On May 17, 2021 Onconova Therapeutics, Inc. (NASDAQ: ONTX) ("Onconova"), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, reported financial results for the three months ended March 31, 2021 and provided a business update (Press release, Onconova, MAY 17, 2021, View Source [SID1234580149]).

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Highlights for the first quarter of 2021 and subsequent weeks include:

The Phase 1 solid tumor study with ON 123300 in China is ongoing with no dose-limiting toxicities observed in the first two cohorts. Enrollment to the third cohort (120 mg) will now proceed.
The Phase 1 study with ON 123300 in the United States is open for enrollment, and actively screening patients.
The first patient has been dosed in an investigator-initiated Phase 2 study designed to assess the efficacy and safety of rigosertib in patients with recessive dystrophic epidermolysis bullosa (RDEB)-associated locally advanced/metastatic squamous cell carcinoma (SCC).
The investigator-initiated Phase 1/2 study evaluating rigosertib in combination with the checkpoint inhibitor nivolumab in KRAS mutated non-small cell lung cancer has progressed nicely and has reached the maximum dose of oral rigosertib per the current protocol.
The Company strengthened its balance sheet with net proceeds of $35.2 million from two equity offerings; cash and cash equivalents as of March 31, 2021 were $48.0 million. The Company believes it has more than 18 months of cash runway.
Management Commentary

"We are off to a strong start in 2021 and remain focused on advancing our clinical programs, in particular with our lead product candidate ON 123300, a multi-kinase inhibitor that potently targets CDK 4 and 6, which are overexpressed in a number of cancers, including HR+ HER 2- metastatic breast cancer, a potential blockbuster commercial opportunity," said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova. "We are delighted that our partner in China, HanX Biopharmaceuticals, is expected to begin the third cohort of their Phase 1 study at 120 mg per dose and that ON 123300 appears to be well tolerated with no dose-limiting toxicities observed to date. Notably, the HanX study is dosing patients on days 1 to 21 of 28-day cycles, while the U.S. Phase 1 study will be investigating a continuous daily dosing regimen. Collectively, we expect these complementary studies to generate important safety data that will inform the design of subsequent trials and potentially provide preliminary signals of efficacy in patients with advanced cancer."

Dr. Fruchtman continued, "Alongside the progress made with our lead product candidate, we have also seen advancements in several investigator-initiated trials evaluating rigosertib. The first patient was recently dosed in a Phase 2 trial evaluating rigosertib monotherapy in advanced squamous cell carcinoma associated with recessive dystrophic epidermolysis bullosa, a disease with a critical unmet medical need. Additionally, the Phase 1/2 study evaluating rigosertib in combination with the checkpoint inhibitor nivolumab in KRAS mutated non-small cell lung cancer continues to progress and has reached the highest dose per the current protocol. We expect to continue leveraging our relationships with leading cancer centers and industry collaborators to advance these trials and commence additional investigator initiated studies in RAS-driven cancers in combination with checkpoint inhibitors. We expect such an approach to facilitate our near- and long-term growth by allowing us to preserve our primary focus and resources on ON 123300 while simultaneously pursuing opportunities to develop rigosertib in high unmet need indications."

First Quarter Financial Results

Cash and cash equivalents as of March 31, 2021 were $48.0 million, compared with $19.0 million as of December 31, 2020. The Company believes that its cash and cash equivalents will be sufficient to fund ongoing clinical trials and business operations for more than 18 months.

Research and development expenses were $1.9 million for the first quarter of 2021, compared with $3.4 million for the first quarter of 2020. The decrease was primarily related to lower expenses for the oral rigosertib combination program and the completed Phase 3 INSPIRE study in the 2021 period.

General and administrative expenses were $2.2 million for the first quarter of 2021, compared with $1.8 million for the first quarter of 2020. The increase was primarily related to higher special stockholder meeting by proxy expenses and insurance costs in the 2021 period.

Net loss for the first quarter of 2021 was $4.7 million, or $0.02 per share on 219.2 million weighted average shares outstanding, compared with a net loss for the first quarter of 2020 of $5.1 million, or $0.03 per share on 160.3 million weighted average shares outstanding.

Conference Call and Webcast

Onconova will host an investment community conference call today beginning at 4:30 p.m. Eastern time, during which management will discuss financial results for the first quarter of 2021, provide a business update and answer questions. Interested parties can participate by dialing (855) 428-5741 (domestic callers) or (210) 229-8823 (international callers) and using conference ID 4895447.

A live webcast of the conference call will be available in the Investors & Media section of the Company’s website at www.onconova.com. A replay of the webcast will be available on the Onconova website for 90 days following the call.

On May 17, 2021 NexImmune, Inc. (Nasdaq: NEXI), a clinical-stage biotechnology company developing a novel approach to immunotherapy designed to orchestrate a targeted immune response by directing the function of antigen-specific T cells, reported its financial results for the first quarter of 2021 (Press release, NexImmune, MAY 17, 2021, View Source [SID1234580148]).

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"With a strong cash position following our successful IPO in February, we remain focused on completing enrollment in our clinical trials for NEXI-001 and NEXI-002, and we expect to present additional clinical data at scientific conferences during 2021 for each," said Scott Carmer, Chief Executive Officer. "We are also advancing new cell therapy programs targeting solid tumors and have initiated IND-enabling pre-clinical experiments that will support the initial application of our ‘AIM Injectable’ modality. We look forward to providing further updates as these important programs mature."

Select 1Q 2021 Business Highlights

On February 17, 2021, NexImmune completed a successful IPO and raised gross proceeds of $126M. The IPO was oversubscribed and priced at the top of the range.
NexImmune announced the appointments of Robert Knight, MD as Chief Medical Officer; Jerome Zeldis, MD, PhD as Executive Vice President, Research and Development; Jeffery Weber, MD, PhD as Chief Scientific Advisor and Scientific Advisory Board Chair; Chad Rubin as SVP of Corporate Affairs; and Grant Verstandig as a member of its Board of Directors.
NEXI-001 and NEXI-002 are both in Phase 1/2 clinical trials. The Company will share preliminary data from the initial safety cohorts of both trials at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting and EHA (Free EHA Whitepaper)2021 Virtual Congress for NEXI-001 and NEXI-002, respectively, with additional results for each trial in Q421.
Select 1Q 2021 Financial Highlights

Cash and cash equivalents for the company as of March 31, 2021 were $118.1M, compared to $5.0M at December 31, 2020. Based upon its current operating plans and cash and cash equivalents the Company expects to have sufficient capital to fund its operating expenses and capital expenditure requirements through the second quarter of 2022.

Research and development expenses were $6.0M in the first quarter of 2021, compared to $4.3M for the same period in the prior year. The increase in R&D expenses were mainly attributable to costs for the two clinical trials as well as personnel-related expenses driven by increased headcount, offset partially by reduced preclinical and regulatory-related spending.

General and administrative expenses were $4.1M, compared to $2.1M for the same period the prior year. The increase was due primarily to increases in headcount and fees related to professional and consulting services.

Net loss, according to generally accepted accounting principles in the U.S. (GAAP), was $8.5M for the quarter, or a basic and diluted GAAP loss per share of $0.71. This compared to a net loss of $6.3M for the same period the prior year.

On May 17, 2021 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, Vincerx Pharma, MAY 17, 2021, View Source [SID1234580147]).

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"The clearance of Vincerx’s first company-sponsored IND is an important milestone that paves the way for the initiation of our planned Phase 1b dose escalation study of VIP152 in patients with relapsed/refractory chronic lymphocytic leukemia and Richter syndrome in the second half of the year," said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx. "This rapid execution is a testament to our team’s capabilities, and builds upon our Phase 1b expansion study in patients with MYC-driven hematologic malignancies and solid tumors, expected to begin patient dosing in the second quarter. Both studies are part of our comprehensive clinical program, which leverages early signals of Phase 1 clinical activity to evaluate the potential of VIP152 in challenging oncology populations. We look forward to continued progress across these important milestones in the clinic, which will also include the presentation of Phase 1 data in patients with double-hit lymphoma at ASCO (Free ASCO Whitepaper)."

Dr. Hamdy continued, "For our preclinical assets, we were pleased to present compelling preclinical data on VIP236 at the AACR (Free AACR Whitepaper) Annual Meeting, highlighting that VIP236 has the potential to provide potent, highly targeted antitumor activity to address the needs of patients with advanced and aggressive cancers. We remain focused on rapidly advancing our pipeline from a clinical and regulatory standpoint and look forward to providing further updates."

Recent Highlights

Announced U.S. Food and Drug Administration (FDA) clearance of Investigational New Drug (IND) Application to initiate a Phase 1b dose escalation study evaluating VIP152, a highly selective PTEFb/CDK9 inhibitor, in patients with relapsed/refractory chronic lymphocytic leukemia (CLL) and Richter syndrome (RS)

Presented preclinical data on VIP236, a novel small molecule drug conjugate (SMDC), at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021

Hosted Key Opinion Leader (KOL) event on bioconjugation and CDK9 inhibitors, featuring presentations by Brian Druker, M.D., Knight Cancer Institute, and Anthony W. Tolcher, M.D., NEXT Oncology

Announced that Phase 1 safety and efficacy dose escalation data from patients with double-hit lymphoma will be presented at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting
Announced formation of Scientific Advisory Board (SAB) composed of world leading academics and industry leaders in cancer research and therapeutics

Announced completion of public warrant redemption and receipt of cash proceeds of approximately $37.3 million.
First Quarter 2021 Financial Results

Vincerx Pharma ended the first quarter with $53.4 million in cash and cash equivalents, which does not include the proceeds from the public warrant redemption noted above, compared to $61.8 million at December 31, 2020.

Net loss for the first quarter ended March 31, 2021 was $6.3 million, or $0.46 per share, basic and diluted.

Research and development (R&D) expenses were $4.8 million for the quarter ended March 31, 2021, consisting primarily of $2.7 million in stock-based compensation expense, $1.3 million in new employee salaries and $0.8 million of outside services in preparation for our anticipated clinical trials.

General and administrative (G&A) expenses were $4.8 million for the quarter ended March 31, 2021, consisting primarily of $2.0 million in stock-based compensation expense, $0.9 million related to new employee salaries and $1.5 million of legal, accounting and other professional services in support of our intellectual property protection and operations as a newly formed public company.

On May 17, 2021 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported the appointment of industry veteran Salim Yazji, M.D., as Chief Medical Officer (Press release, Oncternal Therapeutics, MAY 17, 2021, View Source [SID1234580146]).

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"Salim brings a wealth of relevant experience to Oncternal during this exciting time as we begin to advance our pipeline toward the registrational phase," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "We believe that his extensive background in oncology drug development and strategy, experience with global regulatory authorities, and proven leadership ability make him an ideal fit for Oncternal as we advance our lead clinical product candidate, cirmtuzumab, along with our ROR1 targeted CAR-T program and clinical TK216 product candidate and make key strategic decisions about next steps."

Dr. Yazji stated, "I am excited to be joining Oncternal as the Company’s novel pipeline matures and the organization pivots towards key decisions in its clinical programs in both hematological and solid tumors. I look forward to working with the focused team at Oncternal to advance regulatory strategy and execute upon the Company’s current and future clinical development programs."

Dr. Yazji has held positions of increasing responsibility in both large and entrepreneurial organizations. He founded Elpida Therapeutics in January 2019 and co-founded Ajuta Therapeutics in October 2019, where he served as Chief Executive Officer until February 2021. He has also served on the Board of Directors of Versatope Therapeutics since April 2019. From March 2018 to January 2019, he served as Chief Medical Officer of PMV Pharma, and from November 2016 to February 2018, he served as Executive Vice President and Chief Medical Officer of Calimmune, which was acquired by CSL Behring in August 2017. Prior to that, he served as Vice President & Global Head of Oncology at Baxter International from 2013 to 2015 and its spinoff Baxalta from 2015 until it was acquired by Shire Plc in July 2016. From 2009 to 2013, he held global positions of increasing responsibility within Novartis where he led multiple oncology registrational clinical trials, most recently as Senior Global Clinical Leader. Prior to 2009, he held positions with Exelixis, PDL BioPharma, and Johnson & Johnson.

Dr. Yazji obtained his MD from the Pavlov School of Medicine, University of St. Petersburg, St. Petersburg, Russia, and completed his post-graduate training at the University of Texas M.D. Anderson Cancer Center, Park Plaza Hospital, Houston and the Almozov Hospital, St. Petersburg, Russia.

Equity Inducement Grant

On May 17, 2021, Oncternal granted Dr. Yazji options to purchase a total of 425,000 shares of Oncternal common stock, including options to purchase 70,600 shares of Oncternal common stock granted as an inducement award under Oncternal’s 2021 Employment Inducement Incentive Award Plan, which provides for the granting of equity awards to new employees of Oncternal as an inducement to join the Company. The options have a 10-year term and an exercise price equal to the closing price of Oncternal’s common stock on May 17, 2021. The options vest over a four-year period, with 25% of the options vesting on the first anniversary of Dr. Yazji’s employment start date, and the rest vesting in equal monthly installments over three years thereafter. The inducement award was approved by Oncternal’s compensation committee, comprised entirely of independent directors, as required by Nasdaq Rule 5635(c)(4), and was granted as an inducement material to Dr. Yazji entering into employment with Oncternal in accordance with Nasdaq Rule 5635(c)(4).

On May 17, 2021 ONK Therapeutics Ltd, an innovative natural killer (NK) cell therapy company, reported that it has entered into a research collaboration with the National University of Ireland, Galway (NUI Galway) which provides access to unique expertise in evaluating the cancer cell microenvironment in Acute Myeloid Leukaemia (AML) and targeting of AML stem cells in models mimicking the bone marrow microenvironment (Press release, ONK Therapeutics, MAY 17, 2021, View Source [SID1234580145]). The research will support the optimization of ONK Therapeutics’ dual-targeted NK cell therapy program, ONKT104, being developed for the treatment of AML.

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ONK Therapeutics will fund a year-long research program in the laboratory of Dr. Eva Szegezdi, lecturer in Biochemistry, NUI Galway, Head of the Blood Cancer Network Ireland. She has particular expertise in the AML microenvironment as well as cell death pathways, especially those initiated by ‘so-called’ death ligands (e.g. TRAIL) used by effector immune cells.

AML is the most common form of acute leukemia in adults. It is estimated that some 21,000 patients in the US and 18,000 in Europe are diagnosed with AML each year. It has a high unmet medical need having the lowest survival rate of all types of leukemia.

ONKT104 is a dual-targeted NK cell engineered to express a humanized scFv targeting the leukemic stem cell antigen CLL-1 (also known as CLEC12A) obtained through an option license agreement from Cellerant Therapeutics, together with ONK Therapeutics’ proprietary high-affinity TRAIL variant, targeting death receptor 4 (DR4). CLL-1 is selectively expressed on leukemic stem cells with no expression on normal hematopoietic stem cells, which ensures safer targeting and a lower risk of prolonged toxicity to normal bone marrow cells. In pre-clinical research studies, a monoclonal antibody therapy targeting CLL-1 has revealed potential efficacy against AML cells and shown to be effective in reducing AML burden in a xenograft model. In addition, a CLL-1 CAR-T cell model has shown promising pre-clinical activity and has recently entered the clinic.

ONK Therapeutics believes its dual-targeted NK cell therapy approach may have several advantages over a CAR-T approach including shorter persistence of NK cells, reducing the risk of sustained neutropenia; proven inherent anti-AML activity of NK cells; the reduced likelihood of toxicity due to cytokine release syndrome or neurotoxicity; and the logistically simpler allogeneic, off-the-shelf nature of NK cells, reducing time to treatment once suitable patients are identified.

AML is a very challenging disease in which to achieve sustained, long term disease control due to the high plasticity and adaptability of AML stem cells, and the tendency for resistant cells to emerge and grow. In addition to targeting CLL-1, this project will evaluate multi-targeted approaches by combined targeting of other leukemia stem cell antigens.

ONK Therapeutics’ founder and CSO Prof Michael O’Dwyer said, "Alongside our in-house research, the project team at NUI Galway will explore construct design, as well as the potential added benefit of certain gene edits to enhance NK cell cytotoxicity, cytokine production and persistence in the context of AML strengthening our ONKT104 program. The aim is to select an optimized candidate to take forward into clinical development as a treatment for patients with relapsed/refractory AML."

Dr. Eva Szegezdi said, "The project will evaluate different constructs that may be able to achieve synergistic killing of cancer cells and reduce the emergence of disease resistance. These include the co-expression of CARs targeting other AML antigens, in addition to CLL-1, such as CD96, TIM3, and CD38 alongside the TRAIL variant."

ONK Therapeutics was formed based on technology and intellectual property developed at NUI Galway by Prof. Michael O’Dwyer, who retains his academic position as Professor of Haematology, Consultant Haematologist and HRB Clinician Scientist, alongside his role at the company. Over the past 12 months, ONK Therapeutics has expanded its team and operations at its headquarters and R&D facility in Ireland’s med-tech hub in Galway, where it now has 16 employees, with an additional 5 employees based in its US subsidiary in San Diego.