On March 30, 2021 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 fourth quarter and year ended December 31, 2020 (Press release, Crinetics Pharmaceuticals, MAR 30, 2021, View Source [SID1234577352]).

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"We are entering 2021 poised to achieve several key milestones that we believe will consolidate our position as a leader in the design and development of novel small molecule drugs for endocrine diseases," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "Our positive Phase 2 acromegaly data demonstrate the potential of orally administered paltusotine to replace injectable somatostatin receptor ligand (SRL) depots as the standard-of-care. With input from a recently completed FDA meeting, we have designed the paltusotine Phase 3 program to support its approval for all acromegaly patients who require pharmacotherapy, including both untreated patients and those switching from injected standard of care."

Dr. Struthers continued, "Paltusotine’s success and progress serve as a blueprint for our new therapeutic candidates in endocrinology. We plan to follow a similar path with CRN04777 and CRN04894, both of which recently entered Phase 1 proof-of-concept trials in healthy volunteers. Much like paltusotine’s Phase 1 program, we’ve designed these trials to evaluate safety, drug-like pharmacokinetics, and corresponding pharmacodynamic effects on well-validated hormonal biomarkers for their respective indications. We look forward to reporting results from the Phase 1 trials later this year, and to the continued progression of our clinical and preclinical pipelines."

FULL YEAR 2020 AND RECENT HIGHLIGHTS
Advancing paltusotine to a Phase 3 program for acromegaly following the completion of an FDA meeting. In 1Q 2021, Crinetics met with the U.S. Food and Drug Administration (FDA) to discuss its Phase 2 acromegaly data and plans for a Phase 3 program. Based on these interactions and those with other regulators, the Company plans to initiate two Phase 3 studies in 2021. The first of these studies, entitled PATHFNDR-1, is a double-blind, placebo-controlled, nine-month clinical trial evaluating the safety and efficacy of paltusotine in acromegaly patients who are biochemically controlled (IGF-1 ≤ 1.0 × upper limit of normal [ULN]) and who are on stable doses of SRL monotherapy (octreotide LAR or lanreotide depot). The second study, PATHFNDR-2, is a double-blind, placebo-controlled, twelve-week trial in acromegaly patients with elevated IGF-1 levels who are medication naïve or who are not being treated with pharmacotherapy (untreated patients). The primary endpoint for both studies will be the proportion of patients achieving biochemical control comparted to placebo. If successful, Crinetics believes these trials could support registration of paltusotine in the United States and Europe for all acromegaly patients who require pharmacotherapy, including untreated patients and those switching from standard of care. The Company expects to initiate PATHFNDR-1 in 2Q 2021 and PATHFNDR-2 in 2H 2021.
Showcased broad clinical-stage pipeline at ENDO 2021. In March 2021, Crinetics showcased the breadth of its pipeline with presentations on its three clinical programs at the Endocrine Society’s annual ENDO 2021 congress. Posters on the company’s acromegaly program included a summary of the previously announced ACROBAT Edge Phase 2 results, as well as details of a new tablet formulation of paltusotine. Presentations related to the company’s earlier stage clinical programs included a poster with preclinical data supporting the development of CRN04777 as a treatment for congenital hyperinsulinism (congenital HI) and a live oral presentation with preclinical evidence supporting the further evaluation of CRN04894 in Cushing’s disease and congenital adrenal hyperplasia (CAH).
Advanced ACTH antagonist CRN04894 into a Phase 1 study designed to provide clinical proof of concept. In February 2021, Crinetics initiated a Phase 1 study of CRN04894, an investigational, oral, nonpeptide adrenocorticotropic hormone (ACTH) antagonist being developed for the treatment of diseases associated with excess ACTH such as Cushing’s disease and CAH. In addition to evaluating the safety and tolerability of CRN04894 in healthy volunteers, the placebo-controlled study aims to provide clinical proof-of-concept data by measuring the effect of CRN04894 on the suppression of ACTH-stimulated adrenal secretion of cortisol and related steroids. These endocrine biomarkers are used as key endpoints in patient studies and reflect the ability of CRN04894 to block ACTH-stimulated adrenal function. This Phase 1 trial is expected to provide important information for dose selection and be predictive of efficacy in Cushing’s disease and CAH trials. Crinetics expects to report preliminary safety, pharmacokinetic, and endocrine biomarker data from the single ascending dose portion of the trial in 1H 2021.
Advanced SST5 agonist CRN04777 into a Phase 1 study designed to provide clinical proof of concept. In February 2021, Crinetics initiated a Phase 1 study of CRN04777, an investigational, oral, nonpeptide somatostatin receptor type 5 (SST5) agonist. CRN04777 is being developed as a treatment for congenital HI, a rare genetic disease in which excess insulin secretion causes life-threatening hypoglycemia (low blood glucose). In addition to evaluating the safety and tolerability of CRN04777 in healthy volunteers, the placebo-controlled study aims to provide clinical proof-of-concept data by measuring the ability of CRN04777 to inhibit glucose- and sulfonylurea-induced insulin secretion and observe the corresponding effects on blood glucose levels. These endocrine biomarkers are indicative of the ability of CRN04777 to prevent hypoglycemia and are expected to provide information for dose selection and be predictive of efficacy in patients with hyperinsulinism. Crinetics expects to report preliminary safety and pharmacological effect data from the single ascending dose portion of the trial in mid-2021.
Reported positive topline results for the ACROBAT Phase 2 program of paltusotine in acromegaly patients. In October 2020, Crinetics reported positive top-line data from its Phase 2 program evaluating paltusotine for the treatment of acromegaly. Data showed that levels of insulin-like growth factor 1 (IGF-1), the accepted biomarker of disease control in acromegaly, were maintained in patients switching from standard-of-care injected SRL depots to once-daily oral paltusotine.
Received Rare Pediatric Disease Designation for CRN04777 for the treatment of congenital HI. In September 2020, the FDA granted CRN04777 Rare Pediatric Disease (RPD) Designation for the treatment of congenital HI. Based on this designation, Crinetics may be eligible to receive a priority review voucher (PRV) following approval of CRN04777 if the marketing application submitted for the candidate satisfies certain additional conditions. If issued, a PRV may be redeemed for priority review of a subsequent marketing application for a different product candidate, or the PRV could be sold or transferred to another sponsor.
Received Orphan Drug Designation for paltusotine for the treatment of acromegaly. In July 2020, the FDA granted paltusotine Orphan Drug Designation for the treatment of acromegaly, qualifying Crinetics for certain development incentives such as exemption from FDA prescription drug user fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. upon FDA approval.
Strengthened company leadership with appointments to clinical team and Board of Directors. Throughout 2020, Crinetics continued to reinforce its in-house expertise in endocrinology, rare disease drug development, and clinical trial management through the appointments of Drs. Alessandra Casagrande, Peter Trainer, and Hjalmar Lagast to the clinical team and the appointment of Dr. Camille L. Bedrosian to the Board of Directors.

FOURTH QUARTER AND FULL YEAR 2020 FINANCIAL RESULTS
Research and development expenses were $16.8 million and $57.0 million for the three months and full year ended December 31, 2020, respectively, compared to $12.1 million and $41.5 million for the same periods in 2019. The increases were primarily attributable to clinical development and manufacturing activities for paltusotine as well as the company’s preclinical programs.
General and administrative expenses were $5.0 million and $18.0 million for the three months and full year ended December 31, 2020, compared to $3.4 million and $13.5 million for the same periods in 2019. 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 December 31, 2020 was $21.6 million, compared to a net loss of $14.5 million for the same period in 2019. For the year ended December 31, 2020, the company’s net loss was $73.8 million compared to a net loss of $50.4 million for the year ended December 31, 2019.
Unrestricted cash, cash equivalents and investments totaled $170.9 million as of December 31, 2020, compared to $186.8 million as of September 30, 2020 and $118.4 million as of December 31, 2019.
As of February 28, 2021, the company had 33,028,876 common shares outstanding.

WEBCAST AND CONFERENCE CALL INFORMATION
Crinetics will hold a live webcast and conference call today, March 30, 2021 at 4:30 p.m. Eastern Time to discuss its 2021 clinical plans and 2020 financial results. To access the webcast, please visit this link to the event. To participate by phone, please dial 877-407-0789 (domestic) or 201-689-8562 (international) and refer to conference ID 13717687. Following the live event, the archived webcast will be available for 90 days.

On March 30, 2021 Amgen Inc. (NASDAQ:AMGN) and Rodeo Therapeutics Corporation (Rodeo) reported an agreement under which Amgen will acquire Rodeo, a privately held biopharmaceutical company based in Seattle that develops small-molecule therapies designed to promote regeneration and repair of multiple tissues (Press release, Amgen, MAR 30, 2021, View Source [SID1234577351]). Rodeo’s 15-PGDH program is a strong strategic fit with Amgen’s inflammation portfolio and efforts to develop first-in-class therapeutics for patients.

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Under terms of the agreement, Amgen will acquire all outstanding shares of Rodeo in exchange for a $55 million upfront payment as well as future contingent milestone payments potentially worth up to an additional $666 million in cash. The transaction has been approved by the shareholders and the Board of Directors of Rodeo.

Rodeo is focused on developing first-in-class, orally available modulators of prostaglandin biology that play an important role in tissue regeneration and repair. Rodeo’s lead 15-prostaglandin dehydrogenase (15-PGDH) modulators have generated compelling data in extensive preclinical studies and have clinical potential in multiple indications.

"The enzyme 15-PGDH plays a key role in many disease-relevant processes such as stem cell self-renewal and epithelial barrier repair. Given the encouraging preclinical data to date, we are excited about the opportunity to develop a novel therapy with potential in a range of important inflammatory disease indications," said Raymond Deshaies, Ph.D., senior vice president of Global Research at Amgen.

Thong Q. Le, president and chief executive officer of Rodeo commented, "We are thrilled that Amgen recognizes the potential value and differentiated profile of our 15-PGDH inhibitor program. With decades of experience in developing, manufacturing and commercializing innovative therapies for patients suffering from a broad range of immunologic diseases and conditions, Amgen is ideally positioned to rapidly advance our program into the clinic."

Cooley LLP acted as legal advisor to Rodeo and Gunderson Dettmer LLP acted as legal advisor to Amgen on this transaction.

On March 30, 2021 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported financial results for the three-month period and full year ended December 31, 2020 (Press release, Spectrum Pharmaceuticals, MAR 30, 2021, View Source [SID1234577350]).

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"The Fast Track designation for poziotinib is a significant achievement towards an expedited review," said Joe Turgeon, President and CEO of Spectrum Pharmaceuticals. "In addition, we are delighted that the FDA has scheduled the pre-approval inspection at the ROLONTIS manufacturing facility for May 2021. The company has made tremendous progress advancing our development programs and conducting our clinical trials, despite the challenges of the global pandemic. I am proud of our employees who demonstrated resiliency and creativity during these unprecedented times."

Pipeline Updates

Poziotinib, an irreversible tyrosine kinase inhibitor targeting EGFR and HER2 mutations

Poziotinib received Fast Track designation from the FDA for the treatment of non-small cell lung cancer (NSCLC) in previously treated patients with HER2 exon 20 insertion mutations. Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious and life-threatening conditions and fill unmet medical needs.
Spectrum is preparing a new drug application (NDA) for poziotinib in the treatment of patients with previously treated locally advanced or metastatic NSCLC with HER2 exon 20 insertion mutations after a successful pre-NDA meeting with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2020. Submission of the NDA based on the positive results of Cohort 2 from the ZENITH20 clinical trial is planned for later this year.
Preliminary safety and efficacy data for poziotinib from Cohort 5 of the ZENITH20 clinical trial demonstrated improved tolerability with BID dosing, reduced dose interruption compared to once daily (QD) dosing, and a reduction in treatment emergent Grade 3 or higher adverse events. The preliminary data also demonstrated improved anti-tumor activity with 8mg BID dosing. These results were presented at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Virtual Congress 2021 in early March. Spectrum will be presenting further data on BID dosing at the upcoming AACR (Free AACR Whitepaper) Annual Meeting 2021.
ROLONTIS (eflapegrastim), a novel long-acting G-CSF

The FDA’s pre-approval inspection of the ROLONTIS manufacturing facility has been scheduled for May 2021. In October 2020, the FDA deferred its action on the Biologics License Application (BLA) for ROLONTIS due to an inability to inspect the drug substance manufacturing facility, citing travel restrictions related to the COVID-19 pandemic.
IGN002, interferon/CD20 monoclonal antibody fusion protein

Anti-CD20-IFNα, an antibody-interferon fusion molecule directed against CD20 has two sites open for enrollment in a Phase 1 study for treating relapsed or refractory non-Hodgkin’s lymphoma patients, including diffuse large B-cell lymphoma.
Three-Month Period Ended December 31, 2020 (All numbers are from Continuing Operations)

GAAP Results

Spectrum recorded net loss of $49.9 million, or $0.36 per basic and diluted share, in the three-month period ended December 31, 2020, compared to net loss of $40.2 million, or $0.36 per basic and diluted share, in the comparable period in 2019. Total research and development expenses were $47.2 million in the quarter, as compared to $23.3 million in the same period in 2019. Selling, general and administrative expenses were $15.7 million in the quarter, compared to $15.1 million in the same period in 2019.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $28.9 million, or $0.20 per basic and diluted share, in the three-month period ended December 31, 2020, compared to non-GAAP net loss of $33.4 million, or $0.30 per basic and diluted share, in the comparable period in 2019. Non-GAAP research and development expenses were $17.1 million, as compared to $22.4 million in the same period of 2019. Non-GAAP selling, general and administrative expenses were $12.3 million, as compared to $11.6 million in the same period in 2019.

Twelve-Month Period Ended December 31, 2020 (All numbers are from Continuing Operations)

GAAP Results

Spectrum recorded net loss of $171.3 million, or $1.38 per basic and diluted share, in the twelve-month period ended December 31, 2020, compared to net loss of $135.4 million, or $1.22 per basic and diluted share, in the comparable period in 2019. Total research and development expenses were $109.4 million for the year, as compared to $79.3 million in the same period in 2019. Selling, general and administrative expenses were $60.4 million for the year, compared to $61.4 million in the same period in 2019.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $120.9 million, or $0.97 per basic and diluted share, in the twelve-month period ended December 31, 2020, compared to non-GAAP net loss of $111.9 million, or $1.01 per basic and diluted share, in the comparable period in 2019. Non-GAAP research and development expenses were $75.6 million, as compared to $72.0 million in the same period of 2019. Non-GAAP selling, general and administrative expenses were $47.2 million, as compared to $45.5 million in the same period in 2019.

Cash Position and Guidance

Spectrum reported cash, cash equivalents, and marketable securities of approximately $180.0 million as of December 31, 2020, compared to $224 million at December 31, 2019.

This conference call will also be webcast. Listeners may access the webcast, which will be available on the investor relations page of Spectrum Pharmaceuticals’ website: View Source on March 30, 2021 at 4:30 p.m. Eastern/1:30 p.m. Pacific.

On March 30, 2021 HUYABIO International (HUYABIO), the leader in accelerating global development of China’s pharmaceutical innovations, reported that it had entered into a clinical collaboration agreement with Bristol-Myers Squibb Company (NYSE: BMY) to evaluate the combination of HUYABIO’s HBI-8000, an epigenetic immunomodifier, and Opdivo (nivolumab), a PD-1 blocking antibody (Press release, HUYA Bioscience, MAR 30, 2021, View Source [SID1234577349]). The Phase 3 trial is designed to evaluate the safety and efficacy of the combination in subjects with unresectable or metastatic melanoma not previously treated with anti-PD-1 therapy.

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"This Phase 3 trial follows our open label Phase 2 study which showed impressive clinical results for the combination of Opdivo and HBI-8000 to treat melanoma," said Dr. Mireille Gillings, CEO & Executive Chair of HUYABIO. "HBI-8000 has been shown to enhance the efficacy of Opdivo through one of its mechanisms of action that controls acetylation and nuclear transportation of PD-L1."

HUYABIO will be the sponsor of the trial. Bristol Myers Squibb will provide Opdivo clinical drug supply for the study. Opdivo is a registered trademark of Bristol Myers Squibb.

About HBI-8000

HBI-8000 is an epigenetic immunomodulator approved for the treatment of lymphoma and metastatic breast cancer in China. This oral agent targets class I histone deacetylases causing cell cycle arrest and tumor cell death as the mechanism underlying its single agent activity against lymphoma. The drug also has immunomodulatory impact by increasing the efficacy of checkpoint inhibitors in preclinical animal models. The Company recently reported results from its ongoing Phase 2 study for the nivolumab combination demonstrating an overall objective response rate above 70% with a disease control rate over 90% in a cohort of checkpoint naïve patients with melanoma.

On March 30, 2021 CorMedix Inc. (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of infectious and inflammatory disease, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided an update on recent business developments (Press release, CorMedix, MAR 30, 2021, View Source [SID1234577348]).

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Recent Corporate Highlights:

CorMedix announced in early March 2021 that the US Food and Drug Administration (FDA) cannot approve the New Drug Application (NDA) for DefenCath in its present form. FDA noted concerns at the third-party manufacturing facility after a review of records requested by FDA and provided by the manufacturing facility, and has requested a manual extraction study to demonstrate that the labeled volume can be consistently withdrawn from vials.
CorMedix continues to work closely with our third-party manufacturing facility and is planning for a meeting with the FDA in mid-April to obtain agreement on the adequacy of our proposed plans for resolution of the deficiencies.
CorMedix transitioned to Nasdaq in early February 2021, which has become the primary trading platform for biopharmaceutical peers and other growth companies.
CorMedix has strengthened its balance sheet via equity financing activity over the past year and early 2021.
CorMedix has been approved by the New Jersey Economic Development Authority (NJEDA) to transfer substantially all of the $1.3 million of its available tax benefits to an unrelated, profitable New Jersey corporation pursuant to the New Jersey Technology Business Tax Certificate Transfer (NOL) program for State Fiscal Year 2020, for approximately $1.3 million in net proceeds. Closing is anticipated in 2Q of 2021.
Khoso Baluch, CorMedix CEO commented, "While we were disappointed that the DefenCath NDA was not approved at its PDUFA date, we remain confident in our efforts to bring DefenCath to hemodialysis patients as an important novel antimicrobial catheter lock solution to reduce catheter related blood stream infections in patients receiving hemodialysis via central venous catheters. We believe we have the right team and resources to accomplish this as we advance DefenCath closer to regulatory approval."

4th Quarter 2020 Financial Highlights

For the fourth quarter of 2020, CorMedix recorded a net loss of $6.1 million, or $0.19 per share, compared with a net loss of $5.3 million, or $0.21 per share, in the fourth quarter of 2019, an increase in net loss of $0.8 million. The increase in net loss in the fourth quarter of 2020 compared with 2019 was primarily driven by increases in employee costs and market research costs. Operating expenses during the fourth quarter of 2020 were $6.1 million, compared with $5.4 million in the fourth quarter of 2019, an increase of approximately $0.7 million.

Full Year 2020 Financial Highlights

For the year ended December 31, 2020, CorMedix recorded a net loss of $22.0 million, or $0.77 per share, compared with a net loss during the year ended December 31, 2019 of $16.4 million before recognition of deemed dividends, or $0.68 per share, an increase in net loss of $5.6 million. The increase in net loss was driven primarily by increases in operating expenses.

Operating expenses during the year ended December 31, 2020 amounted to $27.3 million compared with $20.9 million during the comparable period in 2019, an increase of $6.4 million, or 30%, due to a 21% increase in R&D expense and 41% increase in SG&A expense.

Total cash on hand and short-term investments as of December 31, 2020 amounted to $46.3 million, excluding restricted cash of $0.2 million. The Company believes that, based on the Company’s cash resources at year end plus the $41.5 million in net proceeds from ATM issuances since the beginning of 2021, it has sufficient resources to fund operations at least into the second half of 2022.

Conference Call Information

The management team of CorMedix will host a conference call and webcast today, March 30, 2021, at 4:30 PM Eastern Time, to discuss recent corporate developments and financial results. Call details and dial-in information is as follows: