarter and Full Year Ended December 31, 2020

Cash, cash equivalents, and marketable securities, excluding restricted cash as of December 31, 2020, were $283.4 million as compared with $334.6 million as of December 31, 2019. The decrease of $51.2 million as of December 31, 2020 as compared to December 31, 2019 primarily reflects cash used to fund operations of $44.9 million and cash used to purchase fixed assets of $7.2 million, offset by a reduction in restricted cash of $1.5 million.
Research and development expenses were $46.6 million and $12.1 million for the year and quarter ended December 31, 2020, respectively, as compared with $34.2 million and $11.4 million for the year and quarter ended December 31, 2019, respectively. The increase was primarily related to increase in headcount and clinical research costs related to advancing NC318 and NC410.
General and administrative expenses were $17.0 million and $4.1 million for the year and quarter ended December 31, 2020, respectively, as compared with $9.6 million and $2.6 million for the year and quarter ended December 31, 2019, respectively. The increase in general and administrative expenses for the year and quarter primarily related to increasing infrastructure costs and being a public company for a full year in 2020.
Revenue was $22.4 million for the year ended December 31, 2020. There was no revenue recognized in the quarter ended December 31, 2020, as compared with $6.3 million and $2.0 million for the year and quarter ended December 31, 2019, respectively. Revenue generated was from our former research and development agreement with Eli Lilly.
Net loss was $36.6 million and $15.5 million for the year and quarter ended December 31, 2020, respectively, as compared with $33.7 million and $10.9 million for the year and quarter ended December 31, 2019, respectively. The increases in net loss for the year and quarter were primarily due to increased research and development expenses and increased general and administrative expenses from an increase in headcount, offset by the recognition of the remaining deferred revenue under the former agreement with Lilly.
Conference Call
NextCure will host a virtual R&D update event today at 5 pm Eastern time. To participate via telephone, please dial (877) 665-6632 from the U.S. or (602) 563-8471 from outside the U.S., using the conference ID 6773907. To participate via live or replay webcast, a link is available through the Investors section of the company’s website at www.nextcure.com.

About NC318
NC318 is a first-in-class immunomedicine against S15, a novel immunomodulatory target found on highly immunosuppressive cells called M2 macrophages in the tumor microenvironment and on certain tumor types including lung, ovarian and head and neck cancers. In preclinical research, it was observed that S15 promoted the survival and differentiation of suppressive myeloid cells and negatively regulated T cell function, allowing cancer to avoid immune destruction. In preclinical studies, NC318 blocked the negative effects of S15. NextCure believes NC318 has the potential to treat multiple cancer types.

About NC410
NC410 is a first-in-class immunomedicine designed to block immune suppression mediated by LAIR-1, an immunomodulatory receptor expressed on T cells and dendritic cells, a type of antigen presenting cell. In preclinical research, it was observed that LAIR-1 inhibited T cell function and dendritic cell activity allowing tumor cells to grow. In preclinical studies, NC410 blocked the negative effects of LAIR-1 and promoted T cell function and dendritic cell activity. NextCure believes NC410 has the potential to treat multiple cancer types.

About NC762
NC762 is a monoclonal antibody that binds specifically to B7-H4, a protein expressed on multiple tumor types. We believe NC762 acts by inhibiting tumor cell growth and killing tumor cells, including by enhancing immune response. We have observed in preclinical studies that NC762 inhibits the growth of human melanoma tumors in mice, and we believe that NC762 has the potential to treat multiple tumor types. Our research indicates that NC762 inhibits tumor cell growth independently of immune cell infiltration in the tumor microenvironment.