On December 16, 2019 Medivir AB (Nasdaq Stockholm: MVIR) reported that the independent safety committee (IDMC) for the phase II study of birinapant in combination with pembrolizumab (Keytruda) in patients with MSS colorectal cancer has performed the planned futility analysis (Press release, Medivir, DEC 16, 2019, View Source [SID1234552410]). The primary objective of the phase II study was an improved clinical response to treatment measured as 20% ORR (Overall Response Rate). IDMC’s recommendation is that the study should be terminated as the analysis indicates that it is unlikely that the study’s objectives will be met. Medivir has therefore decided to discontinue the recruitment of patients and to end the study.

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"We are disappointed that the combination therapy with birinapant and pembrolizumab did not work better in this difficult-to-treat patient group where monotherapy with pembrolizumab has very limited effect", said Dr Uli Hacksell, CEO of Medivir. "But we still see a potential for other combination therapies where birinapant could offer patients improved treatment. One example is the ongoing phase I study of a combination of birinapant and radiation therapy in patients with head and neck cancer."

A total of 15 patients with advanced MSS colorectal cancer were recruited in the phase II study, 14 of which were included in the futility analysis. The combination therapy was considered acceptable from a safety perspective, but none of the patients showed a clear clinical efficacy response. Four patients were judged to have stable disease while 10 were judged to have disease progression.

The phase II study was conducted as a collaboration between Medivir and MSD, who provided pembrolizumab.

For further information, please contact:
Uli Hacksell, CEO, Medivir AB, phone: +46-(0)-8-5468-3100.

Medivir AB is obliged to make this information public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 16.00 CET on 16 December, 2019.

About Birinapant

Birinapant is being developed to enhance responses, and extend survival, of patients with solid tumors where existing treatments do not provide sufficient survival benefit, or where patients no longer have treatment options. Based on its unique design and mechanism, birinapant has the potential to enhance patients’ responses in combination with other treatments.

On December 16, 2019 Platelet BioGenesis, Inc. (PBG), the leader in stem cell-derived, on-demand human platelets (PLTs+) and genetically engineered platelet-based therapeutics, reported it received an award of $2.3 million from the Medical Technology Enterprise Consortium (MTEC) – a 501(c)(3) biomedical technology consortium collaborating with the U.S. Army Medical Research and Development Command (USAMRDC) (Press release, Platelet BioGenesis, DEC 16, 2019, View Source [SID1234552409]). PBG will use the funding to accelerate the development of an automated, mobile bioreactor that produces PLT+ packs for use at field hospitals and other military medical posts.

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MTEC partners with private industry, academic institutions, government agencies and other research organizations seeking to accelerate the development of medical solutions that prevent and treat injuries to U.S. military members. One of the organization’s current objectives is to identify devices or therapies that empower medics to do more treatment at the point of injury. MTEC’s work with PBG will enable the development of a mobile bioreactor that can produce PLTs+ on-demand in the regions where military operations are underway, alleviating platelet shortages in conflict zones caused by lack of donor availability, short platelet shelf life and sterility concerns.

"Military conflict zones are often in remote locations, meaning that medical technology innovations are paramount to effective interventions and recoveries," said Kathy Zolman, MTEC Director of Program Operations. "The work Platelet BioGenesis is doing could help resolve the shortages that often affect U.S. military units. Developing the bioreactor for remote applications would enable platelets to be available on-demand, which would make an immense difference in treating traumatic injuries sustained during military activity."

In another important development, PBG has added Uma Lakshmipathy, Ph.D. and Teresa Foy, Ph.D. to its Scientific Advisory Board (SAB) to support the continued research and development of platelet-based therapeutic solutions. Dr. Lakshmipathy is the R&D Director at Thermo Fisher Scientific and is involved in the development of reprogramming, engineering and characterization solutions that support translation of primary and stem cells towards novel cell therapy platforms. Dr. Foy is Senior Vice President at Bristol-Myers Squibb (BMS, formerly Celgene), leading the Thematic Research Center for Immuno-Oncology (IO) and Cellular Therapy (CT). She is responsible for BMS’s IO/CT discovery and early development pipeline, leading a team that is advancing novel drug and cell therapy candidates from discovery through clinical proof-of-concept.

"Dr. Lakshmipathy and Dr. Foy bring to PBG extensive experience in the development of cell therapies, which will be invaluable as we enter IND-enabling studies of our human stem cell-derived, donor-independent platelets in 2020. We look forward to working closely with both of these distinguished industry veterans as we continue to advance our novel platelet-based cell therapy platform," said Sam Rasty, Ph.D., the company’s President and CEO. "In addition to bolstering our SAB with these highly experienced advisors, the MTEC award provides us with further non-dilutive funding and validation of our science and technology platform. We look forward to partnering with MTEC to advance the development of our mobile, automated PLT+ bioreactor."

On December 16, 2019 CEL-SCI Corporation (NYSE American: CVM) reported financial results for the fiscal year ended September 30, 2019 (Press release, Cel-Sci, DEC 16, 2019, View Source [SID1234552408]). The Company also reported key clinical and corporate developments achieved during fiscal 2019.

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Clinical and Corporate Developments included:

In March and October 2019, the Independent Data Monitoring Committee (IDMC) for the Company’s pivotal Phase 3 head and neck cancer study of its investigational immunotherapy Multikine* (Leukocyte Interleukin, Injection) had an official review of the study data and recommended that the trial continue until the appropriate number of events has occurred. The data from all 928 enrolled patients were provided to the IDMC by the clinical research organization (CRO) responsible for data management of this Phase 3 study. At the most recent IDMC meeting in October 2019 the IDMC reviewed "progression free and overall survival and limited demographic and safety data available for the aforementioned protocol."
CEL-SCI is now awaiting final study results in its Phase 3 head and neck cancer trial. All that remains to be done in this pivotal Phase 3 study, the largest in the world in head and neck cancer, is to continue to track patient survival until it can be determined if the primary endpoint of the study, a 10% improvement in overall survival of the Multikine* treatment regimen plus Standard of Care (SOC) vs. SOC alone will be met. The primary endpoint will be determined after a total of 298 events (deaths) have occurred in the two main comparator arms of the study and have been recorded in the study database. These final results could be available soon, since the last cancer patients were treated in September 2016, and the first cancer patients in the study were treated in early 2011.
On May 11, 2019 and July 3, 2019, new data were presented on CEL-SCI’s experimental LEAPS therapeutic antigen-specific treatment for rheumatoid arthritis. The work was performed in conjunction with researchers at Rush University Medical Center, Chicago, Illinois. In June 2019, CEL-SCI was also an exhibitor and showcased its presentation at the BIO International Convention where it was selected to be part of the Innovation Zone sponsored by the U.S. National Institutes of Health (NIH). The focus was the Company’s experimental LEAPS platform technology and CEL-SCI’s ongoing development of a LEAPS based therapeutic antigen-specific treatment for rheumatoid arthritis.
The U.S. Patent and Trademark Office granted CEL-SCI two patents for its LEAPS technology during fiscal 2019. As announced in January 2019, the patents relate to methods for diagnosing, preventing, and treating disease by generating or modulating the immune response through the use of specific peptides.
Two scientific articles regarding CEL-SCI’s LEAPS program were published in March and July 2019. The Journal of Clinical & Cellular Immunology published "Why Don’t We Have a Vaccine Against Autoimmune Diseases?" co-written by Dr. Ken Rosenthal of Roseman University College of Medicine, and CEL-SCI’s Roy Carambula, Research Associate and Daniel Zimmerman Ph.D., Senior Vice President of Cellular Immunology. International Immunopharmacology published "Lessons From Next Generation Influenza Vaccines For Inflammatory Disease Therapies" authored by Dr. Zimmerman and two other CEL-SCI scientists, along with Dr. Rosenthal of Northeast Ohio Medical University and Roseman University.
On June 28, 2019, CEL-SCI joined the broad-market Russell 3000 Index that was effective after the US market opened on July 1, 2019.
CEL-SCI raised approximately $14.5 million during fiscal 2019 through the exercise of warrants.
"Following the two most recent data reviews of our Phase 3 head and neck cancer trial, the Independent Data Monitoring Committee (IDMC) recommended we continue the study until the appropriate number of events (deaths) have occurred in the two main groups. We found this recommendation very encouraging because it has been almost nine years since our study started, and more patients in our trial are surviving longer than had been anticipated," stated CEL-SCI CEO, Geert Kersten.

"Since CEL-SCI is blinded to the study results, we do not know what proportion of the patients who are living longer than expected were treated with the Multikine regimen and what proportion received Standard of Care. However, it seems reasonable that a significant proportion of the patients who are living longer than predicted would have received the Multikine regimen because, to our knowledge, there has been no reported improvement in survival of the Standard of Care patients since our study began. We also believe the IDMC would not have recommended that we continue with this Phase 3 study if, after review of the clinical data, they had not seen that Multikine helps these cancer patients."

"The goal of our Phase 3 study is to keep patients alive longer by improving the current ‘intent to cure’ first line cancer treatment. We believe the delay in reaching the required number of events, 298, to conclude our study may be a predictor of a better overall survival outcome than we originally hypothesized. Yervoy, the first approved cancer immunotherapy blockbuster drug had a similar situation in its Phase 3 trial and this turned out to be a huge blessing for both patients and Bristol-Myers Squibb shareholders. At CEL-SCI, we are hoping for a similar outcome," Kersten concluded.

CEL-SCI reported a net loss of $22.1 million in fiscal year 2019 versus a net loss of $31.8 million in fiscal 2018. The decreased net loss in 2019 was mainly due to the non-cash derivative loss of approximately $0.8 million and $8.6 million recorded during the years ended September 30, 2019 and 2018, respectively, and the decrease in net interest expense of approximately $2.4 million during the year ended September 30, 2019 compared to the year ended September 30, 2018. The decrease in interest expense was primarily due to interest incurred relating to CEL-SCI’s convertible debt, all of which was converted by September 30, 2018. The derivative loss variation was the result of the change in fair value of the derivative liabilities during the period which was caused by an increase in the share price of CEL-SCI’s common stock.

The Company’s audited financial statements contained an audit opinion from its independent registered public accounting firm that included an explanatory paragraph related to the Company’s ability to continue as a going concern.

On December 16, 2019 NANOBIOTIX (Paris:NANO) (Euronext: NANO – ISIN: FR0011341205 – the "Company"), a clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer, reported receipt of the French 2019 Prix Galien Award for Most Innovative MedTech (Press release, Nanobiotix, DEC 16, 2019, View Source [SID1234552407]). The Company’s lead product, HENSIFY (NBTXR3) brand name for the treatment of locally advanced Soft Tissue Sarcoma (STS), was recognized after receiving European market approval (CE marking, DM class III, on April 2, 2019) earlier this year. The Prix Galien Award recognizes outstanding biomedical and medical technology product achievements that improve the human condition.

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HENSIFY is a first-in-class "radioenhancer" consisting of an aqueous suspension of crystalline hafnium oxide nanoparticles. The product is administered only once, directly into the tumor, before a patient’s first radiotherapy session. After intratumoral injection, the nanoparticles penetrate the tumor cells and, when activated by ionizing radiation, deliver a larger energy deposit within the tumor where the nanoparticles are present, thereby increasing the tumor-killing effect of treatment without increasing damage to surrounding healthy tissues. HENSIFY has a universal, physical mode of action and is inert within the human body outside of the presence of ionizing radiation.

The product is approved in Europe for the treatment of Soft Tissue Sarcoma (STS). Positive results from the Company’s phase III STS study were featured the August 2019 edition of The Lancet Oncology.

Moving forward, the Company is engaged in global clinical development of the product across fifteen (15) clinical trials in STS and other indications with a primary focus on global registration for the treatment of Head and Neck cancers. These trials include the expansion phase of a European phase I evaluating the safety and feasibility of NBTXR3 activated by radiation therapy(Study NBTXR3-102); an immuno-oncology (I/O) basket trial (Study NBTXR3-1100) which evaluates NBTXR3 in combination with anti-PD-1 in the United States (US); phase I trials evaluating NBTXR3 activated by radiation therapy for the treatment of liver and prostate cancer; a partnership with PharmaEngine in Asia evaluating NBTXR3 in combination with cisplatin; and a clinical collaboration with The University of Texas MD Anderson Cancer Center (MD Anderson) including nine (9) trials across several indications.

About the PRIX GALIEN

Created in France in 1970, the Prix Galien is the most prestigious award in the field of pharmaceutical research and innovation. Referred to as the "Nobel Prize of pharmaceutical research, it recognizes the efforts and achievements of pharmaceutical research and development.

About NBTXR3

NBTXR3 is a first-in-class product designed to destroy tumors through physical cell death when activated by radiotherapy. NBTXR3 has a high degree of biocompatibility, requires one single administration before the first radiotherapy treatment session, and has the ability to fit into current worldwide standards of radiation care. The physical mode of action of NBTXR3 makes it applicable across solid tumors such as lung, prostate, liver, glioblastoma, and breast cancers.

NBTXR3 is actively being evaluated in head and neck cancer with locally advanced squamous cell carcinoma of the oral cavity or oropharynx in elderly and frail patients unable to receive chemotherapy or cetuximab with limited therapeutic options. Promising results have been observed in the phase I/II trial regarding local control. In the United States, the company has started the regulatory process in regard to the clinical authorization to the phase II/III in locally advanced head and neck cancers.

Nanobiotix is also running an Immuno-Oncology development program. The Company received approval FDA to launch a clinical trial of NBTXR3 activated by radiotherapy in combination with anti-PD-1 antibodies in locoregional recurrent (LRR) or recurrent and metastatic (R/M) head and neck squamous cell carcinoma (HNSCC) and lung or liver metastasis (mets) with HNSCC not amenable to re-irradiation or non-small cell lung cancer (NSCLC) as the primary tumor.

The other ongoing NBTXR3 trials are treating patients with liver cancers (hepatocellular carcinoma and liver metastasis), locally advanced or unresectable rectal cancer in combination with chemotherapy, head and neck cancer in combination with concurrent chemotherapy, and prostate adenocarcinoma. Furthermore, the company has a large-scale, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center (9 new phase I/II clinical trials in the United States) to evaluate NBTXR3 across head and neck, pancreatic, thoracic, lung, gastrointestinal and genitourinary cancers.

On December 16, 2019 Organovo Holdings, Inc. ("Organovo") (Nasdaq: ONVO) and Tarveda Therapeutics, Inc. ("Tarveda"), a privately-held, 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 that they have entered into a definitive merger agreement under which Tarveda would merge with a wholly-owned subsidiary of Organovo in an all-stock transaction (Press release, Organovo, DEC 16, 2019, View Source [SID1234552406]). Upon completion of the merger, the merged company would operate under the name Tarveda Therapeutics, Inc. and trade on the Nasdaq Stock Market LLC under the ticker symbol "TVDA."

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Tarveda is primarily focused on the development of its pipeline of Pentarin miniature drug conjugates designed to selectively accumulate and retain anti-cancer payloads in solid tumor malignancies. Following the closing of the merger, Tarveda intends to continue to focus on advancing its two clinical stage oncology programs, PEN-866 and PEN-221, and on further development of novel conjugates from its proprietary miniature drug conjugate platform. At the closing of the merger, it is estimated that the combined company will have approximately $35 million of cash on hand that is expected to provide sufficient funding into the second half of 2021 to achieve key upcoming clinical data milestones on both clinical programs.

"After completing an extensive and thorough review of strategic alternatives, we are extremely pleased to announce this transaction with Tarveda, which we believe is in the best interest for our stockholders," said Taylor J. Crouch, President and Chief Executive Officer, Organovo. "Tarveda is advancing an innovative pipeline of clinical stage cancer therapies derived from the company’s proprietary miniature drug conjugate platform. Tarveda is supported by a strong syndicate of investors including Novo A/S, Versant Ventures and ND Capital (NanoDimension) and a highly seasoned management team with prior public company experience."

"Our growing portfolio of miniature drug conjugates has the potential to represent much needed new treatment options for patients with solid tumor malignancies," said Drew Fromkin, President and Chief Executive Officer of Tarveda. "We are encouraged by the activity and tolerability demonstrated in Phase 1 human studies of our two clinical programs, PEN-866 and PEN-221. Our Pentarin miniature drug conjugates are designed to incorporate the best properties of small molecule drugs and antibody drug conjugates to form miniature drug conjugates that are effective at rapidly and deeply penetrating solid tumors while minimizing damage to healthy tissue. We are excited about this merger with Organovo and believe that this is the right point in Tarveda’s trajectory to move forward as a publicly traded company given several upcoming clinical data milestones that we expect to be achieved in 2020 and 2021."

Tarveda expects the merger to provide the capital required to advance its two lead programs through the next set of clinical milestones and to generate novel conjugates from its Heat Shock Protein 90 (HSP90) binding miniature drug conjugate platform. PEN-866, the initial clinical program from Tarveda’s HSP90 binding miniature drug conjugate platform, is designed to bind to the activated form of HSP90 in solid tumors to accumulate and retain its potent topoisomerase 1 inhibitor (SN-38) payload. PEN-866 is completing the Phase 1 dose escalation and safety portion of its "all comers" trial in various types of solid tumors and has shown to be well tolerated and demonstrated early clinical activity in heavily treated, advanced patients with a range of solid tumor malignancies. Beginning in early 2020, it is expected that PEN-866 will be evaluated in a Phase 2a study both as a single agent and as a combination therapy across a range of solid tumors that are sensitive to topoisomerase 1 inhibitors. PEN-221 is a miniature drug conjugate in clinical evaluation for the treatment of patients with solid tumors expressing somatostatin receptor 2 (SSTR2) on the cell surface and is linked to the potent tubulin inhibitor payload, DM1. In a Phase 1 study, PEN-221 was well tolerated and demonstrated early clinical activity. PEN-221 is currently being evaluated in a Phase 2a study for the treatment of patients with neuroendocrine tumors and small cell lung cancer.

About the Proposed Merger

Under the terms of the merger, it is anticipated that Tarveda stockholders will own approximately 75% of the combined company and current Organovo stockholders will own approximately 25% of the combined company on a fully-diluted basis. The exchange ratio is based on valuation assumptions for both companies subject to potential adjustments for certain financial metrics prior to the completion of the merger.

The transaction has been approved by the boards of directors of both companies. The merger is anticipated to close in the first quarter of 2020, subject to the approval of Organovo and Tarveda stockholders as well as other customary closing conditions.

Roth Capital Partners served as financial advisor, and Gunderson Dettmer Stough Villeneuve Franklin & Hachigian, LLP served as legal counsel to Organovo. Canaccord Genuity served as financial advisor, and Cooley LLP served as legal counsel, to Tarveda.

Management and Organization

Following the merger, the combined company will be led by the current Tarveda management team, including Drew Fromkin as President, Chief Executive Officer and Chairman; Jeffrey D. Bloss, M.D., Chief Medical Officer; Brian Roberts, Chief Financial Officer; Mark Bilodeau, Ph.D., Chief Scientific Officer; and Sudhakar Kadiyala Ph.D., Executive Vice President, Strategy.

The Board of Directors of the combined company will be comprised of eight directors, including six directors to be named by Tarveda and two directors to be named by Organovo. The corporate headquarters will be located in Watertown, MA.

Conference Call

Organovo and Tarveda will host a conference call at 8:30 a.m. ET on December 16, 2019, to discuss the proposed transaction. The conference call may be accessed by dialing (866) 405-4577 (domestic) or (602) 563-8680 (international) and using the conference ID 3679123. To help ensure the conference call begins in a timely manner, please dial in five minutes prior to the scheduled start time. The conference call will also be simultaneously webcast at View Source

Non-Solicitation

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

Important Information and Where to Find It

This communication may be deemed to be solicitation material in respect of the proposed transaction between Organovo and Tarveda. In connection with the proposed transaction, Organovo intends to file relevant materials with the SEC, including a registration statement on Form S-4 that will contain a proxy statement/prospectus/information statement. BEFORE MAKING ANY VOTING OR INVESTMENT DECISION, INVESTORS AND STOCKHOLDERS ARE URGED TO READ THESE MATERIALS CAREFULLY AND IN THEIR ENTIRETY WHEN THEY BECOME AVAILABLE BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT THE PROPOSED TRANSACTIONS. Stockholders may obtain, free of charge, copies of the definitive proxy statement and any other documents filed by Organovo with the SEC in connection with the proposed transactions at the SEC’s website (View Source) and at Organovo’s website.

Organovo and its directors and executive officers and Tarveda and its directors and executive officers may be deemed to be participants in the solicitation of proxies from the stockholders of Organovo in connection with the proposed transaction. Information regarding the special interests of these directors and executive officers in the merger will be included in the proxy statement/prospectus/information statement referred to above. Additional information regarding the directors and executive officers of Organovo is included in Organovo’s Definitive Proxy Statement on Schedule 14A relating to the 2019 Annual Meeting of Stockholders, filed with the SEC on July 26, 2019. This document is available free of charge at the SEC website (www.sec.gov) or at Organovo’s website.