Cell Medica Awarded USD 8.7 Million CPRIT Grant to Accelerate CMD-502 Off-the-shelf CAR-NKT Cell Therapy into Clinical Development

On February 26, 2019 Cell Medica reported that it has been awarded an $8.7 million research grant from the Cancer Prevention and Research Institute of Texas (CPRIT) (Press release, Cell Medica, FEB 26, 2019, View Source [SID1234533676]). The grant will support preclinical and clinical development of the company’s off-the-shelf chimeric antigen receptor-natural killer T cell (CAR-NKT) therapies to treat hematological and solid tumors.

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The CPRIT grant will support development programs being conducted in collaboration with Baylor College of Medicine (BCM) that are designed to address the limitations of the current first-generation autologous CAR-T cell therapies. The aim is to deliver an off-the-shelf approach, with simplified manufacturing, that can serve larger patient numbers, and which allows treatment closer to the time of patient presentation. The program currently includes four therapies in early-stage development.

CMD-502 is Cell Medica’s most advanced off-the-shelf therapy and a first-in-human study is expected to start in the second half of 2019. Called the ANCHOR study, it will be a dose escalation evaluation of CMD-502 in adults with relapsed or refractory (R/R) diffuse large B cell lymphoma (DLBCL), acute lymphoblastic leukemia (ALL), and chronic lymphocytic leukemia (CLL).

CPRIT awarded a $15.3 million grant to Cell Medica in 2012, to support the establishment of operations in Houston, Texas and fund earlier development programs. Through the current grant, CPRIT-funded research will be conducted at both BCM and Cell Medica’s Houston facility.

Chris Nowers, CEO of Cell Medica, commented: "CPRIT was instrumental in enabling us to establish our US operations in Texas, so we are delighted to extend that collaboration through a further grant. This funding will accelerate development of our off-the-shelf CAR-NKT pipeline and, given CPRIT’s deep and broad review, also brings a strong independent validation of our platform and approach."

Dr. Carlos Ramos, Associate Professor, Center for Cell and Gene Therapy, at BCM and principal investigator in Phase 1 ANCHOR study, added: "It has been a great pleasure working with the multi-disciplinary team at Cell Medica in the development of its versatile CAR-NKT platform. Off-the-shelf CAR-NKT cell therapy has the potential to become a better and simpler approach to CAR therapy for patients with hematological and solid tumors.

"Although existing autologous CAR-T cell therapies have demonstrated impressive response rates, the patient-specific manufacturing process is technically challenging, costly, and time-consuming, and comes with complex logistics and substantial treatment delays. The unique properties of NKT cells bring the potential to solve these challenges."

Kleo Pharmaceuticals to Present at the 2019 BIO Asia International Conference

On February 26, 2019 Kleo Pharmaceuticals Inc., a unique immuno-oncology company developing next-generation bispecific compounds designed to emulate or enhance the activity of biologics, reported that its Chief Executive Officer Douglas J. Manion, M.D., FRCP(C), will present at the 16th Annual BIO Asia International Conference, to be held March 5-6, 2019, at the Grand Hyatt Tokyo in Tokyo, Japan (Press release, Kleo Pharmaceuticals, FEB 26, 2019, View Source [SID1234533675]).

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Dr. Manion will present an overview of Kleo’s development strategy for its pipeline of small molecule and synthetic peptide compounds that function similar to highly complex biologics, while offering multiple potential advantages. The overview will also outline Kleo’s key value drivers including the three proprietary technology platforms that drive in-house drug development and partnership opportunities, the multiple in-house and collaborative channels through which it is building its immuno-oncology pipeline, and its discovery collaboration with PeptiDream.

Kleo is well-funded to achieve multiple preclinical and early clinical milestones following the November 2018 closing of an oversubscribed $21 million Series B financing to advance its first clinical candidates through IND-enabling studies and into the clinic in 2020.

Details of Kleo’s presentation are as follows:

Event: 16th Annual BIO Asia International Conference

Date: Tuesday, March 6th, 2019

Time: 8:45 AM JST

Location: Grand Hyatt Tokyo, Drawing Room, 2nd floor

TG Therapeutics Announces Initiation of Phase I First-in-Human Clinical Trial of its Anti-CD47/CD19 Bispecific Antibody, TG-1801, in Patients with Relapsed or Refractory B-cell Lymphoma

On February 26, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX), a biopharmaceutical company dedicated to developing medicines for patients with B-cell mediated diseases, reported the commencement of a Phase I first-in-human trial of TG-1801, the Company’s novel first-in-class anti-CD47/CD19 bispecific antibody, in patients with relapsed or refractory B-cell lymphoma (Press release, TG Therapeutics, FEB 26, 2019, View Source [SID1234533673]).

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TG-1801 is the first therapy to target both CD19, a B-cell specific marker widely expressed across B-cell malignancies, and CD47, the "don’t eat me" signal used by both healthy and tumor cells to evade macrophage mediated phagocytosis. CD47 is expressed ubiquitously on normal cells, including red blood cells and platelets. By co-targeting both CD47 and CD19, TG-1801 has the potential to overcome the limitations of existing CD47 targeted therapies by avoiding the side effects caused by indiscriminate blockade of CD47 on healthy cells.

This Phase I open label, multi-center trial is designed to assess the safety, pharmacokinetics, efficacy and recommended Phase 2 dose of TG-1801. The primary objective of the study is to determine the Maximum Tolerated Dose (MTD) and characterize the safety profile of TG-1801, with secondary endpoints including characterization of pharmacokinetics and preliminary anti-cancer activity.

Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer stated, "We are extremely pleased to announce the commencement of clinical development for TG-1801, our proprietary first-in-class anti-CD47/CD19 bispecific antibody which we licensed from Novimmune last year." Mr. Weiss continued, "CD47 targeted therapy has yielded promising early clinical results, and we look forward to exploring the potential of this dual targeted immunotherapy with a long-term goal of combining TG-1801 with our other in-house immunotherapies and targeted agents to create novel treatment options for patients with B-cell cancers."

Vericel Reports Fourth Quarter and Full-Year 2018 Financial Results and Provides Full-Year 2019 Financial Guidance

On February 26, 2019 Vericel Corporation (NASDAQ:VCEL), a leader in advanced cell therapies for the sports medicine and severe burn care markets, reported financial results and business highlights for the fourth quarter and year ended December 31, 2018 and provided full-year 2019 financial guidance (Press release, Vericel, FEB 26, 2019, View Source [SID1234533670]).
Fourth Quarter 2018 Financial Highlights

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Total net product revenues increased 41% to $31.3 million compared to $22.2 million in the fourth quarter of 2017;

Gross margins of 72% compared to gross margins of 64% in the fourth quarter of 2017;

Net income of $5.2 million, or $0.11 per share, compared to $0.3 million, or $0.01 per share, in the fourth quarter of 2017;

Non-GAAP adjusted EBITDA of $7.7 million compared to $2.2 million in the fourth quarter of 2017; and

As of December 31, 2018, after retiring all outstanding debt, the company had $82.9 million in cash and short-term investments compared to $26.9 million in cash at December 31, 2017.

Full-Year 2018 Financial Highlights

Total net product revenues increased 45% to $90.9 million compared to $62.8 million in 2017;

Gross margins of 65% compared to gross margins of 53% in 2017;

Net loss of $8.1 million, or $0.20 per share, compared to a net loss of $17.3 million, or $0.52 per share, in 2017; and

Non-GAAP adjusted EBITDA of $4.7 million compared to a loss of $9.4 million in 2017.

Recent Business Highlights
During and since the fourth quarter of 2018, the company:

Reported record fourth quarter revenues, marking the seventh consecutive quarter with record revenues for the reported quarter;

Reported full-year positive adjusted EBITDA;

Expanded the MACI sales force from 40 to 48 territories; and

Reported publication of outcomes data from 954 burn patients treated with Epicel in the Journal of Burn Care and Research.

"We delivered record fourth quarter and full-year revenues and gross margins, and reported full-year positive adjusted EBITDA for the first time in the company’s history," said Nick Colangelo, president and CEO of Vericel. "Based on MACI’s performance in 2018 and the feedback that we are receiving from surgeons and patients, it is clear that MACI is filling what has been a large unmet need in the cartilage repair market, and we expect continued strong revenue and profit growth in 2019."

2019 Financial Guidance
The company expects total net product revenues for 2019 to be in the range of $108 million to $112 million. The company also expects margins to continue to increase, with approximately 80% of marginal revenue over 2018 to contribute to gross profit and approximately 50% of marginal revenue over 2018 to contribute to adjusted EBITDA. Quarterly seasonality, which impacts both revenues and margins, is expected to follow the same pattern as 2018.

Fourth Quarter 2018 Results
Total net product revenues for the quarter ended December 31, 2018 increased 41% to $31.3 million compared to $22.2 million in the fourth quarter of 2017. Total net product revenues for the quarter included $25.1 million of MACI (autologous cultured chondrocytes on porcine collage membrane) net revenue and $6.2 million of Epicel (cultured epidermal autografts) net revenue, compared to $16.1 million of MACI net revenue and $6.1 million of Epicel net revenue, respectively, in the fourth quarter of 2017. Total revenues for the fourth quarter of 2017 also included $1.2 million in license revenue related to the company’s license agreement with Innovative Cellular Therapeutics (ICT).
Gross profit for the quarter ended December 31, 2018 was $22.7 million, or 72% of net revenues, compared to $15.0 million, or 64% of net revenues, for the fourth quarter of 2017.
Total operating expenses for the quarter ended December 31, 2018 were $16.7 million compared to $13.8 million for the same period in 2017. The increase in operating expenses was primarily due to $1.4 million in service fees paid to MACI pharmacy distributors, an incremental $1.2 million in employee-related expenses associated with the expanded MACI sales force, and an incremental $0.5 million in stock-based compensation expense.
Vericel’s net income for the quarter ended December 31, 2018 was $5.2 million, or $0.11 per share, compared to a $0.3 million, or $0.01 per share, for the fourth quarter of 2017.
Non-GAAP adjusted EBITDA was $7.7 million for the quarter ended December 31, 2018 compared to $2.2 million in the fourth quarter of 2017. See table reconciling non-GAAP measures for more details.

As of December 31, 2018, the company had $82.9 million in cash and short-term investments compared to $26.9 million in cash at December 31, 2017.
Full-Year 2018 Results
Total net product revenues for the year ended December 31, 2018 increased 45% to $90.9 million compared to $62.8 million in 2017. Total net product revenues for the year included $67.7 million of MACI net revenue and $23.1 million of Epicel net revenue, compared to $43.9 million of MACI and Carticel (autologous cultured chondrocytes) net revenue and $18.9 million of Epicel net revenue, respectively, in 2017. Total revenues in 2017 also included $1.2 million in license revenue related to the company’s license agreement with ICT.
Gross profit for the year ended December 31, 2018 was $58.7 million, or 65% of net revenues, compared to $33.6 million, or 53% of net revenues, for 2017.
Total operating expenses for the year ended December 31, 2018 were $62.6 million compared to $48.6 million in 2017. The increase in operating expenses was primarily due to an incremental $4.5 million in employee-related expenses associated with the expanded MACI sales force, an incremental $2.7 million in stock-based compensation expense and an increase of $2.6 million in service fees paid to MACI pharmacy distributors.
Vericel’s net loss for the year ended December 31, 2018 was $8.1 million, or $0.20 per share, compared to a loss of $17.3 million, or $0.52 per share, in 2017.
Non-GAAP adjusted EBITDA was $4.7 million for the year ended December 31, 2018 compared to a loss of $9.4 million in 2017. See table reconciling non-GAAP measures for more details.
Conference Call Information
Today’s conference call will be available live at 8:30am Eastern time in the Investor Relations section of the Vericel website at View Source." target="_blank" title="View Source." rel="nofollow">View Source Please access the site at least 15 minutes prior to the scheduled start time in order to download the required audio software if necessary. To participate in the live call by telephone, please call (877) 312-5881 and reference Vericel Corporation’s fourth-quarter 2018 investor conference call. If calling from outside the U.S., please use the international phone number (253) 237-1173.
If you are unable to participate in the live call, the webcast will be available at View Source until February 26, 2020. A replay of the call will also be available until 11:15am (EDT) on March 3, 2019 by calling (855) 859-2056, or from outside the U.S. (404) 537-3406. The conference ID is 3485006.

Hummingbird Bioscience Awarded $13.1 Million Grant by the Cancer Prevention and Research Institute of Texas (CPRIT)

On February 25, 2019 Hummingbird Bioscience reported it has been awarded a product development grant totalling $13.1 million from the Cancer Prevention and Research Institute of Texas (CPRIT) (Press release, Hummingbird Bioscience, FEB 25, 2019, View Source [SID1234554020]).

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The grant will support the Phase IA/B clinical trials of the company’s first-in-class anti-VISTA therapeutic antibody for the treatment of VISTA mediated suppression of anti-tumor immunity in solid tumors and lymphomas that are unresponsive to existing therapies. Hummingbird Bioscience is a systems-biology enabled biotech company focused on the discovery and development of novel cancer therapeutics.

The CPRIT review for this grant included an in-depth evaluation of HMBD-002, an antibody that uniquely neutralizes VISTA function, by a panel of scientific, medical, and industry experts. The evaluation included rigorous review of the pre-clinical data, as well as regulatory, research and development, and intellectual property due diligence.

The grant will support the Phase IA/B clinical study and preparatory steps including GMP manufacturing and Investigational New Drug (IND) submission for the HMBD-002 program. The grant will also support the relocation of the company’s U.S. headquarters to Texas. Clinical trials for HMBD-002 are anticipated to begin in 2020. Hummingbird Bioscience was the only successful application of its type for this grant cycle.

The award follows the recent announcement of Nobel Laureate James P. Allison, Ph.D. and Padmanee Sharma, M.D., Ph.D. joining Hummingbird’s Scientific Advisory Board.

"We are delighted to be awarded this highly-selective grant from CPRIT and to start building our operations in Texas," said Piers Ingram, Ph.D., CEO of Hummingbird Bioscience. "Our company applies systems biology approaches to generate uniquely differentiated therapeutic antibodies. We are thrilled CPRIT’s panel of experts agrees that developing this anti-VISTA immuno-oncology program has the potential to significantly benefit patients."