On January 8, 2020 Kitov Pharma Ltd. ("Kitov") (NASDAQ/TASE: KTOV), a clinical-stage company advancing first-in-class therapies to overcome tumor immune evasion and drug resistance, reported the closing of its previously announced acquisition of privately-held, FameWave Ltd (Press release, Kitov Pharmaceuticals , JAN 8, 2020, View Source [SID1234552926]). The acquisition adds CM-24, a first-in-class inhibitor with a unique and differentiated mechanism of action of a multi-role immune checkpoint inhibition, to Kitov’s oncology pipeline. CM-24 has demonstrated a favorable safety and tolerability profile and an initial signal of monotherapy efficacy in a phase 1 clinical study. As previously announced, Kitov, in collaboration with Bristol-Myers Squibb Company, intends to initiate a phase 1b/2a clinical trial of CM-24 in combination with Bristol-Myers Squibb’s nivolumab (Opdivo) in patients with non-small cell lung cancer in the second half of 2020.

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Kitov has acquired 100% of FameWave from its shareholders in exchange for $10 million worth of Kitov’s newly issued ADSs with a long-term lock-up period, priced at $1.23 per ADS, plus 50% warrant coverage based on an exercise price of $1.98 per ADS with a 4-year term. Under the terms of the agreement, OrbiMed, Pontifax and Arkin Holdings, leading life-science focused investment funds, are exchanging their shares in FameWave for Kitov ADSs and warrants, and investing $3.5 million in Kitov in exchange for additional newly issued ADSs of Kitov. OrbiMed, Pontifax and Arkin Holdings will now each hold approximately 11% of Kitov’s shares on a non-diluted basis.

"The closing of the FameWave acquisition and the addition of CM-24 to our emerging oncology pipeline is a significant milestone for our company and seamlessly aligns with our strategic focus on the development of differentiated oncology product candidates," said Isaac Israel CEO of Kitov Pharma.

"CM-24 demonstrated encouraging signs of monotherapy anti-tumor activity in a successfully completed phase 1 trial, in addition to preclinical data that showed CM-24’s synergistic benefit with anti-PD(L)1s. This product candidate is an exciting addition to our pipeline, and we will concurrently continue to focus on advancing NT-219, our novel dual inhibitor of IRS 1/2 and STAT3, into a phase 1/2 trial for the treatment of recurrent or metastatic head and neck cancer, which we expect to commence in 2020. In addition to acquiring this promising asset, this transaction also further strengthens our leadership team and product development capabilities, as we welcome Dr. Michael Schickler, FameWave’s CEO and pharmaceutical industry veteran, to Kitov."

Dr. Schickler will join Kitov Pharma as the Head of Clinical Operations and will lead the clinical development of CM-24 and NT-219. "The initial clinical development work with the CM-24 program has suggested the antibody’s potential in overcoming the immune system exhaustion that mitigates the effectiveness and therapeutic duration of approved checkpoint inhibitors, and I am thrilled with Kitov’s commitment to advancing its development," said Dr. Schickler. "With manufacturing of the antibody well underway and having collaborated with Bristol-Myers Squibb on the phase 1/2 protocol, I look forward to the anticipated initiation of this study in the second half 2020."

This communication does not constitute an offer to sell or the solicitation of an offer to sell or the solicitation of an offer to buy any securities, nor shall 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. The securities of Kitov will be issued to the selling shareholders in FameWave, and to the investors in the cash investment transaction, on a private placement basis pursuant to applicable exemptions from the prospectus requirements under applicable Israeli securities laws and from the registration requirements of the United States Securities Act of 1933, as amended (the "U.S. Securities Act"). The securities offered have not been registered under the U.S. Securities Act or any U.S. state or Israeli securities laws, and may not be offered or sold in the United States or in Israel, or to, or for the account or benefit of, United States persons or persons in Israel absent registration or any applicable exemption from the registration and/or prospectus requirements of the U.S. Securities Act and applicable U.S. state and/or Israeli securities laws.

About CM-24
CM-24 is a clinical-stage monoclonal antibody blocking CEACAM1, a well-validated target which is highly expressed in many solid tumors as well as on immune cells and plays a pivotal role in the immune system by blocking immune cells’ access to tumors by CEACAM1-CEACAM1 and CEACAM1-CEACAM5 interaction. CEACAM1 was also shown to regulate TIM3 which induce immune fatigue. This unique mechanism of action positions CM-24 with a differentiated inhibitor of a multi-role immune checkpoint. In a monotherapy phase 1 study, CM-24 demonstrated safety and efficacy with standard dose in about 30% of patients.

On January 8, 2020 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported independent centrally reviewed top-line data for pralsetinib in patients with RET fusion-positive non-small cell lung cancer (NSCLC) (Press release, Blueprint Medicines, JAN 8, 2020, View Source [SID1234552846]). The data from the ongoing Phase 1/2 ARROW clinical trial of pralsetinib showed a 61 percent objective response rate (ORR) and prolonged durability, with a median duration of response (DOR) not reached, in patients with RET fusion-positive NSCLC previously treated with platinum-based chemotherapy. Designed by Blueprint Medicines, pralsetinib is a potent and highly selective once-daily oral inhibitor of RET fusions and mutations, including predicted resistance mutations.

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In addition, Blueprint Medicines announced it has initiated the submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for pralsetinib for the treatment of patients with RET fusion-positive NSCLC. The company expects to complete the NDA submission in the first quarter of 2020. Blueprint Medicines also plans to submit an NDA to the FDA for pralsetinib for the treatment of patients with medullary thyroid cancer (MTC) previously treated with an approved multi-kinase inhibitor in the second quarter of 2020.

"As the clinical data for pralsetinib have matured, with deep and durable responses along with robust evidence of activity against brain metastases, our confidence has continued to grow in the potential of pralsetinib to provide lasting benefit to a broad population of patients with RET fusion-positive NSCLC, including those with newly diagnosed unresectable or metastatic disease," said Andy Boral, M.D., Ph.D., Chief Medical Officer of Blueprint Medicines. "Now, with strong, centrally reviewed top-line data, we feel a profound sense of urgency and have taken the first step toward making pralsetinib broadly available to patients by initiating a rolling NDA submission to the FDA."

Top-line Data from Phase 1/2 ARROW Trial in RET Fusion-Positive NSCLC

Results from the Phase 1/2 ARROW clinical trial of pralsetinib will be used to support the NDA submission for pralsetinib for the treatment of patients with RET fusion-positive NSCLC. The registration endpoints are ORR and DOR based on independent central radiology and Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST 1.1) criteria.

Top-line efficacy data were reported for patients treated with pralsetinib who were evaluable for response assessment per RECIST 1.1, as determined by blinded independent central review. All patients received the proposed indicated dose of 400 mg once daily (QD).

In 80 patients with RET fusion-positive NSCLC previously treated with platinum-based chemotherapy, the ORR was 61 percent (95% CI: 50-72%) per independent central review (two responses pending confirmation) as of a data cutoff date of November 18, 2019. Overall, 95 percent of patients had tumor shrinkage, including 14 percent of patients with complete regression of target tumors. The median DOR was not reached (95% CI: 11.3 months, not estimable).

In 26 patients with treatment-naïve RET fusion-positive NSCLC, the ORR was 73 percent (95% CI: 52-88%) per independent central review (all responses confirmed), with 12 percent of patients achieving a complete response. All patients had tumor shrinkage.

Top-line safety data were consistent with those previously reported. Pralsetinib was well-tolerated, and most adverse events (AEs) were Grade 1 or 2. Across all patients enrolled in the ARROW trial treated with the proposed indicated dose of 400 mg QD (N=354), only four percent of patients discontinued treatment with pralsetinib due to treatment-related AEs.

Blueprint Medicines plans to present the full registration dataset at a scientific meeting later this year.

Planned Phase 3 AcceleRET Lung Trial in Treatment-Naïve RET Fusion NSCLC

In addition, Blueprint Medicines plans to initiate the first clinical trial site for its Phase 3 AcceleRET Lung clinical trial in January 2020. The primary objective of the AcceleRET trial is to evaluate the potential of pralsetinib to extend progression free survival (PFS) compared to platinum-based chemotherapy with or without pembrolizumab in patients with first-line RET fusion-positive NSCLC.

The global, randomized AcceleRET trial will enroll approximately 250 patients with advanced or metastatic RET fusion-positive NSCLC who have received no prior systemic therapy for metastatic disease. Participants will be randomized to receive either pralsetinib or the investigator’s choice of platinum-based chemotherapy regimen with or without pembrolizumab. The trial’s primary endpoint is PFS and secondary endpoints include overall survival, ORR and DOR. Patients may receive local testing to identify a RET fusion. In addition, patients randomized to the control arm may crossover upon progression to receive pralsetinib. Multiple trial sites are planned in North America, Europe and Asia.

About RET-Altered Solid Tumors

RET activating fusions and mutations are key disease drivers in many cancer types, including NSCLC and MTC. RET fusions are implicated in approximately 1 to 2 percent of patients with NSCLC and approximately 10 to 20 percent of patients with papillary thyroid cancer (PTC), while RET mutations are implicated in approximately 90 percent of patients with advanced MTC. In addition, oncogenic RET alterations are observed at low frequencies in colorectal, breast, pancreatic and other cancers, and RET fusions have been observed in patients with treatment-resistant, EGFR-mutant NSCLC.

Currently, there are no approved therapies that selectively target RET-driven cancers, although there are several approved multi-kinase inhibitors (MKIs) with RET activity being evaluated in clinical trials. To date, clinical activity attributable to RET inhibition has been uncertain for these approved MKIs, likely due to insufficient inhibition of RET and off-target toxicities. There is a need for precision therapies that provide durable clinical benefit by selectively targeting RET alterations and anticipated resistance mutations.

About Pralsetinib

Pralsetinib is an investigational, once-daily oral precision therapy specifically designed for highly potent and selective targeting of oncogenic RET alterations. Blueprint Medicines is developing pralsetinib for the treatment of patients with RET-altered NSCLC, MTC and other solid tumors. The FDA has granted Breakthrough Therapy Designation to pralsetinib for the treatment of RET-fusion positive NSCLC that has progressed following platinum-based chemotherapy, and RET-mutant MTC that requires systemic treatment and for which there are no acceptable alternative treatments.

Pralsetinib was designed by Blueprint Medicines’ research team, leveraging the company’s proprietary compound library. In preclinical studies, pralsetinib consistently demonstrated sub-nanomolar potency against the most common RET fusions, activating mutations and predicted resistance mutations. In addition, pralsetinib

demonstrated markedly improved selectivity for RET compared to pharmacologically relevant kinases, including approximately 90-fold improved potency for RET versus VEGFR2. By suppressing primary and secondary mutants, pralsetinib has the potential to overcome and prevent the emergence of clinical resistance. Blueprint Medicines believes this approach will enable durable clinical responses across a diverse range of RET alterations, with a favorable safety profile.

Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of pralsetinib, avapritinib and fisogatinib in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for all three drug candidates in the rest of the world.

On January 8, 2020 Checkmate Pharmaceuticals Inc., a clinical stage biopharmaceutical company focused upon activation of innate immunity to treat cancer, reported that it will make a presentation at the 38th Annual J.P. Morgan Healthcare Conference (Press release, Checkmate Pharmaceuticals, JAN 8, 2020, View Source [SID1234552864]). The company will provide a corporate update on Wednesday, January 15, 2020 at 4:30 pm PT at the Westin St. Francis in San Francisco.

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On January 8, 2020 Transcenta Holding Limited ("Transcenta"), a global biotherapeutics company with fully-integrated capabilities in discovery, R&D, and manufacturing of antibody-based therapeutics, reported that it has sealed a 100 million USD series B+ round (Press release, Transcenta, JAN 8, 2020, View Source [SID1234552880]). CR-CP Life Science Fund ("CR-CP") and Fortune Capital co-led the financing round, participated by new investors including Epiphron Capital, CCT China Merchant Buyout Fund, China Equity Group, as well as the existing shareholders including Lilly Asia Ventures, Temasek, Hillhouse Capital, Teng Yue Partners, Sequoia Capital China, and ARCH Venture Partners. China Renaissance acted as the exclusive financial advisor for this transaction.

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Since the merger at the beginning of last year, Transcenta has made significant progresses, for example, it has built a well-rounded team with extensive global industrial experience, assembled a robust pipeline with over 10 innovative molecules in oncology, bone disorders and nephrology, and adopted integrated continuous process and manufacturing in its Hangzhou facility, signed an exclusive license agreement for Greater China with Eli Lilly and Company regarding a portfolio of novel antibodies for bone diseases, including the phase 2-completed Blosozumab for osteoporosis, etc.

"We very much appreciate the continuous support from our existing investors and the trust from new investors. The proceeds from new funding will be used to advance lead programs, including a second generation Claudin 18.2 antibody, a PDL1-TGFβ bispecific antibody and other oncology assets with undisclosed targets, as well as Blosozumab, a transformative agent for the treatment of severe osteoporosis. In addition, Transcenta is to further expand its continuous manufacturing capability and employ it at full scale in order to reduce cost of goods, as an important competitive edge. Finally, we are planning to strengthen our senior management team to prepare for IPO," commented Dr. Xueming Qian, Transcenta’s Co-Founder and CEO.

"This is the first and successful financing round of Transcenta as the merged company. We are very thankful in particular to our new investors for their faith in us. And we will focus our resources on delivering our pipeline," said Transcenta’s Co-Founder and Executive Chairman Dr. Jonathan Y. Zhao. "Transcenta has become a fully integrated biotherapeutics company with comprehensive capabilities. We are committed to transcending the best and bringing quality and affordable biotherapeutics to all the patients."

Mr. Da Liu, Managing Director of CR-CP, stated "We are pleased to team up with other prominent investors investing in Transcenta and supporting the elite team anchored by the two top minds, Dr. Qian and Dr. Zhao. Transcenta has firmly established itself as an integrated biotech company with cutting-edge technology platforms ranging from discovery to manufacturing. We share the Company’s mission to providing patients worldwide with innovative and affordable biologics, and will actively extend resources from CR and CP to support the Company’s growth. I have confidence that this company will become a unicorn."

Mr. Shen Qi, Senior Vice President and Head of Healthcare Investment at Fortune Capital, said "We appreciate having the chance to support Transcenta team in this financing round together with the other investors. Transcenta has built up, through its integrated technology platform, an innovative and robust pipeline with huge potentials. We believe that Transcenta will become the next-generation biotech company with the most competitive edges by adhering to its core advantages as ‘innovation, quality, speed and cost-efficiency.’"

Kevin Xie, Managing Director of China Renaissance, stated "We are honored and excited advising Transcenta in its first and successful financing round upon the merger. We believe with its integrated capabilities and the world-class management, Transcenta is to become the flagship company in biotherapeutics arena."

On January 8, 2020 NexImmune, a clinical-stage biopharmaceutical company developing novel immune-therapeutics based on a proprietary Artificial Immune Modulation (AIM) nanotechnology platform, reported that industry veterans Han Myint, MD, and John Trainer, MBA, have been appointed as the Company’s Chief Medical Officer and Chief Financial Officer, respectively (Press release, NexImmune, JAN 8, 2020, View Source [SID1234554873]).

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Dr. Myint commented, "I am very excited to join NexImmune at such a time where I can leverage my professional experience to oversee the Company’s first two clinical trials for NEXI-001 and NEXI-002 in patients with relapsed forms of Acute Myeloid Leukemia (AML) and Multiple Myeloma, respectively. The Company has developed a novel technology that direcects endogenous T cells against multiple tumor antigen targets. For patients suffering from a variety of cancers, I believe this approach has the potential to deliver potent anti-tumor activity while minimizing potential off-target toxicities, and I am looking forward to working with team to make this a reality for these patients."

Trainer commented, "The tremendous potential of the NexImmune AIM platform in multiple types of cancer and other disease areas is a significant growth opportunity. Scaling up and financing this growth through additional capital raises, partnerships and a potential IPO will be a top priority. I’m excited to be a part of the NexImmune team as we push forward on our mission to help patients."

Dr. Myint most recently served as the Vice President of Global Medical Affairs – Myeloid Diseases at Celgene. While in that role, he led the Global Franchise Team – Myeloid, and worked very closely with cross-functional teams representing commercial, market access, regulatory, translational and clinical functions to create a cohesive global disease strategy for all development stage compounds in the Myeloid disease pipeline including AML, Myelodysplastic Syndrome (MDS), Myelofibrosis and Thalassemia. Prior to his six year tenure at Celgene, Dr. Myint practiced academic hematology at multiple prestigious institutions, including Rush University in Chicago, and became a Fellow at the Royal College of Physicians and Royal College of Pathologists in London, England. He has expertise and clinical experience in hematological malignancies and stem cell transplantation. Additionally, Dr. Myint built a FACT-accredited and Center of Excellence-Designated Transplant Program at the University of Colorado, Denver, while becoming a fellow of the American College of Physicians.

Trainer most recently served as Vice President and Head of Partnering & Strategy for MedImmune, the biologics-focused R&D unit of AstraZeneca. There, he led all in-licensing, out-licensing and collaboration work for MedImmune including such transactions as the spin-out of Viela Bio (which raised a $250m Series A). Trainer was also responsible for working with partners such as Johns Hopkins University and the NIH to help develop promising new companies and technologies. Previous to that role, Trainer was Vice President, Corporate Development for AstraZeneca and the commercial leader for AstraZeneca’s Infection, Neuroscience and Gastrointestinal group globally, as well as holding a variety of financial, transactional and commercial roles at MedImmune. He received his MBA from Harvard Business School and his undergraduate degree from Harvard College.

Scott Carmer, President and CEO of NexImmune, commented on the additions to the Company’s Executive Leadership Team, "I am thrilled to have both Han and John join our team. Individually, each is a highly respected and accomplished industry leader. Together, their combined experience, expertise and leadership will be a strong complement to those of our existing executive team. As we initiate our first clinical trials in 1Q2020, NexImmune is now poised to exploit the full potential of our unique technology platform for the benefit of patients in need."