On August 12, 2021 A Buffalo-based biotechnology company reported that it has received a $2.2 million federal grant to begin clinical trials on a treatment for brain cancer using light therapy (Press release, Photolitec, AUG 12, 2021, View Source [SID1234587063]).

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Photolitec LLC, which spun off from Roswell Park Comprehensive Cancer Center in 2010, received the three-year grant from the National Institutes for Health through the Small Business Innovation Research (SBIR) program, along with approvals from the Food & Drug Administration to begin phase one human trials.

The trials will allow the company to use its Photobac compound in combination with photodynamic therapy (PDT) to treat glioblastoma, a type of brain cancer.

This next step in the drug development process follows orphan drug status approval for Photobac by the FDA in late 2017; and an initial SBIR grant of $1.9 million awarded earlier that year.

Ravindra Pandey, founder and chief scientific officer, is director of pharmaceutical chemistry at Roswell Park and first developed the compounds there in his lab. They were later licensed back to the company and have been further developed in collaboration with global partners AMI-Organics in India and HISUN Pharma in China.

With no approved treatments yet for these types of brain tumors and the recurrence rates, the orphan drug status will definitely help speed up the process, Pandey said. The process helps surgeons be more precise in attacking tumors with better specificity, he said.

"No treatment works except PDT and this compound can treat tumors not only with the margins that you want, but deep-seated tumors. So the chances are we’ll have a much better response," he said.

Scott Friedman, general counsel at Photolitec and chairman at Lippes Mathias Wexler Friedman LLP, said though the process is taking longer than initially projected, the company is making great progress.

"Ravi is continuing to make great progress," he said. "There are hoops that early-stage companies without necessarily hundreds of millions or billions of dollars are being asked to jump through, so it’s a challenging environment to succeed in and it does occasionally take more time than might be expected. But I believe great companies figure out how to move forward."

Though PDT is not new, Photolitec’s approach applies light to a photosensitizer, which destroys tumor cells when combined with the oxygen present in the cancer. The company’s Photobac allows for deeper penetration into the tumor, which also limits the impact on surrounding tissue and reduces long-term skin phototoxicity.

Plans call for submitting clinical protocols to Roswell Park’s review board for approval, with a goal of beginning trials soon thereafter. Pandey is hopeful that process will be hastened with the FDA approval already in hand.

"Generally what happens is we send it for IRB first and then FDA but in this case it was the other way around," he said. "We hope that will make it faster. We already have the drug formulations and we’re all ready to go."

Pandey said the new funding from NIH, plus a score of "outstanding" on the research by peer reviewers also helps boost the chances the drug will attract lots of attention from big pharma when it comes time to manufacture and market Photobac. Whether investors will keep manufacturing here in Western New York is another question.

"We’re waiting on phase one data first, and that could take a couple of years," he said.

On August 12, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported financial results and business highlights for the second quarter of 2021 (Press release, Hookipa Biotech, AUG 12, 2021, View Source [SID1234586380]).

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"During the second quarter, we gained considerable momentum in highlighting the promise of our novel arenaviral platform to redefine success in cancer immunotherapy. Our HB-200 program in advanced Human Papillomavirus 16-positive (‘HPV16+’) cancers was featured prominently at both AACR (Free AACR Whitepaper) and ASCO (Free ASCO Whitepaper), with compelling tumor antigen-specific T cell responses and tumor control that replicate pre-clinical results," said Joern Aldag, Chief Executive Officer at HOOKIPA. "We are focused on advancing our oncology and infectious disease programs through the second half of the year, as we aim to deliver first-in-class arenaviral immunotherapies that induce potent, targeted immune responses to fight or prevent serious disease."

Quarter Highlights

At the virtual American Association for Cancer Research (AACR) (Free AACR Whitepaper) (‘AACR’) Annual Meeting in April 2021, HOOKIPA presented positive preliminary Phase 1 immunogenicity data for HB-200 for the treatment of advanced HPV16+ cancers. These data demonstrated a robust increase in HPV16+-specific T cells, including an increase of up to 8% of antigen-specific circulating CD8+ T cells, after one dose of HB-201 or HB-202. Early HB-201 monotherapy data also highlighted immune system activation of increasing interferon-gamma and other immune stimulatory cytokines with a single dose.

In June, HOOKIPA reported positive Phase 1 data from the ongoing Phase 1/2 study of HB-200 for the treatment of advanced HPV16+ cancers. The clinical data, presented as an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (‘ASCO’) Annual Meeting, showed HB-200 is highly immunogenic, inducing unprecedented levels of activated, tumor antigen-specific CD8+ T cells (an average of 6 percent and up to 40 percent of the T cell pool). In addition, HB-201 monotherapy showed an 18 percent overall response rate and median progression-free survival of 3.45 months in heavily pre-treated head and neck cancer patients who progressed on standard of care, including checkpoint inhibitors.

HOOKIPA completed enrollment in the Phase 2 clinical trial of its prophylactic Cytomegalovirus (‘CMV’), vaccine candidate, HB-101; the last patient was enrolled in June 2021. We expect to report additional safety, immunogenicity, and efficacy data from evaluable patients in the second half of 2021, with final top-line data readout in the first half of 2023. The protocol requires a 12-month follow up after transplantation, which is typically two to three months after enrollment.

Jean-Charles Soria resigned as a Non-Executive Director of HOOKIPA’s Board of Directors to avoid any conflicts of interest following a decision to join Amgen as senior vice president and lead of the oncology therapeutic area.
Upcoming Milestones

Additional HB-201/HB-202 Phase 1/2 data in HPV16+ cancers and recommended Phase 2 dose for the HB-200 program in the fourth quarter of 2021
HB-101 CMV Phase 2 data in the second half of 2021
Advancing our HB-300 program to IND for the treatment of metastatic prostate cancer
HBV and HIV collaboration with Gilead Sciences advancing towards clinical studies
Second Quarter 2021 Financial Results

Cash Position: HOOKIPA’s cash, cash equivalents and restricted cash as of June 30, 2021 was $102.9 million compared to $143.2 million as of December 31, 2020. The decrease was primarily attributable to cash used in operating activities.

Revenue was $5.4 million for the three months ended June 30, 2021, and $6.7 million for the three months ended June 30, 2020. The decrease was primarily due to receipt of a $1.0 million milestone payment under the Gilead collaboration during this time period in 2020, but not in 2021.

Research and Development Expenses: HOOKIPA’s research and development expenses were $19.6 million for the three months ended June 30, 2021, compared to $11.6 million for the three months ended June 30, 2020.

The primary drivers of the increase in direct research expenses were an increase in manufacturing and quality control expenses of $1.6 million, and an increase in clinical operations expenses of $1.4 million, along with a general increase in other direct research and development expenses and laboratory expenses of $2.4 million. These expenses were mainly due to the progress in our HB-201 and HB-202 clinical trial, in particular for monitoring and testing activities, and manufacturing and quality control work in preparation of a further extension of the trial. Manufacturing and quality control expenses were also driven by the progress towards clinical development in our Gilead-partnered programs.

Internal research and development expenses increased by $2.6 million, mainly due to our increased research and development headcount.

General and Administrative Expenses: General and administrative expenses for the three months ended June 30, 2021 were $5.1 million, compared to $4.3 million for the three months ended June 30, 2020. The increase was primarily due to an increase in personnel-related expenses, partially offset by a decrease in professional and consulting fees. The increase in personnel-related expenses resulted from a growth in headcount along with increased salaries in our general and administrative functions, and increased stock compensation expenses.

Net Loss: HOOKIPA’s net loss was $17.2 million for the three months ended June 30, 2021, compared to a net loss of $7.1 million for the three months ended June 30, 2020. This increase was due to an increase in research and development expenses, an increase in general and administrative expenses, a decrease in revenues from collaboration and licensing and a decrease in other income, partially offset by an increase in grant income.

On August 12, 2021 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage oncology company developing potentially first-in-class therapeutics that combine both targeted and immune-mediated mechanisms, reported financial results and provided a corporate update for the second quarter ended June 30, 2021 (Press release, Tempest Therapeutics, AUG 12, 2021, View Source [SID1234586426]).

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"The second quarter of 2021 was an exciting period as Tempest became a public company and the team drove progress in all three of our novel programs," said Steve Brady, chief executive officer of Tempest. "We look forward to the planned opening of the TPST-1120 randomized study in first line hepatocellular carcinoma in collaboration with Roche and the first combination study of TPST-1495, and remain focused on delivering potentially value-creating milestones over the next year and beyond."

Recent Highlights

Public Company Transition: successfully closed merger and concurrent PIPE financing, allowing Tempest to become a public company listed on the Nasdaq Capital Market, and extending runway into 2023 through multiple potential catalysts.
TPST-1495 (clinical dual EP2/4 prostaglandin receptor antagonist): continued enrollment in monotherapy dose optimization towards recommended Phase 2 dose ("RP2D").
TPST-1120 (clinical PPARα antagonist): (i) completed monotherapy dose escalation and selected 600mg BID as RP2D; (ii) observed stable disease ("SD") in 50% of the monotherapy-treated patients, including prolonged SD in patients with refractory cholangiocarcinoma; and (iii) observed a deep, confirmed partial response in a patient with checkpoint inhibitor-refractory fourth line renal cell carcinoma in the combination study with nivolumab (->60% by RECIST 1.1, durable through 4 scans and ongoing).
TREX-1 Inhibitor (preclinical, tumor-selective STING pathway activator): (i) progressed lead series to picomolar IC50 potency in biochemical assays; and (ii) demonstrated significant proof of concept in a mouse tumor model with systemic delivery of a lead series molecule.
Board of Directors: Christine Pellizzari, J.D., Geoff Nichol, M.B., Ch.B., M.B.A., and Ronit Simantov, M.D., joined the Board of Directors, bringing deeper financial, legal, and clinical development expertise to Tempest.
Planned Near-Term Milestones

TPST-1495 (clinical dual EP2/4 prostaglandin receptor antagonist): (i) selection of monotherapy RP2D expected in the first half of 2022; (ii) commencement of a combination study with an anti-PD-1 checkpoint inhibitor expected prior to the end of 2021; and (iii) commencement of monotherapy expansion in targeted indications and biomarker-selected patient populations expected in the first half of 2022.
TPST-1120 (clinical PPARα antagonist): (i) identification of RP2D of TPST-1120 in combination with nivolumab expected prior to the end of 2021; and (ii) commencement of first line randomized Phase 1b/2 study in hepatocellular carcinoma patients, under a collaboration with F. Hoffman La Roche, expected within the third quarter.
TREX-1 Inhibitor (preclinical tumor-selective STING pathway activator): planned selection of development candidate in the first half of 2022.
Financial Results

Second Quarter

Tempest ended the second quarter of 2021 with $68.5 million in cash and cash equivalents and short-term restricted cash, compared to $18.8 million in December 31, 2020. The increase was primarily due to the merger and concurrent PIPE, which closed in June 2021.
Net loss and net loss per share for the second quarter of 2021 were $7.1 million and $7.63, respectively, compared to $5.2 million and $11.42, respectively, for the second quarter of 2020. The increase was primarily due to an increase in compensation expense and professional fees associated with the merger.
Research and development expenses for the second quarter of 2021 were $4.2 million, compared to $4.1 million for the same period in 2020. The $0.1 million increase was primarily attributable to increased compensation expenses.
For the three months ended June 30, 2021, general and administrative expenses were $2.6 million compared to $1.1 million for the same period in 2020. The increase was primarily due to growth in compensation expense and professional fees associated with the merger.
Year-to-Date

Net cash used in operations for the six months ended June 30, 2021 was $6.2 million.
Net loss and net loss per share for the six months ended June 30, 2021 were $12.4 million and $17.30, respectively, compared to $9.5 million and $21.28, respectively, for the same period in 2020.
Research and development expenses for the six months ended June 30, 2021 were $7.8 million compared to $7.1 million for the same period in 2020. The $0.7 million increase was primarily due to increased compensation expenses and consulting services.
For the six months ended June 30, 2021, general and administrative expenses were $4.1 million compared to $2.4 million for the same period in 2020.

On August 12, 2021 Bolt Biotherapeutics, Inc. (NASDAQ: BOLT), a clinical-stage biotechnology company pioneering a new class of immuno-oncology agents that combine the targeting precision of antibodies with the power of both the innate and adaptive immune systems, reported financial results for the second quarter ended June 30, 2021 and provided an update on recent business highlights (Press release, Bolt Biotherapeutics, AUG 12, 2021, View Source [SID1234586442]).

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"We continue to build strong momentum with our business strategy and remain on target for a BDC-1001 Phase 1/2 clinical data update later this year," said Randall C. Schatzman, Ph.D., Chief Executive Officer of Bolt. "Our recently announced Genmab collaboration expands our proprietary Boltbody platform into novel bispecific ISAC applications, while fortifying our strong cash position. Furthermore, our CEA-targeted candidate BDC-2034 made steady progress towards an IND filing that is expected next year. I am proud of the passionate and experienced team we have assembled at Bolt, including recent additions to our leadership, who share our commitment to advancing targeted immuno-oncology therapies that will benefit patients with cancer."

Recent Business Highlights and Anticipated Milestones

Lead program BDC-1001 on track for anticipated Phase 1/2 trial data update in 2H21 – In June 2021, Bolt presented a poster at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting that expanded on the preliminary data, as of January 29, 2021, from the first 20 patients in an ongoing BDC-1001 Phase 1/2 clinical trial. The monotherapy dose-escalation portion of the trial is proceeding on plan, with a further data update expected in the second half of 2021. This Phase 1/2 trial is being conducted in four parts: [1] Phase 1 monotherapy dose escalation, [2] Phase 1 dose escalation in combination with PD-1 checkpoint inhibitor, [3] Phase 2 monotherapy expansion cohorts, and [4] Phase 2 expansion cohorts in combination with a PD-1 checkpoint inhibitor. Bolt also remains on track to initiate the monotherapy Phase 2 dose-expansion cohorts and the dose-escalation of BDC-1001 in combination with an anti-PD-1 antibody in the second half of 2021.

Announced oncology research and development (R&D) collaboration with Genmab to develop multiple bispecific ISACs – In June 2021, Bolt announced an oncology R&D collaboration with Genmab to discover and evaluate novel bispecific immune-stimulating antibody conjugate (ISAC) products for the treatment of multiple types of cancer. The collaboration will combine Bolt’s BoltBody ISAC platform with Genmab’s proprietary antibodies and bispecific technology, and Genmab will fully fund three programs through initial clinical proof-of-concept. Bolt received a $10 million USD upfront payment and a $15 million USD equity investment from Genmab, is eligible to receive up to $285 million USD for each program exclusively developed and commercialized by Genmab, and has the option to participate in the development and commercialization of one candidate after seeing clinical proof-of-concept data.

Expanded leadership team, adding expertise across research, clinical development, regulatory, quality and technical operations over the last year.
Amreen Husain, M.D., Vice President, Clinical Development and Translational Medicine. Dr. Husain brings more than a decade of experience in oncology drug development with a focus on breast and gynecological cancers and immuno-oncology. Dr. Husain joined Bolt from Roche/Genentech. Prior, Dr. Husain was as a practicing oncologist and clinical researcher at Stanford University Medical Center.
Bruce Hug, M.D., Ph.D., Vice President, Early Development and Research Collaborations. Dr. Hug joins Bolt from GlaxoSmithKline, bringing more than 16 years of oncology, hematology and immunotherapy experience, with a focus on early development.
Karen L. Bergman, Vice President, Communications and Investor Relations. Ms. Bergman has more than two decades of experience in biopharma communications, spanning corporate roles at companies such as ALZA and FibroGen, and 15 years heading a life science practice specializing in strategy, positioning, communications, and investor relations.
Liang Fang, Ph.D., Vice President, Biometrics and Bioinformatics. Dr. Fang brings more than 15 years of experience in developing and applying statistical methods and data sciences to drug development in oncology and the biotechnology industry from MyoKardia, Gilead Sciences, Genentech, and Amgen.
Triona O’Hanlon, Vice President, Program Management. Ms. O’Hanlon joined Bolt from Gilead, where she led program and portfolio management for hematology/oncology and cell therapy. Ms. O’Hanlon brings more than 20 years of experience in program and alliance management from Gilead Sciences, Kite Pharma, Elan Pharmaceuticals, and Ambit Biosciences.
Wesley Burwell, Vice President, Head of Human Resources. Mr. Burwell most recently worked at Global Blood Therapeutics and brings more than 20 years of experience building and driving HR strategy for biopharma companies.

Cash, cash equivalents, and marketable securities were $310.9 million as of June 30, 2021, which is expected to fund operations and the advancement of its oncology product pipeline to achieve multiple key milestones through the end of 2023.
Upcoming Events

Bolt Biotherapeutics will be attending the following conferences in September 2021:
Citi’s 16th Annual Biopharma Virtual Conference, September 8-10
Wells Fargo Virtual Healthcare Conference, September 9-10
Morgan Stanley Global Healthcare Conference, September 9-15
Cantor Fitzgerald Global Healthcare Conference, September 27-30
Second Quarter 2021 Financial Results

Cash Position – Cash, cash equivalents, and marketable securities were $310.9 million as of June 30, 2021, compared to $302.9 million as of March 31, 2021. Bolt expects its cash balance to fund operations through the end of 2023.

Research and Development Expenses – R&D expenses were $19.7 million for the quarter ended June 30, 2021, compared to $9.2 million for the same quarter in 2020, primarily due to increases in manufacturing expenses related to BDC-1001 and BDC-2034, increased personnel expenses relating to an increase in headcount, increased facility-related expenses, and increased clinical trial expenses.

General and Administrative (G&A) Expenses – G&A expenses were $4.1 million for the quarter ended June 30, 2021, compared to $2.0 million for the same quarter in 2020, primarily due to increased personnel expenses relating to an increase in headcount and increased professional services expenses related to consulting services, legal fees and other professional services.

Loss from Operations – Loss from operations was $23.8 million for the quarter ended June 30, 2021 compared to $11.1 million for the same quarter in 2020.

On August 12, 2021 Nimbus Therapeutics, a biotechnology company designing breakthrough medicines through structure-based drug discovery and development, reported the extension of its collaboration with Schrödinger, Inc. to discover and design small molecule therapeutics for a number of high-value targets using cutting-edge structure-based drug design approaches (Press release, Nimbus Therapeutics, AUG 12, 2021, View Source [SID1234586461]).

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The agreement extends the collaboration between the two companies, which began in 2009, to advance the usage of computational methods in drug discovery. Since then, the collaboration has generated numerous promising therapeutic candidates, including small molecule inhibitors of acetyl–CoA carboxylase (ACC) and tyrosine kinase 2 (TYK2), which are now in the clinic, and a hematopoietic protein kinase 1 (MAP4K1/HPK1) inhibitor, which Nimbus plans to advance to the clinic later this year.

"Nimbus’ collaboration with Schrödinger over the past 12 years has been groundbreaking, deploying computational horsepower at an unprecedented scale in the industry," said Jeb Keiper, M.S., MBA, Chief Executive Officer of Nimbus. "Schrödinger’s deep computational capabilities, paired with Nimbus’ demonstrated expertise in structural biology, small molecule drug discovery and clinical development, provides fertile ground for designing breakthrough medicines over the next decade."

"We are very pleased to embark on this new chapter of Schrödinger’s collaboration with Nimbus and to further expand the frontiers of combining physics-based drug discovery with machine learning," said Ramy Farid, Ph.D., Chief Executive Officer at Schrödinger. "Our collaborative track record underscores the potential of our combined multidisciplinary drug discovery team to design medicines that could provide meaningful benefits for patients."