panCELLA receives research and development support for cell therapy

On April 7, 2020 panCELLa reported that it will receive advisory services and up to $400,000 from the National Research Council of Canada Industrial Research Assistance Program (NRC IRAP) to help further research and development of its technology platforms for cell therapy (Press release, panCELLa, APR 7, 2020, View Source [SID1234556171]).

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Support from NRC IRAP will go toward the research and development of a two-step safety switch known as panKiller. While panCELLa’s safety switch, known as FailSafeTM, is specifically designed to eliminate only "rogue" or deregulated therapeutic cells, the panKiller system further enhances this platform and provides a means to remove a graft entirely once it has fulfilled its therapeutic purpose. When the therapeutic cells are no longer required, and to avoid serious adverse events, for further safety measures they would be eliminated using panKiller. The panKiller strategy is an effective solution that addresses serious limitations of current cell therapies which involve removal of these therapeutic cells through irradiation. panKiller will enable more prolonged, effective treatment in vivo; and will destroy the therapeutic cells at the appropriate time without the use of radiation to prevent the possibility of a malignancy.

Ascentage Pharma Announces Approval for the Phase Ib/II Clinical Study of Bcl-2 Inhibitor APG-2575 for the Treatment of Patients With Relapsed/Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma in China

On April 7, 2020 Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that it has received approval from the Center for Drug Evaluation of the China National Medical Products Administration for the Phase Ib/II study of APG-2575, the company’s novel Bcl-2‒selective inhibitor, for the treatment of patients with relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (r/r CLL/SLL) (Press release, Ascentage Pharma, APR 7, 2020, View Source;small-lymphocytic-lymphoma-in-china-301036549.html [SID1234556188]).

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This open-label, Phase Ib/II dose-escalation and dose-expansion study is designed to evaluate the safety, tolerability, and preliminary anticancer activity of APG-2575 as a single agent or in combination therapies for the treatment of patients with r/r CLL/SLL in China.

APG-2575 is a novel, orally administered Bcl-2‒selective inhibitor being developed by Ascentage Pharma. APG-2575 is designed to treat several hematologic malignancies by selectively blocking Bcl-2 to restore the normal apoptosis process in cancer cells.

Recent studies have demonstrated that Bcl-2 inhibitors are effective in CLL/SLL, and Bcl-2 inhibitors in combination with Bruton tyrosine kinase inhibitors or CD20 monoclonal antibodies can deepen patient responses and prolong patient survival. These findings provide the rationale for investigating APG-2525 as a single agent or in combination therapies. The Phase I study of APG-2575 monotherapy, previously commenced in the US, Australia, and China, has shown a favorable safety profile and promising anticancer activity, and the preliminary data support the continued clinical investigation of APG-2575.

CLL/SLL is a hematologic malignancy caused by mature B-cell neoplasms, and it mainly affects middle-aged and elderly populations. The condition is characterized by accumulation of cancerous lymphocytes in peripheral blood, bone marrow, spleen, and lymph nodes. CLL/SLL is the most common type of adult leukemia in North America and Europe but is less common in Asian countries such as China and Japan. However, with an aging population, as well as dietary and lifestyle changes in China, CLL/SLL incidences have been increasing, and more patients develop the condition at younger ages and display greater disease progression. Despite significant initial responses to current first-line treatments, many patients with CLL/SLL need continued treatment to maintain these responses, and relapse often portends a poor prognosis. At present, there are even fewer treatment options for patients with CLL/SLL in China than in Europe or North America, hence representing an urgent clinical need for more effective and safe therapies.

"APG-2575 is a key drug candidate in our development pipeline targeting apoptosis, and the first China-made Bcl-2‒selective inhibitor to enter clinical studies, with great potential as a monotherapy and in combination therapies in several hematologic malignancies. Upon receiving recent approvals and clearances for several Phase Ib/II studies of APG-2575 in China, Australia, and the US, we are now advancing its clinical development globally," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "There is an enormous unmet clinical need in the treatment of CLL/SLL globally. We will soon initiate this Phase Ib/II study of APG-2575 in China, which hopefully will provide a new treatment option to patients with CLL/SLL."

Amphista Therapeutics Raises $7.5m Series A Round to Advance Targeted Protein Degradation Assets in Cancer

On April 7, 2020 Amphista Therapeutics, a biopharmaceutical company creating first-in-class cancer therapeutics that harness the body’s natural processes to selectively and efficiently degrade and remove disease causing proteins, reported the closing of a USD $7.5m Series A round, led by Advent Life Sciences (Press release, Amphista Therapeutics, APR 7, 2020, View Source [SID1234556189]). Seed round funders the Scottish Investment Bank, with backing from the Scottish Growth Scheme, and the European Investment Fund joined the round, along with new investor, US-based life sciences BioMotiv.

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Amphista’s CEO Nicola Thompson said, "This international financing provides Amphista with a firm foundation to underpin a Series B round to progress our oncology pipeline to the clinic. Our vision is to create a leading protein degradation company on the global stage that delivers ground-breaking new medicines to patients in areas of high unmet need."

Raj Parekh, General Partner at lead investor Advent Life Sciences said, "We are excited to support Amphista in its next stage of development. The Company has a potentially unique approach to targeted protein degradation when compared with traditional proteolysis targeting chimera (PROTAC) platforms. We believe that Amphista has great potential with its differentiated proprietary technology to address traditionally undruggable targets."

Satish Jindal, CEO of BioMotiv, and newly appointed Amphista Chairman, commented, "We are a mission-driven accelerator, and we are excited by Amphista’s focus to rapidly produce potent bifunctional small molecules to augment the body’s own processes to remove disease-associated proteins. We see huge potential to accelerate Amphista’s breakthrough technology platform into medicines."

Amphista’s scientific founder, Professor Alessio Ciulli, based at the University of Dundee, is an internationally renowned expert in the field of targeted protein degradation (TPD). "Highly specific TPD is a transformative new modality for tackling previously undruggable targets with high therapeutic value," said Ciulli.

Amphista’s TPD small molecules instruct the cell to degrade the target directly rather than activating or inhibiting the target protein function. As protein-protein interactions are involved in disease progression, removing the target protein provides a clear therapeutic advantage over simple inhibition. Specifically, Amphista’s platform is independent of traditional E3 ubiquitin ligases used by the field, potentially expanding the available target scope of TPD approaches and should overcome recently identified PROTAC resistance mechanisms.

Mersana Therapeutics Raises $65 Million in Gross Proceeds Through its At-the-Market Facility

On April 7, 2020 Mersana Therapeutics, Inc. (Nasdaq: MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported that it has raised gross proceeds of approximately $65 million through its At-the-Market (ATM) facility with participation based on interest received from Avoro Capital Advisors LLC, Bain Capital Life Sciences, Consonance Capital Investors and David Mott, Mersana’s Chairman of the Board (Press release, Mersana Therapeutics, APR 7, 2020, View Source [SID1234556208]). The Company sold approximately 8.9 million shares of the Company’s common stock at a purchase price of $5.59 and 2.0 million shares at the closing price of $7.74, in each case the market price at the time of sale. Cowen is acting as the sales agent for the ATM facility.

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The additional funds raised through the ATM strengthen the Company’s balance sheet and will be used to advance its pipeline, including the clinical development of XMT-1536 and XMT-1592, as well as for working capital and other general corporate purposes.

The shares of common stock described above were sold by the Company pursuant to a shelf registration statement on Form S-3 (File No. 333-226055), including a preliminary prospectus supplement, which was declared effective by the SEC on September 17, 2018. A final prospectus supplement will be filed with the SEC. Copies of the final prospectus supplement and the accompanying prospectus may be obtained, when available, from Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY, 11717, Attn: Prospectus Department, by email at [email protected] or by telephone at (833) 297-2926. Electronic copies of the final prospectus supplement and the accompanying prospectus will also be available on the SEC’s website at View Source

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

Bicycle Therapeutics Announces Pipeline Progress Update

On April 7, 2020 Bicycle Therapeutics plc (NASDAQ: BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported progress updates across its wholly-owned and partnered programs in oncology and non-oncology indications (Press release, Bicycle Therapeutics, APR 7, 2020, View Source [SID1234556174]).

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"We and our partners have recently achieved important progress in the advancement of our pipeline that we believe could catalyze the next stage of growth for Bicycle," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "Cancer Research UK has identified a recommended dose, which is within the therapeutic range predicted by preclinical models, for the Phase IIa study of BT1718, which we expect to commence this year. Our next-generation Bicycle Toxin Conjugates (BTCs), BT5528 and BT8009, are quickly progressing through respective stages of development. In addition, we believe our recently announced early discovery collaboration with Genentech will allow us to make considerable investments in expanding our portfolio of immuno-oncology assets, including our wholly-owned programs nearing late preclinical development. Beyond oncology, we continue to make progress on our partnered programs and to execute on our strategy of leveraging our collaborators’ expertise in specific therapeutic areas to advance the development of Bicycle-based medicines across a broad range of serious diseases."

Dr. Lee continued: "While we expect that the evolving COVID-19 pandemic may impact the pace of clinical development, at this time, we believe the strong progress we’ve achieved across our pipeline to date will enable us to reach our anticipated 2020 milestones as planned."

BT1718, a potential first-in-class BTC targeting key tumor antigen MT1-MMP

Cancer Research UK Completed Phase I Dose Escalation of BT1718 in Patients with Solid Tumors, Established Recommended Phase II Dose (RP2D) at 20 mg/m2. The key objectives were met in a Phase I dose escalation sponsored by Cancer Research UK and evaluating safety and tolerability of BT1718 in an unselected group of patients with advanced solid tumors. Based on study results, Cancer Research UK established an RP2D for the expansion cohorts at 20 mg/m2 administered once weekly. This recommended dose is within the efficacious dose range predicted by preclinical models, in which an equivalent dose level was associated with complete responses. With once-weekly dosing, BT1718 appeared tolerable, with manageable adverse events.

Though not a key objective of the Phase I portion of the Phase I/IIa study, preliminary signs of anti-tumor activity were observed during the dose escalation. As reported at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Annual Congress, 13 of 24 evaluable patients (54%) had stable disease at the eight-week timepoint, including a patient who experienced a 45% reduction in a target lesion, with findings generally remaining consistent as the trial progressed. Today, Bicycle announced that, in addition, one patient with small cell lung cancer experienced a partial response, with a 68% reduction in a target lesion.

The Company anticipates that Cancer Research UK will initiate the Phase IIa portion of the Phase I/IIa study of BT1718 in 2020, although timing may be dependent on the ongoing COVID-19 pandemic. These expansion cohorts will include patients determined to be MT1-MMP-positive based on a prespecified tumor membrane H-score. Initially, patients will be enrolled into two expansion cohorts, one in squamous non-small cell lung cancer (NSCLC) and the other in an all-comers "basket" cohort. Depending on results from these first two cohorts, additional cohorts may be initiated.
BT5528, a BTC targeting EphA2, a potentially high-value target for which antibody-based approaches have been unsuccessful

Doses of BT5528 Administered to Date Appear Safe, Well Tolerated in Ongoing Phase I/II Trial. The second cohort of patients has completed dosing in the Phase I dose escalation portion of a Phase I/II study of BT5528 in patients with advanced solid tumors associated with EphA2 expression. Unlike previous antibody drug conjugate (ADC) programs to target EphA2, BT5528 did not show coagulopathy in preclinical studies. Initial doses of BT5528 administered in the Phase I portion of the study are estimated to deliver six to 12 times the amount of toxin that was delivered by an ADC targeting EphA2 in an unsuccessful clinical trial and, to date, BT5528 has appeared well tolerated with manageable adverse events.

The Phase I/II multi-center, open-label trial is evaluating BT5528 administered once-weekly as a single agent and in a lagging cohort in combination with nivolumab. The Phase I portion is a dose escalation study primarily designed to assess the safety and tolerability of BT5528 and to determine an RP2D. Bicycle expects the first patient in the combination arm will be dosed in 2020, subject to potential timing and other impacts of the ongoing COVID-19 pandemic.
BT8009, a Nectin-4 targeting BTC with a potentially differentiated profile to marketed ADC

Phase I/II Trial of BT8009 Currently On Track to Initiate in 2020. In preclinical studies, BT8009 demonstrated highly target-dependent and improved anti-tumor activity over comparator Nectin-4-targeting ADCs. Bicycle believes the characteristics of BT8009 may result in a favorable safety profile and could circumvent certain challenges in treating cancers believed to be associated with Nectin-4 expression that are not addressed by current ADC approaches. The Company plans to initiate a Phase I/II study of BT8009 in patients with advanced solid tumors in indications associated with Nectin-4 expression this year, subject to potential timing and other impacts of the ongoing COVID-19 pandemic.
Novel, fully synthetic Bicycle systemic immune cell agonists and tumor-targeted immune cell agonists (TICAs)

IND Preparation for BT7480 On Track, Enabling Potential Clinical Start in 2021. BT7480 is a novel, fully synthetic TICA that contains two Bicycles, one targeting Nectin-4 and a second agonizing CD137, that has been shown in preclinical models to rapidly penetrate tumors, effect anti-tumor activity, and facilitate immune memory. IND-enabling activities for BT7480 are ongoing and remain on track to enable the initiation of clinical development in 2021, subject to potential timing and other impacts of the ongoing COVID-19 pandemic.
Bicycle Expands IO Pipeline, Selecting BT7455 as New TICA Candidate. BT7455 is a novel, fully synthetic TICA containing two Bicycle arms, one targeting EphA2 and the other agonizing CD137. EphA2 is highly expressed in a number of tumor types of high unmet medical need. In preclinical models, BT7455 exhibits highly potent EphA2-dependent stimulation of CD137 and robust in vivo anti-tumor activity against EphA2 expressing tumors.
Cancer Research UK Advancing Preclinical Development of BT7401. BT7401 is a multivalent, systemic immune cell agonist of CD137 built from multiple CD137 monomeric Bicycles connected by stable linkers through a central hinge. In 2020, Bicycle announced a second collaboration with Cancer Research UK, in which the organization will fund and sponsor development of BT7401 through a Phase I/IIa clinical study. Preclinical development of BT7401 funded by Cancer Research UK is ongoing.
Partnered programs beyond oncology

Oxurion Preparing to Initiate Phase II Trial of Kallikrein Inhibitor Bicycle, THR-149, in Patients with Diabetic Macular Edema (DME). Positive results from the Phase I clinical trial in patients with DME evaluating the safety and tolerability of THR-149, a novel Bicycle plasma kallikrein (PKal) inhibitor, were announced in 2019. The Phase I data demonstrate that a single intravitreal injection of THR-149 resulted in increasing average improvements in best corrected visual acuity (BCVA) starting at Day 1, which were up to 7.5 letters at Day 14 and maintained through Day 90. No dose-limiting toxicities or drug-related adverse events were reported.

Oxurion is preparing to initiate a Phase II trial evaluating multiple doses of THR-149 in DME patients who respond sub-optimally to anti-VEGF therapy.
Three Target Programs Transitioned to AstraZeneca. Under the terms of the collaboration, Bicycle is responsible for identifying Bicycles for an undisclosed number of respiratory, cardiovascular and metabolic disease targets specified by AstraZeneca. Three target programs have been transitioned to AstraZeneca for subsequent optimization towards potential candidate selection. Pursuant to the collaboration agreement, AstraZeneca is responsible for further development and product commercialization, and Bicycle is eligible for over $1 billion in future R&D funding, development, regulatory and commercialization milestone payments, as well as royalties on sales of products resulting from the partnership.
Early Success in Collaboration with Dementia Discovery Fund (DDF). Bicycle, in collaboration with DDF, is developing Bicycles to modulate the activity of proteins implicated in the progression of dementia. The Company has developed Bicycles that show successful binding to a target of interest, representing the first part of the first milestone of the collaboration.