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Compass Therapeutics Announces Publication of Data Demonstrating the Preclinical Efficacy and Safety of CTX-471, a Unique Fully Human Agonist of CD137

On March 30, 2020 Compass Therapeutics, a clinical-stage biotechnology company focused on drugging the human immune synapse, reported the publication of extensive data demonstrating the preclinical efficacy and safety of the company’s lead investigational therapy, CTX-471, which is currently being evaluated in a Phase 1 clinical trial (Press release, Compass Therapeutics, MAR 30, 2020, View Source [SID1234556006]). The paper, "Differentiated Agonist Antibody Targeting CD137 Eradicates Large Tumors without Hepatotoxicity," was published in the peer-reviewed journal JCI Insight. It describes the discovery and preclinical characterization of CTX-471, a fully human antibody that binds and activates a unique epitope of the CD137 receptor and has demonstrated a favorable and differentiated efficacy and safety profile, including monotherapy activity against multiple syngeneic tumor models and excellent tolerability in animals.

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The CTX-471 Phase 1 clinical trial is ongoing in patients with selected metastatic or locally advanced solid cancers who have received at least one prior line of therapy. The dose escalation portion of the study is projected to complete in mid-2020, and a dose expansion phase will follow.

"CTX-471 binds a unique epitope on the CD137 receptor, not targeted by other CD137 agonists, and as a monotherapy, it has demonstrated eradication of large, fast-growing tumors in mice where other immuno-oncology therapies have had minimal effect in these models. Interestingly, our antibody appears to generate long-term, functional immunological memory leading to complete rejection of newly inoculated tumors in those treated animals," said Thomas Schuetz, M.D., Ph.D., co-founder and chief executive officer at Compass Therapeutics. "We know that many patients do not respond to PD-1 or PD-L1 blockers as monotherapies, leaving them with limited alternative therapeutic options. We hope CTX-471 can address those needs and improve the lives of those patients."

Highlights from the publication include:

CTX-471 as monotherapy achieved high rates of complete tumor regression in multiple syngeneic mouse models.
CTX-471 selectively and profoundly reprogrammed the tumor microenvironment, increasing CD8+ T cell infiltration and penetration while reducing T cell exhaustion and regulatory T cell infiltration.
The anti-tumor activity of CTX-471 was driven by immunoglobulin FC receptor (FcyR)-engagement and required the coordinated involvement of both T cells and NK cells.
CTX-471 is believed to work by stabilizing the receptor trimer while allowing the natural ligand to bind to the receptor.
About CTX-471

CTX-471 is a fully human monoclonal antibody that binds and activates a novel epitope of the co-stimulatory receptor CD137, also known as 4-1BB, a member of the tumor necrosis factor receptor superfamily. The antibody is currently being evaluated in a Phase 1 clinical trial in patients with solid tumors that have progressed despite at least three months on an approved PD-1 or PD-L1 inhibitor. In preclinical studies, CTX-471 has demonstrated potent monotherapy activity against multiple syngeneic tumor models, including the generation of long-term functional immunological memory. Most notably, CTX-471 shows a unique ability to completely eradicate large, established tumors where other preclinical CD137 antibodies and antibodies against PD-1, PD-L1, CTLA-4 and OX40 have minimal effects in these models. In contrast to other CD137 antibodies, CTX-471 shows no evidence of hepatic toxicity in either mice or non-human primates, supporting the potential for a wide therapeutic window in patients.

ElevateBio Closes $170 Million Series B Financing

On March 30, 2020 ElevateBio, LLC, a Cambridge-based creator and operator of a portfolio of innovative cell and gene therapy companies, reported it has closed a $170-million Series B financing that will support and accelerate the company’s next phase of growth. New investors, The Invus Group, Surveyor Capital (A Citadel company), EDBI, and Vertex Ventures, join existing investors, F2 Ventures, MPM Capital, EcoR1 Capital, Redmile Group, and Samsara BioCapital, all of whom also participated in this financing (Press release, ElevateBio, MAR 30, 2020, View Source [SID1234556005]). Additionally, the company announced Germano Giuliani will join its Board of Directors.

"In less than a year since launching ElevateBio, we have firmly established our first-of-its-kind business model in cell and gene therapy, by announcing our initial two therapeutic companies, commencing operations for additional companies, and entering into a 10-year manufacturing partnership with one of the world’s top hospital systems," said David Hallal, Co-Founder and Chief Executive Officer of ElevateBio. "With the support of our new and existing investors, our highly disruptive, capitally efficient model uniquely positions ElevateBio to accelerate the rate of innovation in cell and gene therapy manufacturing, enabling technology platforms, and therapeutic development for many years to come."

The Series B proceeds will enable:

ElevateBio BaseCamp to be fully operational with cGMP manufacturing;
The advancement of clinical development with at least six cell and gene therapies from across its portfolio companies to enter clinical studies this year and into 2021;
Key milestones and continued development of ElevateBio’s enabling technology platforms.
"Cell and gene therapies are recognized as the future of precision medicine globally, and we believe ElevateBio’s platform enables product innovation to be accelerated with their unique business model and manufacturing expertise," said Chu Swee Yeok, Chief Executive Officer and President of EDBI, a Singapore-based global investor in fast growing technology industries. "As ElevateBio continues to expand their global footprint, they are ideally suited to partner with biotech companies and academic centers in Asia to discover and develop novel therapies to treat cancers and rare diseases."

Germano Giuliani, Board Member

Along with the Series B financing, ElevateBio also announced that Germano Giuliani will join ElevateBio’s Board of Directors. Mr. Giuliani is an industry expert who is a member of the Board of Directors at HBM Healthcare Investments AG, a role he has had since 2012. Mr. Giuliani is also a member of the Investment Committee at Royalty Pharma. Additionally, Mr. Giuliani previously served as the Chief Financial Officer, Chairman and Chief Executive Officer of Giuliani SpA – a 131-year-old Italian pharmaceutical company that continues to develop, manufacture, and distribute health and beauty products. He currently serves, or has served, on several boards where he provided his extensive expertise and oversight across all aspects of the businesses. Mr. Giuliani has a degree in economics and commerce from the Catholic University of the Sacred Heart in Milan, Italy.

"We are thrilled to welcome Germano to the ElevateBio Board," said Mr. Hallal. "Germano’s deep pharmaceutical executive experience building long-term value for all stakeholders as an operator, Board member, and investor will play a significant role at ElevateBio as we advance development of life-transforming therapies to serve patients around the world."

Update Regarding ElevateBio’s Recent Milestones

ElevateBio is completing the buildout of its R&D, process development, and manufacturing operation, ElevateBio BaseCamp, a centralized 140,000 square foot cell and gene therapy innovation hub located in Waltham, Mass.
ElevateBio launched AlloVir, a late-clinical stage T-cell immunotherapy company, with technology stemming from the Baylor College of Medicine’s Center for Cell and Gene Therapy in May 2019 with a $120-million Series B financing led by Fidelity Research and Management Company. In 2019, AlloVir received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration. This year, the European Medicines Agency granted AlloVir two designations for Viralym-M (ALVR105), the company’s lead allogeneic, off-the-shelf, multi-virus specific T-cell therapy, targeting six commonly occurring, devastating viruses in allogeneic hematopoietic stem cell transplantation (HSCT) recipients: PRIority MEdicines (PRIME) designation for the treatment of serious infections with BK virus, cytomegalovirus, human herpes virus-6, Epstein Barr virus, and/or adenovirus in allogeneic hematopoietic stem cell transplantation (HSCT) recipients; and Orphan Drug Designation as a potential treatment of viral diseases and infections in patients undergoing HSCT. AlloVir plans to initiate pivotal studies of Viralym-M in stem cell transplant and proof-of-concept studies in solid organ transplant patients and the company will also advance its second product candidate, ALVR106, into a proof-of-concept study for four devastating community acquired respiratory viruses in 2020. AlloVir also announced an expanded research agreement with Baylor College of Medicine to discover and develop a coronavirus-specific T-cell therapy for immunocompromised patients at risk for COVID-19.
In October 2019, HighPassBio was launched with technology from the Fred Hutchinson Cancer Research Institute. HighPassBio is dedicated to advancing novel, T-cell receptor therapies targeting HA1-expressing tumors which are designed to treat, and potentially prevent, relapse of leukemia following hematopoietic stem cell transplant (HSCT). A Phase 1 clinical trial is open and recruiting for adult and pediatric patients who have relapsed with leukemia or related conditions following stem cell transplantation.
In July 2019, ElevateBio announced a 10-year agreement with Massachusetts General Hospital (MGH) to manufacture innovative cell and gene therapies at ElevateBio BaseCamp as well as to create cell and gene therapy-focused companies together over time.
Throughout 2019, ElevateBio commenced operations of additional cell and gene therapy companies, including highly innovative AAV-based gene therapies, TCR, and CAR T cells, which will be publicly disclosed later in 2020.

Soligenix Announces Recent Accomplishments And Year-End 2019 Financial Results

On March 30, 2020 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, 2019 (Press release, Soligenix, MAR 30, 2020, View Source [SID1234556004]).

Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix stated, "We are extremely pleased to have achieved positive top-line results in our pivotal Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial that demonstrates SGX301’s potential to be an important new treatment for early stage cutaneous T-cell Lymphoma (CTCL). Having demonstrated statistical significance in the study’s primary endpoint, we will now look to report results from the extended treatment portion of the trial, which we anticipate announcing in June 2020. Following the positive recommendation received from the independent Data Monitoring Committee, we have successfully achieved our target of 260 patients randomized into the pivotal Phase 3 clinical trial of SGX942 (dusquetide) for the treatment of oral mucositis in patients with head and neck cancer (HNC) receiving chemoradiation therapy; however, due to the uncertainty surrounding the coronavirus pandemic, we have decided to enroll approximately 25 additional patients into the study. We are taking this cautious approach in order to maintain the statistical integrity of the trial, by accounting for patients that may potentially drop out of the study before completing their protocol required study treatment and evaluation. Therefore, the study target to complete enrollment and provide top-line results is being revised from the second quarter of 2020 to the fourth quarter 2020; however, this will remain dependent on the medical and logistical challenges caused by the coronavirus showing a reasonable level of improvement in the relative near-term."

Dr. Schaber continued, "Under our Public Health Solutions business segment, we continue to progress our heat stable ricin vaccine, RiVax, with the support of a National Institute of Allergy and Infectious Disease contract award of up to $24.7 million. We are also excited to advance our work with University of Hawaiʻi at Mānoa (UH Mānoa) beyond filovirus vaccines (protecting against viruses such as Ebola and Marburg) to the development of vaccines to potentially combat coronaviruses, including SARS-CoV-2, the cause of COVID-19. With over $7.5M in cash, not including our State and Federal funding, we anticipate having the cash resources sufficient to achieve multiple inflection points across our rare disease pipeline, including top-line results in our SGX942 Phase 3 clinical trial in oral mucositis."

Soligenix Recent Accomplishments

On March 23, 2020, the Company announced that it expanded its research collaboration with UH Mānoa to investigate potential coronavirus vaccines. The Company and UH Mānoa are expanding the technology platform, developed as part of their filovirus program, to assess compatibility with coronaviruses including SARS-CoV-2, the cause of COVID-19. The resulting vaccines have the potential to be broadly applicable, including to individuals often excluded from common viral vector vaccine approaches such as children, the elderly and the immunocompromised. To view this press release, please click here.

On March 19, 2020, the Company announced positive preliminary top-line results for its pivotal Phase 3 FLASH trial evaluating SGX301 (synthetic hypericin) in the treatment of CTCL. The study enrolled 169 patients randomized 2:1 to receive either SGX301 or placebo, demonstrating statistically significant treatment response (p=0.04) in the Composite Assessment of Index Lesion Score (CAILS) primary endpoint assessment at 8 weeks for Cycle 1. To view this press release, please click here.

On February 13, 2020, the Company announced that its RiVax (heat stable ricin toxin vaccine) development program for prevention of ricin intoxication had received "Fast Track" designation from the FDA. To view this press release, please click here.

On February 3, 2020, the Company announced that its ongoing collaboration with the UH Mānoa and Hawaii Biotech Inc. had resulted in what the Company believes is a significant milestone in the development of heat stable filovirus vaccines, in which the platform has demonstrated feasible thermostable formulations and protection in non-human primate models with both monovalent and bivalent vaccine candidates in the three most deadly human pathogenic filoviruses (Ebola virus, Sudan virus and Marburg virus). To view this press release, please click here.

On February 3, 2020, the Company announced that the Japanese Patent Office had granted the patent titled "Novel Peptides and Analogs for Use in the Treatment of Oral Mucositis." This allowance builds on similar intellectual property in the US, New Zealand, Australia and Singapore and patent applications pending in other jurisdictions worldwide. The new claims cover therapeutic use of dusquetide (active ingredient in SGX942) and related innate defense regulator (IDR) analogs, and add to composition of matter claims for dusquetide and related analogs that have been granted in the US and worldwide. To view this press release, please click here.

On January 14, 2020, 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 December 18, 2019, the Company announced that it had 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 $850,000 in net proceeds. To view this press release, please click here.

On December 3, 2019, the Company announced it had completed patient enrollment in its Phase 3 FLASH study for SGX301 in the treatment of CTCL. The study successfully enrolled 169 subjects, following positive interim analysis, which included a prospectively defined, unblinded assessment of the study’s primary efficacy endpoint by an independent Data Monitoring Committee. To view this press release, please click here.
Financial Results – Year Ended December 31, 2019

Soligenix’s revenues for the year ended December 31, 2019 were $4.6 million as compared to $5.2 million for the year ended December 31, 2018. Revenues included payments on a contract in support of RiVax, in addition to the grants received to support the development of SGX301 for the treatment of CTCL and SGX942 for the treatment of oral mucositis in HNC.

Soligenix’s basic net loss was $9.4 million, or ($0.48) per share, for the year ended December 31, 2019, as compared to $8.9 million, or ($0.68) per share, for the year ended December 31, 2018.

Research and development expenses were $8.1 million as compared to $6.8 million for the years ended December 31, 2019 and 2018, respectively. The increase in research and development spending for the year ended December 31, 2019 was primarily attributable to the expansion of the Phase 3 clinical trial of SGX942 as well as the ongoing Phase 3 clinical trial of SGX301, compared to the same period in 2018.

General and administrative expenses were $3.4 million as compared to $3.0 million for the years ended December 31, 2019 and 2018, respectively.

As of December 31, 2019, the Company’s cash position was approximately $5.4 million.

Intensity Therapeutics Reports Safety Results from First Cohort of the KEYNOTE A10 Combination Clinical Trial of INT230-6 and Keytruda®

On March 30, 2020 Intensity Therapeutics, Inc., a clinical-stage biotechnology company developing proprietary technology and products to kill tumors and increase immune system recognition, reported successful completion of the safety lead in portion of the IT-01 KEYNOTE A10 study arm (NCT03058289) that is testing the combination of INT230-6, the Company’s lead investigational product, and Keytruda (pembrolizumab), Merck’s anti-PD-1 (programmed death receptor-1) therapy (Press release, Intensity Therapeutics, MAR 30, 2020, https://intensitytherapeutics.com/intensity-therapeutics-reports-safety-results-from-first-cohort-of-the-keynote-a10-combination-clinical-trial-of-int230-6-and-keytruda/ [SID1234556003]).

The cohort treated seven patients with different types of advanced cancers that were amenable to superficial injections including triple negative breast cancer (n=3) Merkel cell carcinoma, chordoma, desmoid tumor, and soft tissue sarcoma. Patients’s tumors were treated every two weeks for 5 doses with INT230-6 in combination with 200 mg of Keytruda every three weeks. All seven patients completed the 28 dose limiting toxicity (DLT) period with no DLT’s or drug related serious adverse events.The safety profile appears to be similar to INT 230-6 monotherapy. Following completion of the dosing of INT230-6, patients continue on Keytruda monotherapy for up to 2 years. Scans will be collected regularly on patients to evaluate the efficacy of the combination.

The study steering committee, which is comprised of the principal investigators, reviewed the safety data and approved dosing into deep tumors as well as initiating phase 2 studies. The KEYNOTE A10 phase 2 studies will enroll patients with pancreatic cancer, microsatellite stable colorectal cancer and cholangiocarcinoma, These cancers are typically immunologically cold and historically non responsive to immunotherapies. Intensity also plans a fourth phase 2 cohort to test the combination in squamous cell carcinoma patients who have already failed a PD1/PDL1 agent.

"The confirmation of safety in our first combination cohort marks an important milestone in our evaluation of INT230-6 dosed with Keytruda," commented Lewis H. Bender, President and Chief Executive Officer of Intensity Therapeutics. "We are excited to begin enrolling patients with cancers that are difficult to treat and historically nonresponsive to PD1/PDL1 antibodies alone. Patients with these cancers have limited therapeutic options and are in desperate need of better treatments."

"INT230-6 has previously demonstrated tumor cell killing and immune system activitation resulting in tumor regression in non-injected tumors in patients with several different tumor types in the monotherapy portion of our ongoing Phase 1/2 study," said Ian B. Walters, MD, Chief Medical Officer of Intensity Therapeutics. "Considering the promising results INT230-6 has demonstrated as monotherapy and the encouraging safety observed thus far in combination with Keytruda, we look forward to moving into Phase 2, treating more patients with the combination and expanding upon our existing efficacy data. We believe our approach of releasing tumor antigens derived from the patient’s own tumors to enable an immune attack on the cancer can be further amplified by blocking a checkpoint signal with Keytruda."

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, N.J., USA.

About INT230-6

INT230-6, Intensity’s lead proprietary product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRxSM technology platform. The drug is comprised of two proven, potent anti-cancer agents, cisplatin and vinblastine, and a penetration enhancer molecule that helps disperse the drugs throughout tumors for diffusion into cancer cells. In preclinical studies, INT230-6 eradicated tumors by a combination of direct tumor killing, releasing tumor antigens and recruitment of immune cells to the tumor. Results generated by the National Cancer Institute (NCI) showed treatment with INT230-6 in in vivo models of severe cancer, resulted in substantial improvement in overall survival compared to standard therapies. Further, INT230-6 provided complete responder animals with long-term, durable protection from multiple re-challenges of the initial cancer and resistance to other cancers. The NCI and Intensity collaborative research, published in July 2019, showed that there was also strong synergy when INT230-6 was combined with anti-PD-1 and anti-CTLA-4 antibodies. INT230-6 is being evaluated in a Phase 1/2 clinical study (NCT03058289) in patients with various advanced solid tumors. There have been no dose limiting adverse events observed in patients to date, even when dosing into deep tumors in the lung and liver. Several patients demonstrated tumor shrinkage, symptomatic improvement, and evidence of cancer cell death and immune cell activation on tumor biopsy.

RAPT Therapeutics Reports Fourth Quarter 2019 Financial Results and Provides Business Update

On March 30, 2020 RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in oncology and inflammatory diseases, reported financial results for the fourth quarter ended December 31, 2019 and provided an update on recent operational and business progress (Press release, RAPT Therapeutics, MAR 30, 2020, View Source [SID1234556002])

2019 was an especially productive year for RAPT with the completion of our initial public offering and the generation of encouraging early clinical data for our two lead immunology-based programs: FLX475 for the treatment of multiple cancers and RPT193 for the treatment of atopic dermatitis and other allergic inflammatory diseases," said Brian Wong, M.D., Ph.D., President and CEO of RAPT Therapeutics. "In addition, we partnered with Hanmi for FLX475 in Asia, a region with a high prevalence of ‘charged’ tumors, and we strengthened our balance sheet substantially with our follow-on offering in February 2020."

Dr. Wong continued, "For our ongoing Phase 1/2 study of FLX475, we continue to enroll and treat patients with multiple types of advanced cancer, though we are monitoring the impact of COVID-19 on our clinical trial sites both within and outside of the U.S. Because of the life-threatening nature of the cancers, we are working site by site to ensure that patients receive treatment and follow up as close to protocol-specified intervals as feasible. Our primary objective is patient safety and we will adapt to local circumstances as needed.

"For RPT193, we successfully completed the healthy volunteer portion of the study. We have made the decision to pause the enrollment of patients with atopic dermatitis in the Phase1b portion of our clinical study for RPT193 in an effort to support clinicians and healthcare facilities that are prioritizing the fight against COVID-19, while safeguarding the health and safety of patients and clinicians who would be involved in our trial. We intend to resume enrollment as soon as practical once we expect patients can be treated and followed up consistently under safer public health conditions."

Financial Results for the Fourth Quarter and Full Year Ended December 31, 2019

Fourth Quarter Ended December 31, 2019

Net loss for the fourth quarter of 2019 was $13.2 million, compared to $9.4 million for the fourth quarter of 2018.

Research and development expenses for the fourth quarter of 2019 were $10.2 million, compared to $8.4 million for the same period in 2018. The increase was primarily due to clinical costs related to the advancement of RPT193 and FLX475 as well as the personnel costs associated with these studies offset by outsourced research and development and lab supplies.

General and administrative expenses for the fourth quarter of 2019 were $2.6 million, compared to $1.3 million for the same period in 2018. The increase was due to an increase in consulting costs as well as accounting and audit-related costs as well as other expenses associated with being a public company.

Full Year Ended December 31, 2019

Net loss for the year ended December 31, 2019 was $43.0 million, compared to $36.1 million for the same period in 2018.

Research and development expenses for the year ended December 31, 2019 were $34.9 million, compared to $31.8 million for the same period in 2018. The increase was primarily due to increases in costs relating to the clinical development of RPT193 and FLX475, facilities and personnel, offset by decreases in costs relating to lab supplies and outsourced research and development.

General and administrative expenses for the year ended December 31, 2019 were $8.7 million, compared to $5.2 million for the same period in 2018. The increase was primarily due to increases in professional service fees related to preparations for our initial public offering.

As of December 31, 2019, we had cash and cash equivalents of $77.4 million. In February 2020, we received net proceeds of approximately $69.7 million resulting from our follow-on public offering of 2,500,000 shares of common stock.