On May 13, 2019 TCR2 Therapeutics Inc. (Nasdaq: TCRR), a clinical-stage immunotherapy company developing the next generation of novel T cell therapies for patients suffering from cancer, reported financial results for the first quarter ended March 31, 2019 and provided a corporate update (Press release, TCR2 Therapeutics, MAY 13, 2019, View Source [SID1234536248]).

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"In our first quarter as a public company, TCR2 has continued to execute our vision and objectives," said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics. "During the quarter, we initiated our Phase 1/2 trial for TC-210, progressed TC-110 toward IND submission in the second half of 2019, and highlighted the differentiation of our novel TRuC-T cells in scientific presentations at Keystone Symposia on Cancer Immunotherapy, AACR (Free AACR Whitepaper) and CAR-T Congress USA. These preclinical studies underscore TRuC-T cells’ greater anti-tumor activity, longer persistence, and less cytokine release compared to CAR-T cells."

Recent Developments

In January 2019, the FDA cleared the IND for TC-210. TCR2 initiated its Phase 1/2 trial to treat patients with mesothelin-positive non-small cell lung cancer (NSCLC), ovarian cancer, malignant pleural/peritoneal mesothelioma, and cholangiocarcinoma. TCR2 anticipates providing an update on the trial before the end of the 2019.

In February 2019, TCR2 completed an initial public offering pursuant to which it issued and sold 5,750,000 shares of common stock, including full exercise of the underwriters’ over-allotment option, resulting in net proceeds of $80.2 million after deducting underwriting discounts and commissions.

In February 2019, the FDA granted orphan drug designation to TC-210 for the treatment of mesothelioma.

In February 2019, TCR2 held a pre-IND meeting with the FDA and remains on track to submit an IND for TC-110 in patients with CD19+ non-Hodgkin lymphomas and leukemias in the second half of 2019.

In February 2019, the United States Patent and Trademark Office issued U.S. Patent No.: 10,208,285, with claims covering TCR2’s TRuC-T cells that express anti-mesothelin TCR fusion proteins, including TC-210.

In March 2019, TCR2 presented two poster presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 featuring preclinical data for TC-110 and TC-210.

In March 2019, TCR2 established occupancy of its manufacturing suite at Cell and Gene Therapy (CGT) Catapult in the UK.

In May 2019, TCR2 published preclinical data in the peer-reviewed journal Nature Communications demonstrating the advantages of TRuC-T cells compared to CAR-T cells. In the paper entitled, "Synthetic TRuC receptors engaging the complete T cell receptor for potent anti-tumor response," the findings, consistent with the Company’s previously reported preclinical data, support the Company’s belief that its product candidates could improve the observed efficacy and safety of other adoptive T-cell therapies in development.

TC-210 Clinical Trial Design

The Phase 1/2 clinical trial (NCT03907852) is evaluating the safety and efficacy of TC-210, TCR2’s T-cell receptor fusion construct against mesothelin. The trial is enrolling patients with mesothelin expressing non-small cell lung cancer (NSCLC), ovarian cancer, cholangiocarcinoma, and malignant pleural/peritoneal mesothelioma.

The Phase 1 portion of the clinical trial utilizes a 3+3 design with four escalating dose levels for TC-210. At each dose level, TC-210 will be first tested without lymphodepletion and then following lymphodepleting chemotherapy. The primary objective for the study is patient safety with a key secondary objective to determine the recommended Phase 2 dose (RP2D). In addition to standard measures of safety and efficacy, translational work includes assessment of patients’ tissues for expansion, trafficking, and persistence of TC-210 T cells.

In the Phase 2 portion of the clinical trial, approximately 50 patients are planned to receive TC-210 at the RP2D in four distinct cohorts according to their cancer diagnosis: NSCLC, ovarian cancer, malignant pleural/peritoneal mesothelioma and cholangiocarcinoma. Each cohort includes ten patients, except the NSCLC cohort which includes 20 patients with eight patients to receive TC-210 as single agent and 12 to receive TC-210 in combination with a programmed cell death 1 (PD-1) blocking antibody.

Summary of Recent Presentations and Publications

Keystone Symposia on Molecular and Cellular Biology: Cancer Immunotherapy
Robert Hofmeister, Ph.D., Chief Scientific Officer, delivered an oral presentation, "Preclinical Evaluation of TC-210, a Mesothelin-Specific T Cell Receptor (TCR) Fusion Construct (TRuCTM) T Cells for the Treatment of Solid Tumors."

AACR Annual Meeting 2019
The Company presented two posters on the preclinical evaluation of TC-210 and TC-110 demonstrating superior in vivo anti-tumor activity compared to CAR-T cells with the same binders, including evidence that the anti-tumor activity of TRuC-T cells, both in vitro and in vivo, does not require added co-stimulatory signals.

CAR-T Congress USA 2019
Robert Hofmeister, Ph.D., Chief Scientific Officer, delivered an oral presentation on the features of the TRuC-T cell platform, "Utilizing the Entire T Cell Receptor Independent of HLA for a Broad and Controlled Anti-Tumor Response."

Nature Communications
The Company published preclinical data demonstrating the advantages of TRuC-T cells compared to CAR-T cells. In the paper entitled, "Synthetic TRuC receptors engaging the complete T cell receptor for potent anti-tumor response," the findings, consistent with the TCR2’s previously reported preclinical data, support the Company’s belief that its product candidates could improve the observed efficacy and safety of other adoptive T-cell therapies in development.

Financial Highlights
Cash Position: TCR2 ended 1Q 2019 with $191.7 million in cash, cash equivalents, and investments compared to $123.2 million as of December 31, 2018. Net cash from the Company’s initial public offering in 1Q 2019 was $80.2 million. Net cash used in operations was $10.8 million for 1Q 2019 compared to $3.0 million in 1Q 2018. TCR2 projects net cash use of $45-55 million in 2019.

R&D Expenses: Research and development expenses were $7.9 million for the first quarter of 2019 compared to $2.9 million for the first quarter of 2018. The increase in R&D expenses is primarily related to increase in headcount and activities related to the start of the Phase 1/2 clinical trial of the Company’s lead solid tumor product candidate, TC-210.

G&A Expenses: General and administrative expenses were $2.9 million for the first quarter of 2019 compared to $1.2 million for the first quarter of 2018. The increase in general and administrative expenses was primarily due to an increase in personnel costs and cost associated with operations as a public company.

Net loss: Net loss was $9.9 million for the first quarter of 2019 compared to $4.0 million for the first quarter of 2018, driven predominantly by increased R&D expense in the quarter.

Upcoming Events
TCR2 Therapeutics management are scheduled to present at the following upcoming conferences.

Jefferies Global Healthcare Conference: Alfonso Quintás Cardama, M.D., Chief Medical Officer and Ian Somaiya, Chief Financial Officer, will present on Wednesday, June 5th, 2019 at 10:30am ET in New York, NY.

BMO 2019 Prescriptions for Success Healthcare Conference: Robert Hofmeister, Ph.D., Chief Scientific Officer, will present on Thursday, June 25, 2019 at 3:40pm ET in New York, NY.

Conference Call Details
TCR2 will host a conference call and live audio webcast to discuss results and provide a corporate update at 5:00 PM ET today. The live call may be accessed by dialing (866) 220-8062 for domestic calls or (470) 495-9169 for international calls and referencing conference ID 2063908. A live audio webcast for the conference call will be available on the Investors page of the Company’s website at investors.tcr2.com. Following the conclusion of the call, the webcast will be available for replay for 30 days.

On May 13, 2019 Poseida Therapeutics Inc., a clinical-stage biopharmaceutical company leveraging proprietary non-viral gene engineering technologies to create life-saving therapeutics, reported the United States Food and Drug Administration (FDA) has granted orphan drug designation to P-BCMA-101 for the treatment of relapsed and/or refractory multiple myeloma (Press release, Poseida Therapeutics, MAY 13, 2019, View Source [SID1234536247]). P-BCMA-101 is an autologous CAR-T therapy developed using Poseida’s piggyBac platform technology. P-BCMA-101 is comprised of a high percentage of long-lived, self-renewing stem cell memory T cells targeting cancer cells expressing B-cell maturation antigen (BCMA).

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"FDA orphan designation is an important regulatory milestone in the continued development and commercialization of P-BCMA-101," said Eric Ostertag, M.D., Ph.D., chief executive officer of Poseida. "P-BCMA-101 has demonstrated outstanding potency, with strikingly low rates of toxicity in our phase 1 clinical trial. In fact, the FDA has approved fully outpatient dosing in our Phase 2 trial starting in the second quarter of 2019."

Poseida’s non-viral piggyBac DNA Modification System results in CAR-T product candidates with a high percentage of stem cell memory T cells (Tscm), the only T cell that is self-renewing and long-lived, leading to CAR-T products with improved efficacy, lower toxicity and potentially greater durability than earlier generation CAR-T therapies.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to drugs and biologics which are intended for the treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits and seven years of market exclusivity in the United States following marketing approval by the FDA.

Poseida has received grant funding from the California Institute for Regenerative Medicine to support the clinical development of P-BCMA-101.

On May 13, 2019 Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company leveraging deep understanding of immunobiology to develop products to treat severe autoimmune and inflammatory disorders with high unmet medical need, reported financial results for the first quarter 2019, and recent business highlights Cash and cash equivalents (Press release, Equillium, MAY 13, 2019, View Source [SID1234536246]).

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"We achieved a significant milestone during the first quarter of 2019 with the initiation of the open label Phase 1b portion of our EQUATE trial evaluating itolizumab in acute GVHD, our initial indication," stated Daniel Bradbury, chairman and chief executive officer of Equillium. "In parallel, we continue to advance plans to initiate two additional trials this year – uncontrolled asthma and lupus nephritis – all of which we believe set the stage for multiple opportunities to demonstrate the value of itolizumab in treating severe immuno-inflammatory diseases. The body of evidence implicating the CD6-ALCAM pathway in immuno-inflammatory diseases continues to grow. Itolizumab’s unique mechanism of action, which selectively targets CD6 and downregulates cellular pathways that modulate both the activity and trafficking of effector T cells, represents an entirely new approach to treating these serious diseases. We look forward to data from these important proof-of-concept trials."

Business Highlights:

Initiated the open label Phase 1b portion of the EQUATE trial for the first-line treatment of aGVHD

Received FDA Orphan Drug and Fast Track designations for itolizumab for both the prevention and treatment of aGVHD

Announced plans to develop itolizumab for the treatment of lupus nephritis

Upcoming Milestones:

Initiation of the Phase 1b EQUIP proof-of-concept trial evaluating itolizumab for the treatment of uncontrolled moderate to severe asthma expected during the second quarter of 2019

Initiation of a Phase 1b proof-of-concept trial of itolizumab for the treatment of lupus nephritis expected during the second half of 2019

Data from the EQUATE aGVHD trial expected during the first quarter of 2020, approximately 12 months following initiation

First Quarter 2019 Financial Results

Research and development (R&D) expenses. Total R&D expenses for the three months ended March 31, 2019 were $3.8 million, compared with $0.7 million for the same period in 2018. The increase in R&D expenses was primarily driven by additional costs related to increased headcount and the ramp up in regulatory and clinical activity including initiation of the Phase 1b portion of the clinical trial of itolizumab in aGVHD, and preclinical research activities to support Equillium’s clinical development program.

General and administrative (G&A) expenses. Total G&A expenses for the three months ended March 31, 2019 were $2.6 million, compared with $0.4 million for the same period in 2018. The increase in G&A expenses was primarily driven by additional costs related to increased headcount, legal and other professional fees and costs associated with being a public company.

Net loss. Net loss for the three months ended March 31, 2019 was $6.0 million, or $(0.34) per basic and diluted share, compared with a net loss of approximately $1.6 million, or $(0.15) per basic and diluted share, for the same period in 2018.

Cash and cash equivalents. As of March 31, 2019, Equillium reported total cash, cash equivalents and short-term investments of $61.6 million, compared to $65.9 million as of December 31, 2018.

On May 13, 2019 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported financial results for the quarter ended March 31, 2019 and provided an update on its corporate activities and product pipeline (Press release, Crinetics Pharmaceuticals, MAY 13, 2019, View Source [SID1234536245]).

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"Crinetics continues to make strong clinical progress in 2019, as we dosed the first acromegaly patients in our Phase 2 EVOLVE and EDGE trials for our lead product candidate, CRN00808," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "Looking forward to the remainder of the year, we will continue enrolling the ACROBAT trials and will initiate our Phase 1 trial for CRN01941 aimed at neuroendocrine tumors during the second quarter while continuing to advance additional molecules in our growing pipeline of endocrine product candidates."

First Quarter Highlights

Dosed first patients in Phase 2 clinical trials of CRN00808 for acromegaly. In March 2019, Crinetics dosed the first patients in the ACROBAT EVOLVE and ACROBAT EDGE trials for CRN00808 in patients with acromegaly. The EVOLVE trial is a double-blind, placebo-controlled, randomized withdrawal study designed to evaluate the safety, efficacy, and pharmacokinetics of CRN00808, an oral selective nonpeptide somatostatin receptor type 2 biased agonist, in patients with acromegaly that respond to octreotide LAR or lanreotide depot monotherapy. The EDGE trial is an open label exploratory study designed to evaluate the safety, efficacy, and pharmacokinetics of CRN00808 in patients with acromegaly that are treated with somatostatin analog based treatment regimens but do not respond completely to monotherapy.

Expanded management team. In March 2019, Crinetics appointed Gina Ford, RPh, MBA, as Vice President, Corporate Strategy and Commercial Planning.

Presented at ENDO2019. In March 2019, Crinetics made four poster presentations relating to the company’s pipeline at ENDO2019, the Annual Meeting of the Endocrine Society, in New Orleans.

First Quarter 2019 Financial Results

Research and development expenses were $7.3 million for the three months ended March 31, 2019, compared to $4.7 million for the same period in 2018. The increases were primarily attributable to development and manufacturing activities for CRN00808 as well as the advancement of the company’s preclinical programs and higher personnel costs.

General and administrative expenses were $3.2 million for the three months ended March 31, 2019, compared to $1.2 million for the same period in 2018. The increases were primarily due to costs to operate as a public company, as well as personnel costs to support the company’s growth.

Net loss for the three months ended March 31, 2019 was $9.0 million, compared to a net loss of $5.5 million for the three months ended March 31, 2018.

Cash, cash equivalents and investments totaled $157.2 million as of March 31, 2019, compared with $163.9 million as of December 31, 2018.

As of April 30, 2019, the company had 24,138,177 common shares outstanding.

On May 13, 2019 Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a Phase 3 clinical-stage biopharmaceutical company dedicated to developing and commercializing etripamil for the treatment of cardiovascular indications, reported the closing of its previously announced initial public offering of 5,500,000 of its common shares at a public offering price of $15.00 per share (Press release, Milestone Pharmaceuticals, MAY 13, 2019, View Source [SID1234536240]). The gross proceeds to Milestone, before deducting underwriting discounts, commissions and offering expenses, were approximately $82.5 million. All of the common shares were offered by Milestone.

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Milestone’s common shares are listed on the Nasdaq Global Select Market under the ticker symbol "MIST."

Jefferies LLC, Cowen and Company, LLC, and Piper Jaffray & Co. served as joint book-running managers for the offering. Oppenheimer & Co. Inc. served as lead manager for the offering.

The shares were offered by Milestone pursuant to registration statements that were declared effective by the U.S. Securities and Exchange Commission ("SEC") on May 8, 2019. A prospectus relating to and describing the terms of the offering has been filed with the SEC and is available on the SEC’s website at www.sec.gov.

The offering of these shares was made only by means of a prospectus. Copies of the final prospectus relating to this offering may be obtained from Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at (877) 821-7388, or by e-mail at [email protected], or from Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, by telephone at 631-592-5973 or by email at [email protected], or from Piper Jaffray & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at (800) 747-3924 or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of Milestone’s common shares in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state or jurisdiction.