Tarveda Therapeutics Publishes Preclinical Study Evaluating HSP90 Binding Miniature Drug Conjugate with Pan-PI3K Payload in Solid Tumors

On August 4, 2020 Tarveda Therapeutics, Inc., a clinical stage biopharmaceutical company developing a new class of potent and selective precision oncology medicines, which it refers to as Pentarin miniature drug conjugates, reported the publication of its preclinical study evaluating its HSP90 binding miniature drug conjugate with a pan-PI3K payload, T-2143, in solid tumors (Press release, Tarveda Therapeutics, AUG 4, 2020, View Source [SID1234562839]). The publication, titled "Novel Miniaturized Drug Conjugate Leverages HSP90 Driven Tumor Accumulation to Overcome PI3K Inhibitor Delivery Challenges to Solid Tumors," was published in Molecular Cancer Therapeutics, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper).

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"The PI3K pathway is one of the most frequently dysregulated signaling cascades in cancer and is implicated in a wide range of tumor types. Successful drug development of PI3K inhibitors has been very limited however, due to toxicity observed across numerous programs," said Mark Bilodeau, Ph.D., Chief Scientific Officer of Tarveda. "We have been able to leverage our HSP90 binding miniature drug conjugate platform to develop T-2143, designed to target a pan-PI3K inhibitor selectively to the activated HSP90 in solid tumors, and to mask the activity of the pan-PI3K inhibitor until it is released preferentially in the tumor, which further enhances the systemic tolerability of the payload. The results of the study published today confirm that T-2143 had superior efficacy to a non-targeted pan-PI3K inhibitor alone in a pre-clinical xenograft model, and that the tumor targeting and payload masking successfully reduced hyperglycemia, one of the most common dose-limiting toxicities in this class."

"The T-2143 conjugate design sets the stage for a potentially differentiated pan-PI3K inhibitor, with an improved efficacy and safety profile over existing pan-PI3K inhibitors alone," said Kapil Dhingra, M.D., a leading oncology therapeutics expert and member of the Tarveda Scientific Advisory Board. "The encouraging results of this preclinical study show that T-2143 was able to significantly inhibit tumor growth without the hyperglycemia commonly seen as a dose-limiting toxicity of inhibitors of PI3Ka including the pan-PI3K inhibitor class. If proven in human clinical trials, this approach has the potential to deliver improved efficacy of this important class of inhibitors, with applicability across a wide range of tumor types. As the second drug candidate from this novel platform, this data further supports the notion that HSP90 binding miniature conjugates can allow tumor specific accumulation and retention of a variety of payloads that may address unmet medical needs across a broad spectrum of tumor types, without the usual limitations encountered by antibody-drug conjugates."

The results of the study detail how our HSP90 targeted pan-PI3K inhibitor drug conjugate, T-2143, demonstrates rapid and sustained tumor accumulation of the conjugate, deep pathway inhibition, and superior efficacy in tumor xenograft models than the marketed pan-PI3K inhibitor, copanlisib, on its own. Data from the study show that by leveraging the preferential accumulation of HSP90 targeted conjugates and its payload in tumors, T-2143 can selectively target a pan-PI3K inhibitor, leading to efficacy in multiple xenograft tumor models. The selective targeting of T-2143 and the masking of the inhibitor active site enabled mitigation of hyperglycemia, a dose limiting side effect seen in pan-PI3K inhibitors, while driving higher tumor exposure and enhanced efficacy. The targeted drug conjugate strategy employed in the design of T-2143 is based on Tarveda’s HSP90 binding drug conjugate platform and shows the potential to provide new treatment options to patients with solid tumor malignancies through specific tumor targeting of anticancer payloads and the reduction of toxicity through selective tumor targeting as well as payload masking.

Aldeyra Therapeutics to Participate in Upcoming Virtual Investor Conferences

On August 5, 2020 Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) reported that senior management will be presenting and hosting 1×1 meetings at the following virtual investor conferences in August (Press release, Aldeyra Therapeutics, AUG 4, 2020, View Source [SID1234562838]):

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BTIG Virtual Biotechnology Conference 2020
Presentation Date: Tuesday, August 11, 2020
Time: 11:30 a.m. ET
2020 Wedbush PacGrow Healthcare Virtual Conference
Presentation Date: Wednesday, August 12, 2020
Time: 12:35 p.m. ET
A live webcast of these presentations will be available on the investor relations page of the company’s corporate website at View Source After the live webcast, the events will remain archived on the Aldeyra Therapeutics website for 90 days.

Seismic Bio Partners With Twist Bioscience for Discovery and Optimization of Antibodies

On August 4, 2020 Twist Bioscience Corporation (NASDAQ: TWST), a company enabling customers to succeed through its offering of high-quality synthetic DNA using its silicon platform, and Seismic Bio, Inc., an immuno-oncology company focused on bispecific antibody development, reported a partnership for the discovery and optimization of antibodies to high-value immuno-oncology targets (Press release, Twist Bioscience, AUG 4, 2020, View Source [SID1234562837]).

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"With its advanced Hyperimmune Library series, rapid discovery cycle and high throughput antibody expression capabilities, Twist is an ideal partner for accelerating our quest to develop best-in-class bispecific therapeutic antibodies," said Shelley Force Aldred, Ph.D., founder and CEO of Seismic Bio. "Creating great bispecific antibodies starts with discovering diverse collections of monoclonal antibodies, so our work with Twist is an important first step towards developing highly optimized molecules that will provide hope for patients that have not responded to standard therapies."

Under the terms of the agreement, Twist Biopharma, a division of Twist Bioscience, will use its proprietary Hyperimmune Libraries, two fully human antibody libraries focused on the heavy chain complementary determining region 3 (CDR3) loop diversity involved in antigen recognition, to identify antibodies that bind to the specified immuno-oncology targets. Seismic Bio will then use these monoclonal antibodies as building blocks to create robust bispecific antibody therapeutics. Seismic Bio will pay Twist technology activity fees as well as milestones for all compounds discovered through the agreement.

"Seismic Bio brings together some of the brightest minds with a proven track record in developing multispecific therapeutic antibodies and a focused business model to address cancer head-on by interfering at critical points in the immune system cascade," said Emily M. Leproust, Ph.D., CEO and co-founder of Twist. "While COVID-19 has been at the forefront of healthcare over the last six months, cancer patients continue to need new and safe therapeutic options. We are thrilled to partner with Seismic Bio to discover new compounds expeditiously."

Antibe Therapeutics Provides Corporate Update

On August 4, 2020 Antibe Therapeutics Inc. reported (Press release, Antibe Therapeutics, AUG 4, 2020, View Source [SID1234562836]) .:

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To our shareholders,

As we transition into a Phase III company, we want to take the opportunity to highlight recent progress and to outline our strategy as we pursue monetization of our promising drug candidates and hydrogen sulfide platform.

RECENT DEVELOPMENTS

Analysis of Phase II Dose-Ranging, Efficacy Data Now Complete

Having completed the analysis of primary and secondary data from ATB-346’s recent placebo-controlled efficacy trial, we can confidently report strong results across all key measures.

The WOMAC pain scores constituting the study’s primary endpoint showed 44% improvement from baseline at day 14 for the 200 and 250 mg once-daily doses, and 39% for the 150 mg once-daily dose. NSAIDs typically reach their maximum effectiveness after 6-8 weeks of administration and remain at that level while treatment continues. ATB-346 produced significant decreases in pain within 2 weeks of treatment. All three doses of ATB-346 reflect well in light of results from a recent peer-reviewed meta-analysis of historical NSAID osteoarthritis pain trials from Harvard researchers1. (For further information, please see our website and updated Corporate Presentation).

Beyond the impressive pain relief, ATB-346 also delivered significant improvements in the two secondary endpoints that address therapeutic benefit for patients: the WOMAC scores for stiffness and for difficulty in performing daily activities. These results are important, ‘real-world’ measures for patients with osteoarthritis ("OA") and provide additional support for the drug’s best-in-class positioning.

Supplementing the WOMAC results are the secondary data of cyclo-oxygenase ("COX") enzyme inhibition. NSAIDs reduce pain and inflammation by inhibiting the activity of COX enzymes. ATB-346 exhibited profound inhibition of COX (all doses yielding >90% inhibition) with a very high degree of statistical significance, and with negligible difference observed across the three doses. This is exceptionally strong data that is consistent with self-assessment of pain by the patients.

Adverse events across the three doses of ATB-346 were similar to placebo, with very few serious adverse events or events leading to withdrawal of treatment. Adjudicating for patient-specific factors reflected a liver safety profile for ATB-346 that was comparable to commonly prescribed NSAIDs, and substantially improved over that of acetaminophen.

Trial participants treated with ATB-346 experienced neither an increase nor decrease in blood pressure in contrast with other NSAIDs, which often increase blood pressure. Blood pressure increases are viewed by medical practitioners globally as being an important proxy for the cardiovascular risk of NSAIDs. The absence of an increase in blood pressure has been a consistent finding in all of ATB-346’s clinical trials to-date and suggests a favourable cardiovascular safety profile for the drug.

Human Proof-of-Concept Now Firmly Established for ATB-346

The strong Phase II efficacy results from this trial are a worthy complement to Antibe’s Phase II gastrointestinal ("GI") safety trial, published last year in the British Journal of Pharmacology2. That study demonstrated unequivocal GI safety superiority of ATB-346 over naproxen, one of the most prescribed NSAIDs in the US. This combined evidence for GI safety and efficacy firmly validates ATB-346 as a potential best-in-class therapy, indicating that we are on the verge of solving one of the most pervasive medical problems of our time, namely the ulcers and bleeding caused by NSAIDs.

Third Party Commercial Studies Validate Blockbuster Potential of ATB-346

In addition to completing strong GI safety and efficacy studies over the past two years, we have conducted an extensive series of market and commercial studies in key pharmaceutical markets — a total of four studies were completed, framing an impressive commercial opportunity for ATB-346 and preparing us well for late-stage partner engagement and monetization.

This effort began with a thorough health economics study led by US-based Avalon Health, quantifying the major economic impact to society arising from the damage caused by today’s NSAIDs. A subsequent commercial positioning study executed by an ex-Amgen strategy consulting team validated ATB-346’s target product profile and best-in-class positioning.

The results of these two initial studies served as the foundation for two comprehensive commercial studies for the large markets (namely, the US, the five largest European countries, and Japan), led by Shift Health and LEK, two well-respected strategy consultancies. These studies involved extensive primary and secondary research, including 62 interviews with country-specific clinicians, payors and pharmacy benefit managers – whose preferences and policies together determine the adoption of new drugs. This was followed by an in-depth survey of 80 clinicians, including primary care physicians, rheumatologists and orthopedic surgeons that prescribe NSAIDs on a daily basis. Finally, the studies developed detailed revenue models, utilizing the uptake and pricing information obtained from the research and interview phases of the project.

For the OA market alone, Antibe’s initial focus, peak annual sales of $5.3 billion are projected for ATB-346 in these seven countries, and cumulative revenues in excess of $28 billion by the early 2030s. These projections conservatively assume peak adoption of 21% and only represent 65% of the global market. Pricing and reimbursement, which drive a drug’s adoption and ability to gain market share, were favourable, with limited system resistance and few reimbursement hurdles expected. The forecasts assumed a price of US$6 per day in the US, in line with today’s branded prescription NSAIDs, with corresponding pricing in the other markets. The revenue projections combined with a high anticipated gross margin imply an impressive net present value of ATB-346, which is how potential partners determine a drug’s expected value. (For further information, please see our website and updated Corporate Presentation).

STRATEGY AND NEXT STEPS

With human proof-of-concept development and key commercial studies complete, we now have a clear line-of-sight to the significant revenue and margin potential of ATB-346. Our strategic objective is now to monetize this asset through partnering activity, while at the same time expeditiously moving forward with Phase III development. This parallel strategy is important, as partnering outreach and execution is an extensive, involved process, and the continued advancement of the drug’s development program is expected to increase market value and negotiating leverage.

Executive Team Evolving to Support Late Stage Development and Partnering

We continue to deepen the capabilities of our senior team, including the recent hiring of Dr. Rami Batal to guide our commercial strategy, and Dr. Joseph Stauffer as the Company’s Chief Medical Officer. Dr. Stauffer’s experience as an FDA reviewer, along with more than fifteen years of guiding regulatory strategy for other pain therapeutics, has already been invaluable as we pursue our regulatory, development and commercial strategies in the US and other markets. We are also hiring a US-based senior business development officer to execute the large market partnering, who will be armed with fully validated human proof-of concept data and a robust package of third-party commercial assessments.

Strategic out-licensing and monetization discussions are underway and have benefited from the strength of the recent efficacy data, while partnering discussions in the major markets are expected to accelerate following specific regulatory engagement with both the Food and Drug Administration ("FDA") in the US and the European Medicines Agency ("EMA") in Europe. We are encouraged by the preliminary feedback and hope to report on the progress on this front over the coming months.

Phase III Preparation and Regulatory Strategy Well Underway

With the successful conclusion of ATB-346’s Phase II studies and acceleration of partnering activities, preparation for Phase III trials is well underway. The Phase III program will replicate the Phase IIB GI safety and dose-ranging, efficacy studies with larger sample sizes and longer treatment durations. The first registration trial of the Phase III program will use a multi-arm design to establish the lowest effective dose and is anticipated to begin next spring, with the follow-on trials starting in a staggered fashion every several months thereafter. The strategy is global in nature, with the first trials focusing on the US and the last trial expected to address the unique regulatory requirements for Europe and the key Asian markets. The go-to-market indication will be OA, although we intend to rapidly expand this to encompass ankylosing spondylitis, rheumatoid arthritis and other chronic pain conditions. It is worth noting that successful completion of Phase III development would enable the Company to file a New Drug Application ("NDA") with the FDA and other regulatory agencies for marketing approval. This approval would deliver the first truly novel NSAID in over 20 years, and a major breakthrough in the safe and non-addictive treatment of pain and inflammation.

As part of Phase III preparation, we completed our Pre-IND ("Investigational New Drug") meeting for ATB-346 with members of the US FDA Division of Anesthesiology, Addiction Medicine, and Pain Medicine. Based on the valuable guidance received, we are moving expeditiously to complete and submit an IND application to the FDA for ATB-346. Prior to the start of the first Phase III trial, we anticipate an End-of-Phase II Meeting with the FDA and the equivalent meeting with the EMA, which will strengthen Antibe’s position with potential large market partners.

We are pleased to have recently received approval for ATB-346’s International Nonproprietary Name: otenaproxesul. (The non-proprietary name is equivalent to, for example, the "ibuprofen" of Advil). We will be using otenaproxesul going forward, and our Corporate Presentation and website have been amended as such. In addition, we have now engaged a healthcare branding agency to help us select a proprietary (brand) name (the equivalent to "Advil").

ATB-352 Advancing Towards Clinical Development Next Year

Expanding beyond our lead drug, we have commenced IND-enabling preclinical studies for our second pipeline drug, ATB-352. This drug is being developed as a GI-safe and non-addictive alternative to opioids for the treatment of post-surgical pain, a US$11 billion market3. Many physicians find themselves in a quandary regarding opioids, which have been the drug-of-choice for post-surgical pain. The natural alternative would be the stronger NSAIDs, already shown to equal the pain relief of opioids in such settings. However, such NSAIDs are also the most harmful to the digestive tract. ATB-352, a hydrogen sulfide-releasing version of ketoprofen (a particularly strong NSAID), is being developed as a solution to this longstanding problem. In animal experiments, ATB-352 has delivered greater pain effectiveness than ketoprofen yet demonstrated robust GI safety. We anticipate that preclinical studies of ATB-352 will conclude in calendar Q3 2021, following which we will seek regulatory approval to initiate human trials. We plan to pursue a Fast Track designation with the FDA to expedite the development and regulatory approval process.

Our third pipeline drug, ATB-340, a hydrogen-sulfide releasing version of low-dose aspirin, has been shown to cause negligible GI damage in preclinical studies. Low-dose aspirin is generally regarded as a wonder drug for cardiovascular and cancer protection for people over 50, but its prevalence of GI-damage precludes its broad prescription by clinicians. We are presently evaluating the clinical development strategy for ATB-340 and anticipate commencing IND-enabling preclinical studies in calendar 2021.

Pipeline Expansion Initiatives Launched

The strength of the recent clinical data has also encouraged us to commit to unlocking additional value from our hydrogen sulfide platform. To this end, Antibe recently signed a contract with a leading academic institution in the US known for the quality of its hydrogen sulfide science. The objective of this mandate is to identify promising new hydrogen sulfide-releasing compounds for the treatment of chronic pain, acute pain and other indications, including inflammatory bowel disease. Antibe will retain ownership rights to any new intellectual property arising from this work.

In addition, we are exploring collaborations for additional indications for otenaproxesul, including familial adenomatous polyposis (FAP), a pre-cancerous and often-fatal orphan disease.

Capital Markets Strategy Taking Institutional Focus

The success of otenaproxesul over the past few years has increased our valuation and de-risked our story considerably – this could not have been done without the long-term support of our existing shareholders. The last several months culminated in significant achievements, including a $29 million bought deal financing, that position us well to crystalize the value of our efforts. While we believe the issue price did not reflect the opportunities before us, the financing was essential for executing our plans in the coming 15 months at full speed with a fully funded balance sheet. We will now be pursuing a capital markets strategy to support our next stage of growth, while enabling our share price to reach an inherent value that is more comparable to our Phase III peers.

The completion of human proof-of-concept development for otenaproxesul with such strong results has put us on the radar screens of institutional investors that will be instrumental in supporting this growth. Preparations are largely complete to list on a senior exchange at the optimal time, such as NASDAQ. In addition, we will shortly engage a premier life science-focused investor relations firm in the US to implement and lead a strategic outreach program towards institutional investors, sell-side analysts and bankers.

We are in the process of evaluating a potential sale of Citagenix, our commercial asset that is no longer a strategic fit. This will simplify our story into a "pure play" late-stage biotech company with a clear focus and growth trajectory. And as we attract more institutions and mature as an organization, we will correspondingly require more active and deepened governance to guide our decision-making. We have retained a specialized recruitment firm to help us find a new, US-based Board member, whom we expect to have in place by year end.

We are excited by the opportunities ahead, as we strive to unlock shareholder value and execute our strategy over the course of the coming year.

Sincerely,

Dan Legault

Chief Executive Officer

1 Comparative Pain Reduction of Oral Non-steroidal Anti-inflammatory Drugs and Opioids for Knee Osteoarthritis: Systematic Analytic Review. Osteoarthritis and Cartilage. S.R. Smith et al., 2016.

2 A proof-of-concept, Phase 2 clinical trial of the gastrointestinal safety of a hydrogen sulfide-releasing anti-inflammatory drug. British Journal of Pharmacology. Wallace et al., 2019.

3 Transparency Market Research, 2018.

T-Cure Bioscience Announces Expansion of Collaboration With the National Institutes of Health for T Cell Receptor (TCR) Therapy Targeting HERV-E for the Treatment of Kidney Cancer

On August 4, 2020 T-Cure Bioscience, Inc., a privately held company focused on developing autologous T cell receptor (TCR) therapy products for the treatment of solid tumors, reported that the Company has extended its ongoing research collaboration with the National Heart Lung and Blood Institute (NHLBI), through a formal Collaborative Research and Development Agreement (CRADA) to advance the Company’s HERV-E targeting TCR therapy for renal cell cancer (Press release, T-Cure Bioscience, AUG 4, 2020, View Source [SID1234562835]). Additionally, T-Cure has amended an existing license to expand to worldwide rights for the intellectual property it licensed from NHBI related to the anti-HERV-E TCR product. This TCR therapy is currently in a Phase 1 trial at NHLBI for the treatment of metastatic clear cell Renal Cell Carcinoma (ccRCC) that failed an angiogenic inhibitor and a checkpoint inhibitor. T-Cure has been actively collaborating for the past 18 months with NHLBI on the research and development of the HERV-E-specific TCR.

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Under the CRADA, T-Cure and NHLBI will collaborate to develop a companion test to identify HERV-E transcripts in patients’ tumors as well as to identify additional therapy candidates targeting HERV-E using the Company’s proprietary TCR discovery platform, iSORTTM. Additionally, NHLBI and T-Cure plan to conduct preclinical experiments characterizing novel TCR and will evaluate various drug and TCR combination anti-tumor therapeutic strategies.

"We are extremely excited to work with the NIH to advance this novel HERV-E TCR therapy candidate through preclinical and clinical development," stated Gang Zeng, Ph.D., Chief Executive Officer of T-Cure. "Of note, the TCR was isolated from a dominant killer T cell clone of a late stage metastatic ccRCC patient who responded to an immunotherapy and survived 4 years. The HERV-E-specific TCR represents a rare opportunity for us to specifically target ccRCC, a potential T cell responsive solid tumor."

The HERV-E target is one of the quiescent Human Endogenous Retrovirus (HERV) sequences that are activated during tumor development. A growing number of HERV genes and proteins are expressed in different cancers raising the possibility that HERV derived antigens might represent excellent targets for tumor immunotherapy. Its expression in renal cell carcinoma (RCC) is highly selective, with no transcripts detected in any normal tissues. In contrast to well-studied antigens such as NY-ESO-1 and MAGE, HERV-E represents a new frontier of TCR targets with significant clinical potential for immunotherapy.