On March 24, 2021 Nascent Biotech, Inc. (OTCQB:NBIO) ("Nascent Biotech", "Nascent", or the "Company"), a clinical-stage biotechnology Company pioneering the development of monoclonal antibodies targeting treatment of various cancers and viral infections, reported that the Company has officially opened its Phase I trial to evaluate Pritumumab (PTB), the Company’s lead Monoclonal Antibody (Mab) therapeutic asset, as a treatment option for Brain Cancer, including Malignant Primary Brain Tumors and adult Brain Metastases (Press release, Nascent Biotech, MAR 24, 2021, View Source [SID1234577082]).

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Screening to enroll patients in the trial is already underway. Those interested may review trial requirements here.

PTB is a natural human antibody that works by binding to Cell surface Vimentin (sometimes referred to as ectodomain vimentin, or EDV), a protein expressed on the surface of epithelial cancers. The PTB antibody is used as a targeted immunotherapy, which unlike chemotherapy, targets only the cancer cells without damaging healthy cells.

"Because EDV is found in a variety of cancers, our important clinical trials involving PTB could have implications for a broad range of more common cancers, such as breast, colon and lung," noted Nascent CEO, Sean Carrick. "The current Phase 1 trial has been uniquely designed to ultimately treat a variety of brain cancers from gliomas and other primary brain tumors to brain metastases and leptomeningeal cancers arising from breast, lung and other solid tumors."

On March 24, 2021 Aileron Therapeutics (NASDAQ:ALRN), a chemoprotection oncology company focused on fundamentally transforming the experience of chemotherapy for cancer patients, reported business highlights and financial results for the fourth quarter and year ended December 31, 2020 (Press release, Aileron Therapeutics, MAR 24, 2021, View Source [SID1234577081]).

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"In the fourth quarter of 2020, our Phase 1b trial in SCLC showed that ALRN-6924 has the potential for best-in-class protection against thrombocytopenia, neutropenia and anemia in SCLC patients receiving second-line topotecan chemotherapy. Bolstered by these robust proof-of-concept data, we are on the threshold of initiating our first randomized, double-blind placebo-controlled trial with ALRN-6924 to ‘chemoprotect’ patients with NSCLC who are receiving first-line chemotherapy," said Manuel Aivado, M.D., Ph.D., President and Chief Executive Officer.

Dr. Aivado further commented, "Given our significant capital raises in early 2021, we now believe that we have the resources to strategically invest in CMC, p53 companion diagnostic development and team scale-up, with the explicit goal of rapidly advancing ALRN-6924 into late-stage development for NSCLC following the Phase 1b trial. Bringing selective chemoprotection to NSCLC patients, approximately 50% of whom have a p53 mutation, will be a critical step toward achieving our vision of bringing selective chemoprotection to patients with p53-mutated cancers across all types of cancers and chemotherapies."

Aileron is developing ALRN-6924 to selectively protect healthy cells in patients with cancers that harbor p53 mutations, which are present in over half of all cancer patients, to reduce or eliminate chemotherapy-induced side effects while not interfering with chemotherapy’s attack on cancer cells, a novel concept known as chemoprotection. By reducing or eliminating multiple chemotherapy-induced side effects, ALRN-6924 may improve patients’ quality of life and help them better tolerate chemotherapy. Enhanced tolerability may result in fewer dose reductions or delays of chemotherapy and the potential for improved efficacy. The regulatory pathway has been clarified for chemoprotection with the recent U.S. Food and Drug Administration (FDA) approval of the first myeloprotective drug for patients with extensive-stage SCLC.1 Given Aileron’s biomarker approach, designed to ensure selective chemoprotection, coupled with the high prevalence of p53-mutated cancers, the company’s strategy is to ultimately pursue a tumor-agnostic indication for ALRN-6924.

Recent and Fourth Quarter and Full Year 2020 Highlights

Recent Highlights

Announced enrollment expansion for upcoming Phase 1b clinical trial of ALRN-6924 in patients with advanced NSCLC. In February 2021, Aileron announced a 50% expansion of its enrollment target for its upcoming Phase 1b clinical trial of ALRN-6924 in patients with NSCLC undergoing chemotherapy. Aileron plans to enroll 60 patients, increased from the original target of 40 patients, with advanced p53-mutated NSCLC undergoing treatment with first-line carboplatin plus pemetrexed (with or without immune checkpoint inhibitors). Aileron anticipates this enrollment expansion will enable a more robust exploration of ALRN-6924 as a novel chemoprotective agent to prevent toxicities in the NSCLC patient population and better position the company to rapidly advance ALRN-6924 into late-stage clinical development for NSCLC following the Phase 1b trial.

In the Phase 1b NSCLC trial, patients will be randomized 1:1 to receive either carboplatin/pemetrexed plus 0.3 mg/kg ALRN-6924 or carboplatin/pemetrexed plus placebo for at least four 21-day treatment cycles. Evaluations will include the proportion of treatment cycles free of severe hematological and other toxicities, transfusions and the use of growth factors, as well as the impact on quality of life. Aileron plans to begin enrolling patients in the NSCLC trial in the second quarter of 2021 and anticipates reporting interim data at the end of 2021 and topline results in mid-2022.
Completed Phase 1b trial of ALRN-6924 in patients with SCLC receiving ALRN-6924 prior to topotecan and conducted preliminary evaluation of data from additional cohorts. Since Aileron’s late-breaking presentation of proof-of-concept data from the Phase 1b SCLC trial at the 32nd EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Annual Symposium on Molecular Targets and Cancer Therapeutics (ENA 2020), the company has completed the trial, including the enrollment of additional cohorts that allowed further analysis of the ALRN-6924/topotecan dosing interval and dose-response relationship. A preliminary evaluation of data from 11 additional patients (n=7 patients receiving 0.3 mg/kg ALRN-6924 six hours before topotecan and n=4 patients receiving 0.2 mg/kg ALRN-6924 twenty-four hours before topotecan) was in line with the data presented in October and with Aileron’s expectation that administering ALRN-6924 at 0.3mg/kg and 24 hours before topotecan remains the optimal dose and schedule in this patient population. Aileron expects to submit final results of the Phase 1b SCLC trial for presentation at a medical conference in the second half of 2021.

Raised $55.7 million in aggregate proceeds from the sale of common stock during first quarter 2021, which based on Aileron’s current operating plan, is expected to provide funding into the second half of 2023 and approximately 12 months beyond the anticipated topline results from the NSCLC trial. In January 2021, Aileron completed a registered direct offering of common stock, for a purchase price of $1.10 per share, raising $33.1 million in aggregate net proceeds, after deducting placement agent fees and other offering expenses payable by Aileron. New fundamental investors, including Acorn Bioventures, BVF Partners, L.P., Maven Investment Partners and Grand Oaks Capital, participated in the offering, in addition to several existing Aileron investors, including Satter Medical Technology Partners and Lincoln Park Capital Fund, LLC. In addition to the registered direct offering, since January 1, 2021, Aileron has sold an aggregate of 13,775,399 shares of its common stock for aggregate net proceeds of $22.6 million, after deducting fees and offering expenses, in "at the market" offerings and under its structured equity line with Lincoln Park Capital Fund, LLC.
Fourth Quarter and Full Year 2020 Highlights

Initiated healthy volunteer study to support long-term clinical development strategy for ALRN-6924. In November 2020, Aileron initiated a study of ALRN-6924 in healthy volunteers to gather data to support its goal to bring chemoprotection to patients with p53-mutated cancer who are undergoing chemotherapy regardless of type of cancer or chemotherapy. The ongoing study is designed to characterize the time to onset, magnitude, and duration of cell cycle arrest in human bone marrow relative to ALRN-6924 administration, with a goal of developing a universal dosing regimen for ALRN-6924 for use as a chemoprotection agent across a range of chemotherapies and p53-mutated tumor indications. Aileron plans to submit results from the healthy volunteer study for presentation at a medical conference in the second half of 2021.
Presented proof-of-concept data from Phase 1b SCLC trial of ALRN-6924 in late-breaking presentation at ENA 2020. In October 2020, Aileron presented new positive clinical data from its Phase 1b SCLC trial at the ENA 2020 symposium demonstrating clinical proof of concept that treatment with ALRN-6924 given 24 hours prior to second-line topotecan administration resulted in a protective effect against severe chemotherapy-induced bone marrow toxicities – anemia, thrombocytopenia and neutropenia – in patients with p53-mutated SCLC. The most robust and clinically meaningful protection against toxicities was observed with the 0.3 mg/kg dose of ALRN-6924.
Fourth Quarter and Full Year 2020 Financial Results

Cash Position: Cash, cash equivalents and investments as of December 31, 2020 were $13.8 million, compared to $18.3 million as of December 31, 2019. The company expects, based on its current operating plan, that its existing cash, cash equivalents and investments will fund operations into the second half of 2023.

Research and Development (R&D) Expenses: R&D expenses for the fourth quarter of 2020 were $1.9 million, compared to $4.7 million for the fourth quarter of 2019. The decrease in R&D expenses was primarily due to the completion of the company’s anti-cancer trials in late 2019 and lower spend on the manufacture of ALRN-6924 and research related activities in 2020 as compared to 2019. R&D expenses for the full-year 2020 were $11.2 million, compared to $17.7 million for the prior year.

General and Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2020 were $2.3 million, compared to $2.6 million for the fourth quarter of 2019. The decrease in G&A expenses was largely due to lower spending on employees and facilities in 2020 as compared to 2019. G&A expenses for the full-year 2020 were $9.3 million, compared to $12.3 million for the prior year.

Net Loss: Net loss for the fourth quarter of 2020 was $4.9 million, compared to $7.2 million for the fourth quarter of 2019. Net loss for the full-year 2020 was $21.2 million, compared to a net loss of $29.4 million for the prior year. The basic and diluted net loss per share for the fourth quarter of 2020 was $0.12 compared to $0.26 for the fourth quarter of 2019. The basic and diluted net loss per share for the full-year 2020 was $0.61 compared to $1.20 for the full-year 2019.
(Press release, Aileron Therapeutics, MAR 24, 2021, View Source [SID1234577081])

On March 24, 2021 LAVA Therapeutics B.V. (Nasdaq: LVTX), a biotechnology company focused on applying its expertise in bispecific gamma-delta T cell engagers to transform cancer therapy, reported the pricing of its initial public offering of 6,700,000 common shares at a public offering price of $15.00 per share (Press release, Lava Therapeutics, MAR 24, 2021, View Source [SID1234577080]). In addition, LAVA has granted the underwriters a 30-day option to purchase up to an additional 1,005,000 common shares at the initial public offering price, less underwriting discounts and commissions. All of the shares are being offered by LAVA. The shares are expected to begin trading on the Nasdaq Global Select Market on March 25, 2021 under the ticker symbol "LVTX."

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Gross proceeds of the offering, before deducting underwriting discounts and commissions and other offering expenses payable by LAVA, are expected to be $100.5 million. The offering is expected to close on March 29, 2021, subject to the satisfaction of customary closing conditions.

J.P. Morgan, Jefferies and SVB Leerink are acting as joint book-running managers for the offering. Kempen & Co is acting as lead manager for the offering.

The registration statement relating to these securities became effective on March 24, 2021. The offering will be made only by means of a prospectus, copies of which may be obtained from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by email at [email protected]; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at (877) 821-7388 or by email at [email protected]; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6105, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On March 24, 2021 Seraph Research Institute (SRI) and Enochian BioSciences, Inc., a company focused on gene-modified cellular and immune therapies in infectious diseases and cancer, reported that a 36-year old patient with recurrent glioblastoma achieved complete remission for a period of 15 months (Press release, Enochian BioSciences, MAR 24, 2021, View Source [SID1234577077]). The important findings were published today, in the peer-reviewed American Journal of Case Reports View Source

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A complete remission response such as this is uncommon for this highly aggressive and deadly form of brain cancer.

In compliance with U.S. Food and Drug Administration guidance, SRI treated the patient with natural killer (NK) and dendritic cells (DC) from a relative who had a partial genetic mismatch. The DCs were loaded with a component of cytomegalovirus that is known to be present in more than 90 percent of glioblastomas.

Enochian BioSciences acquired the intellectual property for an approach that is similar to the one used for this successful clinical outcome. The novel, proprietary strategy includes genetic modification of DCs, potentially increasing the immune response and even more effectively attacking tumors. Enochian BioSciences is actively developing this enhanced immune technology, including proof-of-concept in vivo studies of the potential to clear tumors with a focus on pancreatic cancer. In the U.S. alone, more than 60,000 persons are diagnosed with pancreatic cancer, and more than 48,000 die from the disease each year. The treatment strategy also could be applicable to several solid tumors.

Dr. Anahid Jewett, a renowned tumor immunologist and cell and gene therapy investigator at UCLA who has developed a humanized mouse model of pancreatic cancer, said: "This exciting clinical finding, as well as many other directions for therapy which Enochian BioSciences is currently undertaking to develop related approaches, gives hope for the possibility of effective treatment for pancreatic cancer and other solid tumors in the future."

On March 24, 2021 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported financial results and recent corporate highlights for the full year ended December 31, 2020 (Press release, Mustang Bio, MAR 24, 2021, View Source [SID1234577076]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "We are excited by the progress across our cell and gene therapy programs in 2020. Notably, MB-106 (CD20-targeted, autologous CAR T cell therapy) data were presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") Annual Meeting which showed a favorable safety profile and clinical activity, with an 89% overall response rate and 44% complete response rate in patients with relapsed or refractory B-cell non-Hodgkin lymphomas who were treated with the modified cell manufacturing process. In August, we initiated an open-label, multicenter Phase 1/2 clinical trial to evaluate the safety and efficacy of MB-102 (CD123-targeted CAR T cell therapy) in patients with relapsed or refractory blastic plasmacytoid dendritic cell neoplasm ("BPDCN"). In October, initial Phase 1 data on MB-105, a PSCA-targeted CAR T cell therapy administered systemically to patients with PSCA-positive metastatic castration-resistant prostate cancer ("mCRPC"), demonstrated a 94% reduction in prostate-specific antigen, near complete reduction of measurable soft tissue metastasis by computerized tomography, and improvement in bone metastases by magnetic resonance imaging in a 73-year-old male patient with PSCA-positive mCRPC who failed eight prior therapies."

Dr. Litchman continued, "In 2021, we anticipate multiple potential data disclosures from our collaborators’ clinical trials, and we plan to have four open Mustang Investigational New Drug ("IND") applications. We look forward to advancing our lentiviral gene therapy clinical program for the treatment of X-linked severe combined immunodeficiency ("XSCID"), also known as bubble boy disease. In the second quarter, we plan to begin enrollment on our pivotal multicenter Phase 2 trial of MB-107 in newly diagnosed infants with XSCID who are under the age of two and submit an IND application for a pivotal multicenter Phase 2 trial of MB-207 for the treatment of patients with XSCID who were previously treated with hematopoietic stem cell transplant ("HSCT") and for whom re-treatment is indicated. With a robust pipeline of therapies addressing highly challenging diseases, world-class R&D collaborators, a state-of-the-art cell processing facility and an experienced team, I believe we are very well positioned to continue building a fully integrated cell and gene therapy company."

Financial Results:

As of December 31, 2020, Mustang’s cash and cash equivalents and restricted cash totaled $98.8 million, compared to $62.4 million as of December 31, 2019, an increase of $36.4 million year-to-date.
Research and development expenses were $37.2 million for the year ended December 31, 2020. This compares to $30.0 million for 2019. Non-cash, stock-based compensation expenses included in research and development were $1.4 million for the year ended December 31, 2020, compared to $0.9 million for 2019.
Research and development expenses from license acquisitions totaled $10.1 million for the year ended December 31, 2020, compared to $6.3 million for 2019. Non-cash, stock-based compensation expenses included in research and development – licenses acquired were $7.6 million for the year ended December 31, 2020, compared to $4.9 million for 2019.
General and administrative expenses were $9.5 million for the year ended December 31, 2020. This compares to $9.6 million for 2019. Non-cash, stock-based compensation expenses included in general and administrative expenses were $4.0 million for the year ended December 31, 2020, compared to $3.4 million for 2019.
Net loss attributable to common stockholders was $60.0 million, or $1.14 per share, for the year ended December 31, 2020, compared to a net loss attributable to common stockholders of $46.4 million, or $1.29 per share, for 2019.
2020 and Recent Corporate Highlights:

In February 2020, Mustang announced that the first subject treated with the modified MB-106 (CD20-targeted, autologous CAR T cell therapy) manufacturing process, developed in collaboration between Mustang Bio and the Fred Hutchinson Cancer Research Center ("Fred Hutch"), achieved a complete response at the lowest starting dose in an ongoing Phase 1/2 clinical trial. The trial is evaluating the safety and efficacy of MB-106 in subjects with relapsed or refractory B-cell non-Hodgkin lymphomas and chronic lymphocytic leukemia.
In April 2020, Mustang announced that the European Medicines Agency ("EMA") granted Advanced Therapy Medicinal Product ("ATMP") classification to MB-107, a lentiviral gene therapy for the treatment of newly diagnosed infants with XSCID.
In May 2020, Mustang submitted an IND with the U.S. Food and Drug Administration ("FDA") to initiate a pivotal multicenter Phase 2 trial of MB-107 in newly diagnosed infants with XSCID who are under the age of two. The trial is expected to enroll 10 patients who, together with 15 patients enrolled in the current multicenter trial led by St. Jude Children’s Research Hospital, will be compared with 25 matched historical control patients who have undergone HSCT. The primary efficacy endpoint will be event-free survival.
Also in May 2020, City of Hope presented two posters pertaining to MB-104, an innovative CS1 CAR T cell therapy, at the virtual 23rd Annual Meeting of the American Society of Gene & Cell Therapy.
In June 2020, Mustang raised gross proceeds of approximately $37.2 million in an underwritten public offering of common stock, including the exercise of the underwriter’s option.
In August 2020, Mustang announced that the FDA granted Rare Pediatric Disease Designations to MB-107 for the treatment of XSCID in newly diagnosed infants and to MB-207 for the treatment of XSCID in patients who were previously treated with HSCT and for whom re-treatment is indicated.
In September 2020, Mustang announced that the FDA granted Orphan Drug Designations to MB-107 for the treatment of XSCID in newly diagnosed infants and to MB-207 for the treatment of XSCID in patients who were previously treated with HSCT and for whom re-treatment is indicated.
In October 2020, Mustang announced that the first patient was dosed in a Mustang-sponsored, open-label, multicenter Phase 1/2 clinical trial to evaluate the safety and efficacy of MB-102 (CD123-targeted CAR T cell therapy) in patients with relapsed or refractory BPDCN.
Also in October 2020, Mustang announced that initial Phase 1 data on MB-105, a PSCA-targeted CAR T cell therapy administered systemically to patients with PSCA-positive mCRPC, were presented by City of Hope at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat. A 73-year-old male patient with PSCA-positive mCRPC was treated with MB-105 and lymphodepletion (a standard CAR T pre-conditioning regimen) after failing eight prior therapies. On day 28 of the patient’s treatment, MB-105 demonstrated a 94% reduction in prostate-specific antigen, near complete reduction of measurable soft tissue metastasis by computerized tomography, and improvement in bone metastases by magnetic resonance imaging.
Additionally, in October 2020, Mustang in-licensed LentiBOOST technology from SIRION Biotech GmbH for the development of MB-207.
In November 2020, Mustang signed an agreement with Minaris Regenerative Medicine GmbH to enable technology transfer and GMP clinical manufacturing of the MB-107 lentiviral gene therapy program for the treatment of XSCID in Europe.
Also in November 2020, Mustang announced that the European Commission issued a positive opinion on its application for Orphan Drug Designation for MB-107.
In December 2020, Mustang announced positive interim Phase 1/2 data on MB-106 for patients with relapsed or refractory B-cell non-Hodgkin lymphomas, which were presented at the 62nd ASH (Free ASH Whitepaper) Annual Meeting. The data demonstrated an extremely favorable safety profile and clinical activity with an 89% overall response rate and 44% complete response rate over 4 dose levels in 9 patients treated with the modified cell manufacturing process.
Also in December 2020, Mustang announced that a Phase 1 single-center, two-arm clinical trial was initiated to establish the safety and feasibility of administering MB-101 to patients with leptomeningeal brain tumors (e.g., glioblastoma, ependymoma or medulloblastoma).
In February 2021, Mustang announced encouraging MB-107 and MB-207 clinical updates from its investigator-IND XSCID trials, as well as additional consistent safety and efficacy data. On January 28, 2021, the FDA removed a CMC hold on the MB-107 Phase 2 clinical trial IND application after reviewing a comprehensive CMC package that was submitted by Mustang in late December 2020. The company expects to enroll the first patient in this pivotal multicenter trial in the second quarter of 2021 and is targeting topline data from the trial in the second half of 2022. The company also expects to file an IND in the second quarter of 2021 for its pivotal multicenter Phase 2 clinical trial of MB-207.