Bicycle Therapeutics Announces Significant Progress Across Multiple Therapeutic Programs Beyond Oncology

On March 30, 2021 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 for its Bicycle-based partnered programs outside of oncology (Press release, Bicycle Therapeutics, MAR 30, 2021, View Source [SID1234577366]).

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"Over the last five years, Bicycle’s strategy has been to use our novel technology to discover and develop a pipeline of innovative assets in oncology while additionally using non-dilutive funding and collaborations to explore the therapeutic potential of Bicycles in disease areas outside of oncology. Today, for the first time, we are providing an overview on our progress in demonstrating the broad utility of this platform to create molecules with the potential to treat some of the most serious diseases and address future healthcare challenges," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "It’s incredibly exciting to see how the Company has worked innovatively and highly collaboratively with many diverse institutions to address these challenges and, at the same time, generated significant revenues to offset the costs of developing and progressing our internal oncology pipeline. I would like to thank all of our collaborators for their enthusiasm in this endeavor and look forward to continuing our work to advance these important molecules."

Bicycle has achieved the first milestone in its collaboration with Dementia Discovery Fund (DDF) and the University of Oxford’s ARUK Oxford Drug Discovery Institute (ODDI)

The Company identified and optimized nM affinity Bicycles to TREM2, a genetically validated target for the treatment of dementia, and nM affinity Bicycles to transferrin receptor 1 (TfR1), a molecular shuttle. In 2019, Bicycle established a collaboration with DDF, later expanded to include ODDI, to use Bicycle technology for the discovery and development of potential novel therapeutics for the treatment of dementia. The three parties are collaborating to identify and characterize Bicycles that bind to and activate TREM2, a genetically validated dementia target. As part of the collaboration, Bicycle is also using its technology to identify binders to TfR1, a blood brain barrier transporter, that could potentially enable delivery of Bicycles, or any other molecular payload, into the central nervous system (CNS), or other organ systems, expressing TfR1. Under the terms of the collaboration with DDF, Bicycle retains all rights to the TfR1 molecules outside of the license granted for use with targets defined within the DDF collaboration.
Bicycle advances platform in multiple anti-infective areas, including antimicrobials and antivirals

Innovate UK’s Biomedical Catalyst (BMC) awarded the Company funding to advance a Bicycle inhibitor for a key cell wall biosynthesis target in Enterobacterales, Penicillin Binding Protein 3 (PBP3). Bicycle is using its proprietary platform to try to address the significant healthcare challenge of antimicrobial resistance and has identified several potential Bicycle PBP3 inhibitors. Through grants awarded by the UK government and in collaboration with investigators at the University of Warwick, Bicycle intends to progress these PBP3 inhibitors, potentially representing the first novel class of antibiotics identified in decades, to candidate and initial toxicology testing.
Innovate UK, under a specific program targeting key technologies to rapidly respond to the challenge of COVID-19 (UKRI Ideas to Address COVID-19 – Innovate UK Article 25), provided funding to support Bicycle’s efforts to discover new healthcare solutions to the SARS-CoV-2 pandemic. Bicycle has identified numerous discrete families of Bicycles which bind to at least 10 different epitopes on the SARS-CoV-2 spike protein, binders to the Nucleocapsid protein and the viral attachment site on the host ACE2 human receptor. Through Bicycle’s partnership with researchers at the MRC Laboratory of Molecular Biology, Bicycles identified by these screens have demonstrated nanomolar activity in inhibiting SARS-CoV-2 infection of human lung cells. These monomeric Bicycles have been conjugated together to make a diverse range of bi-paratopic and multi-valent small molecules (less than 10kD), some of which are active in the picomolar range in viral entry assays and may be resistant to the current SARS-CoV-2 variants of concern. Bicycle continues to advance these novel compounds in pre-clinical evaluation. Bicycle has also formed partnerships with diagnostic experts to evaluate its novel SARS-CoV-2 binding Bicycles in Lateral Flow Tests and other diagnostic screening formats.
Bicycle has made significant progress through partnerships with biopharmaceutical therapeutic area leaders in indications outside of oncology

The Company has successfully discovered and advanced targets outside of oncology through the ongoing collaboration with AstraZeneca, a global biopharmaceutical company, to discover novel agents for the treatment of respiratory and cardiometabolic diseases. Two assets have been transitioned to AstraZeneca for further development and are currently being progressed in their discovery pipeline. Two additional assets achieved transition criteria and were transitioned to AstraZeneca but were returned to Bicycle by AstraZeneca.
Bicycle has also identified targets in its collaboration with Bioverativ (acquired by Sanofi in 2018) for the treatment of rare hematological diseases. The Bioverativ collaboration successfully identified nM multi-valent inhibitors to P-Selectin, which inhibited human neutrophil binding and rolling, with potential applications in sickle cell disease and other inflammatory diseases. The Bioverativ collaboration also identified the first small molecule Factor VIII mimetic for the potential treatment of Hemophilia A. This mimetic is comprised of a multivalent Bicycle binding to both Factor IX and Factor X, leading to the production of activated Factor X (FXa), which successfully activated thrombin in plasma from Hemophilia A patients ex vivo. In 2019, Sanofi elected not to pursue further development, and the collaboration agreement was terminated. These "lead stage" assets have now been returned to Bicycle.
Kevin Lee commented, "With four Bicycle molecules now in clinical trials for both oncology and non-oncology indications, the technology has shown initial evidence of clinical tolerability and suitability for pharmaceutical development, and I look forward to seeing how Bicycles may bring important new treatment opportunities to poorly served patients in additional therapeutic areas."

VBL Therapeutics to Host Virtual R&D Day on April 6

On March 30, 2021 VBL Therapeutics (Nasdaq: VBLT) reported that it will host a virtual R&D Day for analysts and investors beginning at 10:00am ET on Tuesday, April 6, 2021 (Press release, VBL Therapeutics, MAR 30, 2021, View Source [SID1234577365]). The event will include presentations from Professor Dror Harats, M.D, Chief Executive Officer, Dr. Bradley Monk, M.D., FACS, FACOG, Chair of the OVAL Study Steering Committee, and Dr. Tami Rachmilewitz, M.D, Vice President of Clinical Development, and will focus on the Company’s clinical-stage pipeline including lead program, VB-111. The live event can be accessed at View Source

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Following the event, an archive will be available via the "Events and Presentations" section of the VBL investor relations website at View Source

Soligenix Announces Recent Accomplishments And Year-End 2020 Financial Results

ON March 30, 2021 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported its recent accomplishments and financial results for the year ended December 31, 2020 (Press release, Soligenix, MAR 30, 2021, View Source [SID1234577364]).

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"2021 will be another important year for Soligenix on a number of fronts," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "With our pivotal Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial successfully completed, we are now preparing to begin submission of the rolling new drug application (NDA) in 2Q 2021 for this first-in-class therapy. Compared to the currently approved cutaneous T-cell lymphoma (CTCL) therapies for early disease, the SGX301 treatment response was very rapid, being detected in as little as 6 weeks of treatment (Cycle 1, p=0.0416). Responses continued to improve through 12 weeks of treatment (Cycle 2, p<0.0001 vs end Cycle 1) and 18 weeks of treatment (Cycle 3, p<0.0001 vs end Cycle 1), ultimately enabling nearly half of patients who continued treatment to see sustained and significant improvement in their response rates. Moreover, patients with more difficult to treat plaque lesions also showed this same treatment benefit, an advantage over some other therapies which primarily work only for patch lesions. Under our Public Health Solutions business segment, we continue to advance multiple therapeutic and vaccine candidiates, with the support of non-dilutive government funding, including our heat stable COVID-19 vaccine, CiVax, which recently demonstrated rapid-onset, broad-spectrum, neutralizing antibody and cell-mediated immunity using full-length Spike protein antigens in mice. Overall, our thermostabilization platform has now demonstrated compatibility with two different adjuvants, consistently enabling single-vial vaccine presentations with characteristics for ambient shipping and long-term storage. Further, non-human primate data is expected across our vaccine platform later this year."

Dr. Schaber continued, "With over $30 million in cash, not including our non-dilutive funding, we anticipate having sufficient capital to achieve multiple inflection points across our rare disease pipeline, including moving towards NDA and U.S. commercialization of SGX301 in CTCL. As CTCL is a highly specialized orphan market with a discrete prescriber base, it presents a tailor-made market opportunity for us. We estimate peak U.S. annual net sales to exceed $90 million and the total U.S. revenues during the 10-year forecast period to be greater than $700 million. With a total addressable worldwide market projected to be approximately $250 million annually, we continue to have ongoing confidential discussions regarding ex-U.S. partnership opportunities."

Soligenix Recent Accomplishments

On March 9, 2021, the Company announced that it received preliminary approval for a tax credit from the New Jersey Economic Development Authority’s (NJEDA) New Jersey Technology Business Tax Certificate Transfer program. As a result, the Company anticipates being able to transfer this credit and receive approximately $865,000 in net proceeds. To view this press release, please click here.
On March 4, 2021, the Company announced publication of pre-clinical immunogenicity studies for CiVax (heat stable COVID-19 vaccine program) demonstrating rapid-onset, broad-spectrum, neutralizing antibody and cell-mediated immunity is confirmed using full-length Spike protein antigens. To view this press release, please click here.
On February 24, 2021, the Company issued an update letter from its President and Chief Executive Officer, Dr. Christopher J. Schaber. To view this press release, please click here.
On February 1, 2021, the Company announced that the Hong Kong Registrar of Patents granted a patent for the application titled "Formulations and Methods of Treatment of Skin Conditions" (No. 16102842.8), published on January 29, 2021 under Publication No. 1214771 B. To view the press release, please click here.
On January 26, 2021, the Company hosted an Investor Webcast Event regarding U.S. commercial planning for SGX301 (synthetic hypericin) in the treatment of CTCL. To listen to the webcast, please click here.
On January 7, 2021, the Company announced that it signed an exclusive Supply, Distribution and Services Agreement with Daavlin, securing long-term supply and distribution of a commercially ready light device that is an integral component of the regulatory and commercial strategy for SGX301 for the treatment of CTCL. To view the press release, please click here.
On December 28, 2020, the Company announced that the National Institute of Allergy and Infectious Diseases (NIAID) awarded Soligenix a Direct to Phase II Small Business Innovation Research (SBIR) grant of approximately $1.5 million to support manufacture, formulation (including thermostabilization) and characterization of COVID-19 and Ebola Virus Disease vaccine candidates in conjunction with the CoVaccine HT adjuvant. To view the press release, please click here.
On December 22, 2020, the Company announced preliminary top-line results for its pivotal Phase 3 DOM-INNATE trial evaluating SGX942 in the treatment of severe oral mucositis in patients with head and neck cancer receiving chemoradiation. To view the press release, please click here.
On December 16, 2020, the Company announced that it entered into a $20 million convertible debt financing agreement with Pontifax Medison Debt Financing, the healthcare-dedicated venture and debt fund of the Pontifax life science funds. To view the press release, please click here.
On December 8, 2020, the Company announced that it demonstrated extended protection with its heat stable ricin toxin vaccine, RiVax. To view the press release, please click here.
Financial Results – Year Ended December 31, 2020

Soligenix’s revenues for the year ended December 31, 2020 were $2.4 million as compared to $4.6 million for the year ended December 31, 2019. Revenues included payments on a contract in support of RiVax, our ricin toxin vaccine candidate, grants received to support the development of SGX943 for treatment of emerging and/or antibiotic-resistant infectious diseases, ThermoVax, our thermostabilization technology, and the assessment of SGX942 safety in juvenile animals.

Soligenix’s basic net loss was $17.7 million, or ($0.64) per share, for the year ended December 31, 2020, as compared to $9.4 million, or ($0.48) per share, for the year ended December 31, 2019. The increase in net loss is primarily due to increased expenditures incurred to support both the pivotal Phase 3 trial of SGX301 in the treatment of CTCL and the pivotal Phase 3 trial of SGX942 in the treatment of oral mucositis in head and neck cancer, as well as a $5 million success milestone payment in common stock (based upon an effective per share price of $2.56) as a result of SGX301 demonstrating statistically significant treatment response in the pivotal Phase 3 clinical trial.

Research and development expenses were $10.1 million as compared to $8.1 million for the years ended December 31, 2020 and 2019, respectively. The increase in research and development spending for the year ended December 31, 2020 was related to expenditures incurred in the expansion of the Phase 3 clinical trial of SGX942 as well as the ongoing Phase 3 clinical trial of SGX301.

General and administrative expenses were $4.0 million as compared to $3.5 million for the years ended December 31, 2020 and 2019, respectively.

As of December 31, 2020, the Company’s cash position was approximately $18.7 million.

Cardinal Health to Webcast Discussion of Third-Quarter Results for Fiscal Year 2021 on May 6

On March 30, 2021 Cardinal Health (NYSE: CAH) reported third-quarter financial results for its fiscal year 2021 on May 6 prior to the opening of trading on the New York Stock Exchange (Press release, Cardinal Health, MAR 30, 2021, View Source [SID1234577363]). The company will webcast a discussion of these results beginning at 8:30 a.m. Eastern.

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To access the webcast and corresponding slide presentation, go to the Investor Relations page at ir.cardinalhealth.com. No access code is required. Presentation slides and a webcast replay will be available until May 5, 2022.

Crinetics Pharmaceuticals Announces 2021 Clinical Plans and Reports Fourth Quarter and Full Year 2020 Financial Results

On March 30, 2021 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported financial results for the fourth quarter and year ended December 31, 2020 (Press release, Crinetics Pharmaceuticals, MAR 30, 2021, View Source [SID1234577362]).

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"We are entering 2021 poised to achieve several key milestones that we believe will consolidate our position as a leader in the design and development of novel small molecule drugs for endocrine diseases," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "Our positive Phase 2 acromegaly data demonstrate the potential of orally administered paltusotine to replace injectable somatostatin receptor ligand (SRL) depots as the standard-of-care. With input from a recently completed FDA meeting, we have designed the paltusotine Phase 3 program to support its approval for all acromegaly patients who require pharmacotherapy, including both untreated patients and those switching from injected standard of care."

Dr. Struthers continued, "Paltusotine’s success and progress serve as a blueprint for our new therapeutic candidates in endocrinology. We plan to follow a similar path with CRN04777 and CRN04894, both of which recently entered Phase 1 proof-of-concept trials in healthy volunteers. Much like paltusotine’s Phase 1 program, we’ve designed these trials to evaluate safety, drug-like pharmacokinetics, and corresponding pharmacodynamic effects on well-validated hormonal biomarkers for their respective indications. We look forward to reporting results from the Phase 1 trials later this year, and to the continued progression of our clinical and preclinical pipelines."

Full Year 2020 and Recent Highlights

Advancing paltusotine to a Phase 3 program for acromegaly following the completion of an FDA meeting. In 1Q 2021, Crinetics met with the U.S. Food and Drug Administration (FDA) to discuss its Phase 2 acromegaly data and plans for a Phase 3 program. Based on these interactions and those with other regulators, the Company plans to initiate two Phase 3 studies in 2021. The first of these studies, entitled PATHFNDR-1, is a double-blind, placebo-controlled, nine-month clinical trial evaluating the safety and efficacy of paltusotine in acromegaly patients who are biochemically controlled (IGF-1 ≤ 1.0 × upper limit of normal [ULN]) and who are on stable doses of SRL monotherapy (octreotide LAR or lanreotide depot). The second study, PATHFNDR-2, is a double-blind, placebo-controlled, twelve-week trial in acromegaly patients with elevated IGF-1 levels who are medication naïve or who are not being treated with pharmacotherapy (untreated patients). The primary endpoint for both studies will be the proportion of patients achieving biochemical control comparted to placebo. If successful, Crinetics believes these trials could support registration of paltusotine in the United States and Europe for all acromegaly patients who require pharmacotherapy, including untreated patients and those switching from standard of care. The Company expects to initiate PATHFNDR-1 in 2Q 2021 and PATHFNDR-2 in 2H 2021.

Showcased broad clinical-stage pipeline at ENDO 2021. In March 2021, Crinetics showcased the breadth of its pipeline with presentations on its three clinical programs at the Endocrine Society’s annual ENDO 2021 congress. Posters on the company’s acromegaly program included a summary of the previously announced ACROBAT Edge Phase 2 results, as well as details of a new tablet formulation of paltusotine. Presentations related to the company’s earlier stage clinical programs included a poster with preclinical data supporting the development of CRN04777 as a treatment for congenital

hyperinsulinism (congenital HI) and a live oral presentation with preclinical evidence supporting the further evaluation of CRN04894 in Cushing’s disease and congenital adrenal hyperplasia (CAH).

Advanced ACTH antagonist CRN04894 into a Phase 1 study designed to provide clinical proof of concept. In February 2021, Crinetics initiated a Phase 1 study of CRN04894, an investigational, oral, nonpeptide adrenocorticotropic hormone (ACTH) antagonist being developed for the treatment of diseases associated with excess ACTH such as Cushing’s disease and CAH. In addition to evaluating the safety and tolerability of CRN04894 in healthy volunteers, the placebo-controlled study aims to provide clinical proof-of-concept data by measuring the effect of CRN04894 on the suppression of ACTH-stimulated adrenal secretion of cortisol and related steroids. These endocrine biomarkers are used as key endpoints in patient studies and reflect the ability of CRN04894 to block ACTH-stimulated adrenal function. This Phase 1 trial is expected to provide important information for dose selection and be predictive of efficacy in Cushing’s disease and CAH trials. Crinetics expects to report preliminary safety, pharmacokinetic, and endocrine biomarker data from the single ascending dose portion of the trial in 1H 2021.

Advanced SST5 agonist CRN04777 into a Phase 1 study designed to provide clinical proof of concept. In February 2021, Crinetics initiated a Phase 1 study of CRN04777, an investigational, oral, nonpeptide somatostatin receptor type 5 (SST5) agonist. CRN04777 is being developed as a treatment for congenital HI, a rare genetic disease in which excess insulin secretion causes life-threatening hypoglycemia (low blood glucose). In addition to evaluating the safety and tolerability of CRN04777 in healthy volunteers, the placebo-controlled study aims to provide clinical proof of concept data by measuring the ability of CRN04777 to inhibit glucose- and sulfonylurea-induced insulin secretion and observe the corresponding effects on blood glucose levels. These endocrine biomarkers are indicative of the ability of CRN04777 to prevent hypoglycemia and are expected to provide information for dose selection and be predictive of efficacy in patients with hyperinsulinism. Crinetics expects to report preliminary safety and pharmacological effect data from the single ascending dose portion of the trial in mid-2021.

Reported positive topline results for the ACROBAT Phase 2 program of paltusotine in acromegaly patients. In October 2020, Crinetics reported positive top-line data from its Phase 2 program evaluating paltusotine for the treatment of acromegaly. Data showed that levels of insulin-like growth factor 1 (IGF-1), the accepted biomarker of disease control in acromegaly, were maintained in patients switching from standard-of-care injected SRL depots to once-daily oral paltusotine.

Received Rare Pediatric Disease Designation for CRN04777 for the treatment of congenital HI. In September 2020, the FDA granted CRN04777 Rare Pediatric Disease (RPD) Designation for the treatment of congenital HI. Based on this designation, Crinetics may be eligible to receive a priority review voucher (PRV) following approval of CRN04777 if the marketing application submitted for the candidate satisfies certain additional conditions. If issued, a PRV may be redeemed for priority review of a subsequent marketing application for a different product candidate, or the PRV could be sold or transferred to another sponsor.

Received Orphan Drug Designation for paltusotine for the treatment of acromegaly. In July 2020, the FDA granted paltusotine Orphan Drug Designation for the treatment of acromegaly, qualifying Crinetics for certain development incentives such as exemption from FDA prescription drug user fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. upon FDA approval.

Strengthened company leadership with appointments to clinical team and Board of Directors. Throughout 2020, Crinetics continued to reinforce its in-house expertise in endocrinology, rare disease drug development, and clinical trial management through the appointments of Drs. Alessandra Casagrande, Peter Trainer, and Hjalmar Lagast to the clinical team and the appointment of Dr. Camille L. Bedrosian to the Board of Directors.

Fourth Quarter and Full Year 2020 Financial Results

Research and development expenses were $16.8 million and $57.0 million for the three months and full year ended December 31, 2020, respectively, compared to $12.1 million and $41.5 million for the same periods in 2019. The increases were primarily attributable to clinical development and manufacturing activities for paltusotine as well as the company’s preclinical programs.

General and administrative expenses were $5.0 million and $18.0 million for the three months and full year ended December 31, 2020, compared to $3.4 million and $13.5 million for the same periods in 2019. The increases were primarily due to costs to operate as a public company, as well as personnel costs to support the company’s growth.

Net loss for the three months ended December 31, 2020 was $21.6 million, compared to a net loss of $14.5 million for the same period in 2019. For the year ended December 31, 2020, the company’s net loss was $73.8 million compared to a net loss of $50.4 million for the year ended December 31, 2019.

Unrestricted cash, cash equivalents and investments totaled $170.9 million as of December 31, 2020, compared to $186.8 million as of September 30, 2020 and $118.4 million as of December 31, 2019.

As of February 26, 2021, the company had 33,028,876 common shares outstanding.

Webcast and Conference Call Information

Crinetics will hold a live webcast and conference call today, March 30, 2021 at 4:30 p.m. Eastern Time to discuss its 2021 clinical plans and 2020 financial results. To access the webcast, please visit this link to the event. To participate by phone, please dial 877-407-0789 (domestic) or 201-689-8562 (international) and refer to conference ID 13717687. Following the live event, the archived webcast will be available for 90 days.