On May 31, 2019 the cancer vaccine company Ultimovacs ASA reported that it has raised NOK 370 million in an oversubscribed share issue, and will be listed on the Oslo Stock Exchange on Monday, June 3, 2019 (Press release, Ultimovacs, MAY 31, 2019, View Source [SID1234536989]).

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Investors showed great interest in Ultimovac’s listing on the Oslo Stock Exchange and the issue was oversubscribed. Large Norwegian and international institutional investors have entered the company as new shareholders. Among the new, large shareholders are also specialized health funds. Ultimovacs experienced a broad interest in subscription of shares and the company will have about 1,500 shareholders on listing.

"It is very gratifying for Norwegian health care and for Ultimovacs that national and international investors show the company this confidence. In today’s uncertain market, it is extra pleasant with such great interest, both from international investors and small savers. I look forward to following the company further, "says Jonas Einarsson, chairman of Ultimovacs and CEO of Radforsk.

The funds Ultimovacs collect at the IPO will finance the company’s development plan for the universal cancer vaccine UV1. The main project is a major clinical study to document the treatment effect of the vaccine. In this study, UV1 will be combined with other immunotherapy in patients with melanoma at about 30 hospitals in Norway, Europe, the United States and possibly Australia.

Ultimovacs has already conducted three minor clinical trials of the vaccine at the Radium Hospital in patients with lung cancer, prostate cancer and mole cancer.

«The cancer vaccine has shown promising clinical signals in the studies we have conducted at Radiumhospitalet. Based on this, we have developed a development plan to document that our vaccine has an effect on cancer patients. I am very pleased that we have now completed a successful stock exchange listing. This means that the practical conditions are now in place to implement our development plan, "says Øyvind Kongstun Arnesen, CEO of Ultimovacs.

On May 31, 2019 OncoArendi Therapeutics will present and promote its programs during the 2019 BIO International Convention in Philadelphia, USA, recognized as one of the world’s most important events in the biotechnology and pharmaceutical industry. Over 16,000 participants from 67 countries will attend the conference (Press release, OncoArendi Therapeutics, MAY 31, 2019, View Source [SID1234536728]).

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OncoArendi represented by: Marcin Szumowski, CEO, Nicolas Beuzen, Director of Business Development and Pawel Dobrzanski, Senior Scientific Director has over 40 formally accepted meetings, during which two of the Company’s most advanced assets will be presented:

OATD-01, a drug developed in inflammation-driven fibrotic diseases currently completing phase Ib of clinical development, as well as
OATD-02, an arginase inhibitor for cancer treatment, currently completing IND-enabling studies.
– During this event we will meet new prospects potentially interested in global or regional (i.e. Asian) rights, and continue ongoing discussions, initiated at previous events, some already under confidentiality agreements. At the same time scouting will be conducted to identify novel molecules that could complement our portfolio of early stage programs – says Marcin Szumowski, CEO OncoArendi Therapeutics.

– We are keeping our eye on the global biotechnology market that grows at an increasing pace. Many new innovative ideas appear in areas directly related to research projects currently pursued by OncoArendi. Inhibitors of deubiquitinases, a family of proteins representing potential targets in cancer therapy is just one example. These targets have been recently pursued by several mid-size biotech companies, confirming the attractiveness of these new molecular targets in treatment of various diseases.

On May 31, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported that two presentations on omidubicel1 (formerly known as NiCord), an investigational advanced cell therapy in Phase 3 clinical development designed to enhance the life-saving benefits of hematopoietic stem cell (bone marrow) transplant, took place at the International Society for Cell and Gene Therapy (ISCT) 2019 Annual Meeting being held in Melbourne, Australia (Press release, Gamida Cell, MAY 31, 2019, Gamida Cell Announces Data for Omidubicel Presented at the International Society for Cellular and Gene Therapy 2019 Annual Meeting
[SID1234536776]). The presentations included a summary of clinical and translational data from the completed Phase 1/2 clinical study of omidubicel in patients with high-risk hematologic malignancies, or blood cancers. Results from the Phase 1/2 study showed that patients transplanted with omidubicel had rapid and durable engraftment of neutrophils and platelets, as well as prompt immune reconstitution. An international, randomized Phase 3 study of omidubicel in patients with hematologic malignancies is currently ongoing.2

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"In the Phase 1/2 clinical study, patients who received omidubicel had a clinically meaningful reduction in their time to neutrophil and platelet recovery compared to a real-world cohort of patients who received a standard umbilical cord blood transplant. The neutrophil recovery observed with omidubicel also resulted in fewer days spent in the hospital compared to the comparator cohort," said Joanne Kurtzberg, M.D., Director of the Marcus Center for Cellular Cures and the Carolinas Cord Blood Bank at Duke University Medical Center. "These data suggest an important potential step toward making stem cell transplantation safer and more accessible to patients with lethal blood cancers, and I am pleased to be participating in the Phase 3 study currently enrolling patients."

Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients in the U.S. do not receive one for various reasons, including finding a matched donor.3 Even for patients who do receive a transplant, treatment is not always effective and can lead to serious complications that can dramatically affect quality of life.4 Omidubicel is intended to address the current limitations of bone marrow transplant by providing a therapeutic dose of cells while preserving the cells’ functional therapeutic characteristics.

"At Gamida Cell, our aspiration is to bring the first FDA-approved cell therapy for bone marrow transplantation to patients," stated Ronit Simantov, M.D., chief medical officer at Gamida Cell. "These data demonstrate the potential of omidubicel to give patients with high-risk blood cancers, particularly those who would not otherwise receive a bone marrow transplant from a matched donor, an opportunity for a cure."

Data Presented at ISCT 2019 Annual Meeting

Phase 1/2 Clinical Data

The presentation, "NiCord, an Expanded Cord Blood Product, Accelerates Engraftment After Myeloablative Conditioning," described results from the completed multicenter, Phase 1/2 clinical trial of omidubicel in 36 patients with high-risk hematologic malignancies and no readily available matched sibling or matched unrelated adult donor. The key primary endpoint was the cumulative incidence of neutrophil engraftment at 42 days. Additionally, the omidubicel patient cohort was compared to a retrospective cohort of patients who received standard cord blood transplant using data from the Center for International Blood and Marrow Transplant Research (CIBMTR).

Data from the study demonstrated that patients transplanted with omidubicel had rapid and durable engraftment of neutrophils and platelets. The age-adjusted cumulative incidence of neutrophil engraftment at 42 days following transplantation was 94 percent for omidubicel recipients compared to 85 percent for the CIBMTR cohort. Among patients who engrafted, the median time to neutrophil recovery was 11.5 days (95 percent confidence interval (CI): 9-14 days) for omidubicel recipients compared to 21 days (95 percent CI: 20-23 days) for the CIBMTR cohort (p < 0.001). For patients achieving platelet recovery, the median time to platelet recovery was 34 days (95 percent CI: 32-42 days) and 46 days (95 percent CI: 42-50 days) for the omidubicel and CIBMTR cohorts, respectively (p < 0.001). Omidubicel demonstrated an acceptable safety profile, with hypertension reported as the most common adverse event attributable to omidubicel infusion, and moderate to severe chronic graft vs. host disease reported in 9.8 percent of patients at one year following transplantation. Primary hospital discharge occurred at a median of 20 days following transplantation. Omidubicel recipients spent a median of 73 days alive and out of hospital during the first 100 days following transplantation.

Phase 1/2 Translational Data

The presentation, "Rapid and Robust CD4+ and CD8+ T-, NK-, B- and Monocyte Reconstitution after Nicotinamide-Expanded Cord Blood Transplantation," described in-depth immune reconstitution data from the completed Phase 1/2 clinical study of omidubicel. Immune reconstitution for 27 patients receiving omidubicel was compared to retrospective cohorts of adolescent and young adults with hematologic malignancies receiving unmanipulated cord blood transplantation (unCBT, n = 27) or unrelated bone marrow transplantation (BMT, n =20). The primary endpoint was the probability of achieving CD4+ immune reconstitution (> 50×106/L) within the first 100 days. Secondary endpoints included the recovery of B cells, CD4+ T cells and natural killer (NK) cells during the first year after transplantation. Analyses were performed at the University Medical Centre Utrecht, Laboratory of Translational Immunology.

The analysis showed that 91 percent of patients receiving omidubicel achieved successful immune reconstitution of CD4+ T cells at 100 days after transplantation. Reconstitution of T cells in the omidubicel group (median age 41.5 years) was similar to the unCBT and BMT cohorts (median age 15.4 and 14.3 years, respectively), despite the younger age of the cohorts, who would be expected to reconstitute faster. In addition, reconstitution of a number of cell types, including B cells (p = 0.026) and NK cells (p < 0.001), was significantly faster after transplantation with omidubicel compared to the cohorts, and suggests that omidubicel reconstitutes diverse functions of the immune system.

About Omidubicel

Omidubicel (formerly known as NiCord), the company’s lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers).1 Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well-tolerated.5 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Europe and Asia.2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.6 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On May 31, 2019 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing novel drugs to address treatment resistance, reported updated interim data from its ongoing Phase 1/2 TRIDENT-1 clinical study of lead drug candidate repotrectinib in ROS1-positive non-small cell lung cancer (NSCLC) patients (Press release, Turning Point Therapeutics, MAY 31, 2019, View Source [SID1234536729]).

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The results, which will be presented today during an oral session at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), demonstrate ongoing antitumor activity and a manageable safety profile across multiple dose cohorts in both tyrosine kinase inhibitor (TKI) naïve and TKI-pretreated patients, including patients with intracranial disease.

"With additional follow up and new patients enrolled in the TRIDENT-1 study, repotrectinib continues to demonstrate promising efficacy and a safety profile consistent with a potential best-in-class ROS1 therapy for patients with advanced non-small cell lung cancer," said Dr. Byoung Chul Cho, Yonsei Cancer Center at Severance Hospital, Yonsei University College of Medicine, Seoul, Republic of Korea. "The ongoing Phase 1 data remain encouraging in both TKI-naïve patients with intracranial disease and patients pretreated with crizotinib — including those with difficult to treat solvent front mutations — where there are currently very few therapeutic options."

Dr. Cho is an investigator for the TRIDENT-1 study and will present the data today at the annual ASCO (Free ASCO Whitepaper) conference.

Repotrectinib is an investigational next-generation TKI designed to effectively target ROS1 and TRK A/B/C and systemically overcome resistant mutations that invariably result following treatment with other TKIs. ROS1 rearrangement is an oncogenic driver of tumors in up to 2.6 percent of U.S. advanced NSCLC patients.

As of the March 4, 2019 data cut-off, the interim Phase 1 data showed the following results.

Preliminary Safety Analysis (n=83):

A total of 83 (ROS1+, NTRK+ and ALK+) patients were treated with repotrectinib at dose levels from 40 mg daily (QD) to 200 mg twice daily (BID).
Repotrectinib was generally well tolerated, with the most frequent treatment emergent adverse events (TEAEs) being Grade 1 or 2. The TEAEs found in >25% of pts were dizziness (57%), dysgeusia (51%), dyspnea (30%), fatigue (30%), constipation (29%), paresthesia (29%), and anemia (28%). There were few Grade 3 treatment-related AEs (anemia (n=3); dizziness (n=2); and dyspnea, hypophosphatemia, hypoxia, pleural effusion, and weight increase (all n=1)), and no Grade 4 treatment-related AEs or cases of dizziness leading to treatment discontinuation.
No additional dose-limiting toxicities (DLTs) have occurred since the last data cut-off of Oct. 31, 2018. Prior DLTs included Grade 3 dyspnea/hypoxia (n=1), and Grade 2 (n=1) and Grade 3 (n=2) dizziness. Four total Grade 5 TEAEs (one of four determined to be possibly treatment related) have occurred, with one Grade 5 case of respiratory failure reported as related to disease progression since the last interim data update.
Preliminary Efficacy Analysis (n=33)

Within the ROS1+ NSCLC blinded independent central review (BICR) efficacy evaluable population (n=33), the median number of prior TKI therapies was one (range 0 to 3), the majority of which were crizotinib.
Forty-five percent of patients remained on treatment (15/33), with 12 of the 15 (80%) patients remaining on treatment for more than 12 months.
TKI-Naïve Efficacy Analysis

In TKI-naïve patients, overall response rate (ORR) by BICR was 82 percent (9/11). At a dose of 160 mg QD or above, the likely recommended Phase 2 dose, ORR by BICR remains at 83 percent, consistent with the prior update. Efficacy results are summarized in the following table:

Prior Interim Analysis by BICR
Oct. 31, 2018 Data cut-off Current Interim Analysis by BICR
March 4, 2019 Data cut-off
All ROS1 NSCLC (TKI
Naïve & Pretreated) N=28 N=33
TKI Naïve N=10 N=11
Median Follow up –
(Range) 16.4 months
(5.3-16.6+) 16.4 months
(3.5+-19.4+)
ORR (%)
(95% CI) 9/10 (90)
(56-100) 9/11 (82)
(48-98)
ORR at 160 mg QD or
above 5/6 (83) 5/6 (83)
Median Duration of
Response – Months
(Range) Not Reached
(5.5+-14.9+)
5 of 9 remain in cPR
(5.5+ to 14.9+ months) Not Reached
(5.6-17.7+)
5 of 9 remain in cPR (10.9+ to 17.7+ months)
Intracranial ORR n (%)
(95% CI) 3/3 (100)
(29-100) 3/3 (100)
(29-100)
All remain in cPR
Clinical Benefit Rate (%)
(95% CI) 10/10 (100)
(69-100) 11/11 (100)
(72-100)
TKI-Pretreated Efficacy Analysis

In TKI-pretreated patients, ORR by BICR improved from 28 percent in the prior data cut-off to 32 percent, and from 44 percent to 55 percent in patients pretreated with 1 TKI and dosed at 160 mg QD or above. Efficacy results are summarized in the following table:

Prior Interim Analysis by BICR
Oct. 31, 2018 Data cut-off Current Interim Analysis by BICR
March 4, 2019 Data cut-off
All ROS1 NSCLC (TKI Naïve
& Pretreated) N=28 N=33
TKI Pretreated N=18 N=22
Median Follow up –
(Range) 12.9 months
(0.6-14.5) 14.6 months
(1.4-16.6+)
ORR (%)
(95% CI) 5/18 (28)
(10-53) 7/22 (32)
(14-55)

O
R
R

1 prior TKI 5/15 (33) 7/18 (39)
1 prior TKI
160 mg QD or above 4/9 (44) 6/11 (55)
1 prior TKI
crizotinib only
160 mg QD or above 3/6 (50) 4/7 (57)
2 or more prior TKIs 0/3 (0) 0/4 (0)
Duration of Response –
Months
# still in response (+) 1 of 5 responders still in cPR at 1.9+ months 3 of 7 responders still in cPR at 1.0+ to 7.6+ months
ORR in G2032R Solvent
Front Mutations
(%) 1/4 (25) 2/5 (40)
Intracranial ORR n (%)
1 prior TKI 2/4 (50)
2/3 (67) 3/5 (60)
3/4 (75)
Clinical Benefit Rate (%)
(95% CI) 14/18 (78)
(52-94) 16/22 (73)
(50-89)
Five patients pretreated with crizotinib had a ROS1 G2032R solvent front mutation detected at baseline by plasma cfDNA or next-generation sequencing tests. All five patients had tumor regressions, including two confirmed partial responses (cPR) at the 160 mg QD dose level.

"Since first reporting preliminary TRIDENT-1 interim data for repotrectinib at the 2018 annual ASCO (Free ASCO Whitepaper) meeting, we are very pleased to see the results continue to strengthen with each subsequent data cut-off while maintaining a consistent safety profile in both TKI-naïve and TKI-pretreated ROS1+ NSCLC patients," said Athena Countouriotis, M.D., president and chief executive officer. "The current treatment options for patients with ROS1+ advanced non-small cell lung cancer are limited, particularly when resistance develops to other TKIs. We look forward to finalizing our recommended Phase 2 dose and beginning the registrational portion of TRIDENT-1 in the second half of this year."

Financial Update
The company separately filed its Form 10-Q today for the first quarter of 2019. Operating expenses during the first quarter were $14.1 million compared to $4.9 million in the first quarter of 2018. The $9.2 million increase was primarily due to increased personnel expenses and activities for the development of repotrectinib, TPX-0022 and TPX-0046. Net cash used in operating activities was $10.2 million, an increase from $5.4 million in the prior-year period.

Turning Point expects expenses will increase through the year as it plans to initiate up to four clinical trials in 2019 including: the Phase 2 TRIDENT-1 registrational study for repotrectinib; a Phase 1/2 study of repotrectinib in pediatric patients with advanced solid tumors with NTRK, ALK, or ROS1 alterations; a Phase 1 dose-finding study for TPX-0022, a novel MET, CSF1R and SRC inhibitor in patients with advanced solid tumors harboring genetic alterations in MET; and, pending completion of IND-enabling studies, a Phase 1 study of TPX-0046, a novel RET and SRC inhibitor in patients with advanced solid tumors with oncogenic RET genetic alterations.

Cash and cash equivalents at March 31 were $90 million. In addition, the company raised net proceeds of $175.2 million from its April 2019 initial public offering. The company projects its cash position will be sufficient to fund current operations into the second half of 2021.

On May 31, 2019 Jiangsu Hengrui Medicine reported China regulators approved its PD-1 drug, camrelizumab, as a third-line treatment for recurrent or refractory classical Hodgkin’s lymphoma (Press release, ChinaBio, MAY 31, 2019, View Source [SID1234556331]). Camrelizumab is the third domestic PD-1/PD-L1 drug approved in China; previously, Junshi’s Tuoyi and Innovent’s Tyvyx were granted marketing approvals. Two US-based companies have also entered China’s PD-1/PD-L1 market — Merck with Keytruda (also for melanoma) and Bristol Myers Squibb with Opdivo. Hengrui said it is testing camrelizumab in about 20 China clinical trials.

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