On May 5, 2021 TILT Biotherapeutics, a clinical-stage biotechnology company developing cancer immunotherapeutics, reported that it has dosed its first patients and progressed to the second dose level in a monotherapy phase 1 clinical trial of TILT-123, a dual cytokine armed oncolytic adenovirus (Press release, TILT Biotherapeutics, MAY 5, 2021, View Source [SID1234579226]). The trial is being performed in patients with injectable solid tumors. Patients are being treated at Docrates Cancer Center and Helsinki University Hospital (Helsinki, Finland), leading international treatment facilities specializing in diagnostics, treatment and follow-up of cancers.

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The ‘TILT-T115’ clinical trial (1) is a phase 1, open-label, dose-escalation study of the company’s oncolytic adenovirus coding for Tumor Necrosis Factor Alpha (TNF alpha) and Interleukin 2 (IL-2), known as TILT-123. A total of approximately 20 patients will receive TILT-123 as a monotherapy over a three-month period. The trial’s primary objective is to evaluate the safety of TILT-123. The T115 trial is also designed to deliver insights about the behavior of TILT-123 in humans, such as systemic tumor transduction following intravenous delivery and virus replication in the tumor, as well as immunological responses.

TILT Biotherapeutics’ CEO, Akseli Hemminki, a biotech entrepreneur and cancer clinician who has personally treated almost 300 patients with ten different oncolytic viruses, said, "We are pleased by the clinical progress of TILT despite the recent restrictions caused by the pandemic. With our TILT-T215 melanoma combination trial already up and running in Denmark and France, I’m delighted that our TILT-T115 solid tumor monotherapy trial is now open in Finland, with interim results expected in Q4 2021. We expect the trial to provide valuable data in multiple solid tumor indications as well as provide supportive data for the future development of TILT-123 in combination with checkpoint inhibitors and CAR-T therapies."

Docrates Cancer Center’s CEO Ilpo Tolonen, said, "Docrates is the first and only private cancer center in the Nordic countries, and we have received patients from over 60 countries worldwide. We have harnessed our state-of-the-art technology and the uncompromising competence of our cancer treatment specialists for the best benefit of our patients – accompanied by genuine caring. This collaboration with TILT Biotherapeutics is part of our mission to provide our patients with state-of-the-art individual cancer therapies, cutting-edge know-how and, whenever possible, offer new treatments that are still in the experimental stage, for the benefit of the patient."

The heart of TILT’s approach revolves around the use of armed oncolytic adenoviruses, using cytokines to boost the patient’s immune response to better enable it to find and destroy cancer cells.

On May 5, 2021 vTv Therapeutics Inc. (Nasdaq:VTVT) reported financial results for the first quarter ended March 31, 2021, and provided an update on the progress of its clinical programs (Press release, vTv Therapeutics, MAY 5, 2021, View Source [SID1234579253]).

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"The vTv team continued to build on the clinical success of our type 1 diabetes program during the quarter by obtaining Breakthrough Therapy designation from the FDA for TTP399 and by initiating a phase 1 study to understand TTP399’s impact on ketoacidosis. The FDA’s grant of Breakthrough is an acknowledgment of the significant unmet need for patients with type 1 diabetes, and the encouraging clinical results we have generated to date," said Steve Holcombe, president and CEO, vTv Therapeutics. "We look forward to working closely with the FDA to optimize the development pathway for TTP399 to bring this potential treatment to patients with type 1 diabetes as quickly as possible."

Recent Achievements and Outlook

Type 1 Diabetes

Breakthrough Therapy Designation. As announced in April, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for TTP399 as an adjunctive therapy to insulin for the treatment of type 1 diabetes. Breakthrough Therapy designation is intended to expedite the development and review of a drug candidate that is planned for the treatment of a serious or life-threatening disease where initial clinical evidence indicates that the drug may demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoints.

Mechanistic Study of Ketoacidosis with TTP399. The Company began dosing patients in a mechanistic study of TTP399 in people with type 1 diabetes to determine the impact of TTP399 on ketone body formation during a period of acute insulin withdrawal. vTv expects to report topline results from the mechanistic study in the third quarter of 2021.

Pivotal Study Planning. The Company is planning to initiate the first of two pivotal, placebo-controlled, six-month clinical trials of TTP399 in subjects with type 1 diabetes in the fourth quarter of 2021. The current study designs will be shared with the FDA for its input and advice as part of the collaborative process enabled by the Breakthrough Therapy designation.
Psoriasis

Multiple Ascending Dose Study with HPP737. The Company began dosing healthy subjects in a phase 1 multiple ascending dose study to assess the safety, tolerability, and pharmacokinetic profile of HPP737, an oral PDE4 inhibitor. This phase 1 study is expected to report topline results in the third quarter and will inform dose selection for a planned phase 2 study in psoriasis.
License Partner Updates

Reneo Pharmaceuticals Completes Initial Public Offering. We congratulate our licensee, Reneo Pharmaceuticals, Inc. (Nasdaq: RPHM), for successfully completing an initial public offering in April. The Company is party to a license agreement with Reneo for its lead asset REN001, which Reneo is currently developing in three rare genetic diseases: primary mitochondrial myopathies, long-chain fatty acid oxidation disorders, and glycogen storage disease type V (McArdle disease).
First Quarter 2021 Financial Results

Cash Position: The Company’s cash position as of March 31, 2021, was $8.4 million compared to $5.7 million as of December 31, 2020.

Revenue: Revenue for the first quarter of 2021 was $1.0 million and was $6.4 million for the fourth quarter of 2020. The revenue for the first quarter was non-cash and related to the recognition of revenue pertaining to the Huadong license agreement. The revenue for the fourth quarter was primarily attributable to the upfront consideration, consisting of cash and an equity interest, received in connection with the Company’s license agreement with Anteris Bio.

R&D Expenses: Research and development expenses were $3.1 million and $2.5 million for the three months ended March 31, 2021 and December 31, 2020, respectively. This increase of $0.6 million was driven primarily by the increase in spending for our study of HPP737 in psoriasis.

G&A Expenses: General and administrative expenses were consistent between periods at $2.2 million and $2.0 million for the three months ended March 31, 2021 and December 31, 2020, respectively.

Net (Loss)/Income Before Non-Controlling Interest: Net loss before non-controlling interest was $5.9 million for the first quarter of 2021 compared to net income of $1.6 million for the fourth quarter of 2020.

Net (Loss)/Income Per Share: Diluted net loss per share was ($0.08) for the three months ended March 31, 2021 compared to a diluted net income per share of $0.02 for the three months ended December 31, 2020, based on weighted-average diluted shares of 56.5 million and 74.4 million for the three-month periods ended March 31, 2021 and December 31, 2020, respectively. Non-GAAP net loss per fully exchanged share was ($0.09) for the three months ended March 31, 2021 compared to a net income per fully exchanged share of $0.02 at December 31, 2020, based on non-GAAP fully exchanged weighted-average shares of 79.6 million and 74.4 million for the three months ended March 31, 2021 and December 31, 2020, respectively.

Merck & Co has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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On May 11, 2021Appia Bio, Inc., an early stage biotechnology company developing engineered allogeneic cell therapies from hematopoietic stem cells (HSCs) for cancer patients, reported its launch from stealth backed by $52 million Series A financing led by 8VC (Press release, Appia Bio, MAY 5, 2021, View Source [SID1234583854]). Other investors included Two Sigma Ventures, among others, and participation from seed investors Sherpa Healthcare Partners and Freeflow Ventures.

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Appia Bio is focused on discovering and developing off-the-shelf allogeneic cell therapies across a broad array of cancer indications, utilizing a scalable technology platform with the goal to increase access for patients. With its ACUA technology platform, Appia Bio leverages the biology of lymphocyte development with chimeric antigen receptor (CAR) and T-cell receptor (TCR) gene engineering to generate CAR-engineered invariant natural killer T (CAR-iNKT) cells from HSCs. The ACUA platform originated from groundbreaking research in the laboratory of Lili Yang, PhD, associate professor at University of California, Los Angeles (UCLA), and her collaborations with the Company’s other scientific founders.

Proceeds from the financing will support the advancement of Appia Bio’s pipeline of allogeneic CAR-iNKT cell therapy candidates into the clinic. As part of the Series A financing, Francisco Gimenez, PhD, partner, and David Moskowitz, PhD, principal, at 8VC, join the Company’s board of directors, and David Baltimore, PhD, of the California Institute of Technology (Caltech) was elected chairman of the Board.

"There is tremendous potential to be explored in allogeneic cell therapies. The Board and I look forward to working with this stellar team to create transformative cell therapies that will help patients and the physicians who treat them," said Dr. Baltimore.

"We are grateful to our new and existing investors for their commitment to bring off-the-shelf allogeneic cell therapies to more patients," said JJ Kang, PhD, co-founder and chief executive officer (CEO) of Appia Bio. "This financing will position us to rapidly build out our CAR-iNKT pipeline and establish the clinical potential of our ACUA platform. We are excited to work with an outstanding Scientific Advisory Board that brings together world-class expertise and experience across the many fields necessary to develop cell therapies."

Appia Bio announced the establishment of a Scientific Advisory Board with leaders in immunology, immuno-oncology, and cell therapy engineering. These experts join Appia Bio’s scientific founders in advising the Company’s research and development.

Mitchell Kronenberg, PhD, president and CSO, La Jolla Institute for Immunology

Antoni Ribas, MD, PhD, professor of medicine, UCLA, and director of the Tumor Immunology Program at the Jonsson Comprehensive Cancer Center

Margo Roberts, PhD, former CSO, Kite Pharma and Lyell Immunopharma

"We are thrilled to partner with Appia Bio as it typifies the innovative, founder-driven start-ups we love to empower," said Dr. Gimenez. "Appia unifies compelling biology of iNKT cells with the power of CARs. This is only possible because of their fundamental innovations in manufacturing and engineering these cells."

The Company was founded in 2020 in Los Angeles, California, by a team of experts and industry leaders with a proven track record of cell therapy development and company building.

David Baltimore, PhD, Nobel Laureate, president emeritus and distinguished professor of biology, Caltech

JJ Kang, PhD, CEO, Appia Bio; former partner, The Column Group

Edmund Kim, PhD, chief operating officer of Appia Bio; former vice president of corporate development, Kite Pharma

Pin Wang, PhD, professor of chemical engineering and materials science and biomedical engineering, University of Southern California (USC)

Jeff Wiezorek, MD, chief medical officer, Appia Bio; former head of cell therapy development, Kite Pharma

Lili Yang, PhD, associate professor of microbiology, immunology, and molecular genetics, UCLA

On May 5, 2021 CTI BioPharma Corp. (CTI BioPharma) (NASDAQ: CTIC) reported that management plans to report its first quarter 2021 financial results on Tuesday, June 1, 2021, after the close of the U.S. financial markets (Press release, CTI BioPharma, MAY 5, 2021, View Source [SID1234579147]). Following the announcement, members of the management team will host a conference call and webcast to discuss the results and provide a general corporate update at 4:30 p.m. ET (1:30 p.m. PT).

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To access the live call by phone please dial (877) 735-2860 (domestic) or (602) 563-8791 (international); the conference ID is 9343326. A live audio webcast of the event may also be accessed through the "Investors" section of CTI’s website at www.ctibiopharma.com. A replay of the webcast will be available for 30 days following the event.