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.

On May 13, 2019 Quest Diagnostics Incorporated (NYSE: DGX), the world’s leading provider of diagnostic information services, reported that it is scheduled to speak at the UBS Global Healthcare Conference in New York (Press release, Quest Diagnostics, MAY 13, 2019, View Source [SID1234536239]). Steve Rusckowski, Chairman, President and CEO will discuss the company’s vision, goals and two-point strategy to accelerate growth and drive operational excellence. The presentation is scheduled for Tuesday, May 21, 2019 at 2:30 p.m. Eastern Time.

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The presentation will be webcast live during the conference and will be available on the company’s investor relations page which can be accessed at ir.QuestDiagnostics.com. In addition, the archived webcast will be available within 24 hours after the conclusion of the live event and will remain available until June 20, 2019.