On March 9, 2020 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, 2019 and provided a corporate update (Press release, Crinetics Pharmaceuticals, MAR 9, 2020, View Source [SID1234555317]).

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"Crinetics made significant progress in 2019 advancing our development programs, as highlighted by the initiation of the ACROBAT Phase 2 clinical trials for acromegaly with our lead product candidate paltusotine," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "Paltusotine is a first-in-class nonpeptide small molecule, which demonstrates our ability to develop novel drugs for diseases and patients that have not seen truly innovative therapies in a long time. We anticipate that 2020 will be a year of additional important milestones as our pipeline continues to advance. Importantly, we plan to provide guidance on timing of our acromegaly and neuroendocrine tumor programs early in the second quarter. In addition, we are advancing our ACTH antagonist development candidate for Cushing’s disease and congenital adrenal hyperplasia as well as our sst5 agonist development candidate for congenital hyperinsulinemia towards the clinic as IND enabling activities for both programs are underway."

Full Year 2019 Highlights

Dosed first patients in Phase 2 clinical trials of paltusotine (formerly CRN00808) for acromegaly. In March 2019, Crinetics dosed the first patients in the ACROBAT EDGE and EVOLVE trials for paltusotine in patients with acromegaly. EDGE is an open label exploratory study designed to evaluate the safety, efficacy, and pharmacokinetics of paltusotine, an oral selective nonpeptide somatostatin receptor type 2 (sst2) biased agonist, in patients with acromegaly whose disease is not biochemically controlled by octreotide LAR or lanreotide depot alone. EVOLVE is a double-blind, placebo-controlled, randomized withdrawal study designed to evaluate the safety, efficacy, and pharmacokinetics of paltusotine in patients with acromegaly whose disease is biochemically controlled by octreotide LAR or lanreotide depot monotherapy.

Initiated Phase 1 trial of CRN01941. In May 2019, Crinetics initiated a Phase 1, double blind, randomized, placebo-controlled, single- and multiple-dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of CRN01941 in healthy volunteers. Like paltusotine, CRN01941 is an oral nonpeptide sst2 biased agonist. CRN01941 is initially in development as a potential treatment for neuroendocrine tumors (NETs) that originate from neuroendocrine cells commonly found in the gut, lung, or pancreas. Upon analysis of the cumulative data from the paltusotine and CRN01941 preclinical, nonclinical and clinical programs, Crinetics intends to decide whether to advance CRN01941 or paltusotine into a later stage trial in NETs.

Expanded management team and board of directors. In March 2019, Crinetics appointed Gina Ford, R.Ph., MBA, as Vice President, Corporate Strategy and Commercial Planning. Ms. Ford joined Crinetics from One Joule, LLC a commercial and corporate strategy consulting company she founded, where she provided biopharmaceutical clients with strategic advice on corporate, commercial, marketing and global market access strategy. Among her prior roles, Ms. Ford served as Head of Endocrinology with Ipsen Pharmaceuticals, where she led the endocrine franchise, which included the commercial leadership of a drug to treat acromegaly and neuroendocrine tumors. In July 2019, Crinetics appointed Stephanie S. Okey, M.S. to its board of directors as an independent board member. Ms. Okey brings extensive leadership and management experience in senior commercial roles including, most recently, Head of North America and U.S. General Manager of Rare Diseases at Genzyme.

Fourth Quarter and Full Year 2019 Financial Results

Research and development expenses were $12.1 million and $41.5 million for the three months and full year ended December 31, 2019, respectively, compared to $7.7 million and $24.5 million for the same periods in 2018. The increases were primarily attributable to clinical development and manufacturing activities for paltusotine and CRN01941 as well as the company’s preclinical programs.

General and administrative expenses were $3.4 million and $13.5 million for the three months and full year ended December 31, 2019, compared to $2.6 million and $6.7 million for the same periods 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 December 31, 2019 was $14.5 million, compared to a net loss of $8.5 million for the same period in 2018. For the year ended December 31, 2019, the company’s net loss was $50.4 million compared to a net loss of $27.1 million for the year ended December 31, 2018.

Unrestricted cash, cash equivalents and investments totaled $118.4 million as of December 31, 2019, compared to $131.7 million as of September 30, 2019 and $163.9 million as of December 31, 2018. Crinetics expects that its cash, cash equivalents and investments will fund its current operating plan into the second half of 2021.

As of February 28, 2020, the company had 24,566,896 common shares outstanding.

On March 9, 2020 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported financial results for the year ended December 31, 2019 and provided a corporate update (Press release, Cellectar Biosciences, MAR 9, 2020, View Source [SID1234555315]).

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Fourth Quarter and Recent Corporate Highlights

·Announced CLR 131 achieved primary efficacy endpoints from its Phase 2 CLOVER-1 study in relapsed/refractory B-cell lymphomas and completion of the Phase 1 relapsed/refractory multiple myeloma Dose Escalation study. The data showed:

o42.8% overall response rate (ORR) in median 6th line treatment of multiple myeloma at the 75mCi total body dose

o81.8% of the multiple myeloma patients across all therapeutic doses tested experienced tumor reduction with a strong dose response

o100% ORR and 25% complete response (CR) seen in lymphoplasmacytic lymphoma/Waldenstrom’s macroglobulinemia (LPL/WM) patients

o42.0% ORR and 11% CR in all non-Hodgkin’s lymphoma (NHL) patients

·Received Orphan Drug Designation for CLR 131 in lymphoplasmacytic lymphoma (LPL) from the U.S. Food and Drug Administration

·Oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Conference entitled "Fractionated Dosing of CLR 131 in Patients with Relapsed or Refractory Multiple Myeloma"

·Oral presentation at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress entitled "Interim Evaluation of a Targeted Radiotherapeutic, CLR 131, in Relapsed/Refractory Diffuse Large B-cell Lymphoma Patients"

·Scientific conference poster presentations during the quarter included:

oAmerican Association for Cancer Research (AACR) (Free AACR Whitepaper) San Antonio Breast Cancer Symposium entitled "Phospholipid ether delivery vehicle shows specificity for a broad range of tumor cells and provides a novel and improved approach for targeted therapy"

oAACR-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference entitled "CLR 180099, a lipid raft targeted phospholipid-drug conjugate, shows potent improved safety and efficacy against colorectal tumors"

oUK-AACR Joint Conference on Engineering and Physical Sciences in Oncology entitled "Preclinical evaluation of a novel phospholipid drug conjugate, CLR 2000045 with a combretastatin A-4 analogue for improved breast cancer therapy"

·Strengthened the management team with the appointment of Dr. Igor Grachev, Chief Medical Officer

"Data from our CLR 131 Phase 1 dose escalation study and the Phase 2 CLOVER-1 study demonstrated a unique safety profile and an encouraging response rate of nearly 43% as a sixth-line treatment for relapsed/refractory multiple myeloma," said James Caruso, President and CEO of Cellectar. "Importantly, the 75mCi dose demonstrated excellent activity in very challenging to treat subpopulations, high-risk, triple class refractory and penta-refractory. We plan to enroll additional patients at 100mCi of CLR 131 in the two-cycle dosing optimization regimen, which we believe will further increase response rates, the durability of responses and will likely be used in our pivotal study planned for initiation in Q4 of this year."

2019 Financial Highlights

Cash and Cash Equivalents: As of December 31, 2019, the company had cash, cash equivalents and restricted cash of $10.6 million compared to $13.3 million at December 31, 2018. Cash provided by financing activities was $9.0 million, offset by cash used in operating activities of $11.7 million. Consistent with prior guidance, the company believes its cash on hand is adequate to fund operations into the first quarter of 2021.

Research and Development Expense: Research and development expense for the year ended December 31, 2019 was $9.0 million, compared to $6.8 million for the year ended December 31, 2018. The overall increase in research and development expense of approximately 32% was primarily attributable to an increase in clinical project costs largely related to the startup of the pediatric study, as well as an increase in patient recruitment for the ongoing clinical studies.

General and Administrative Expense: General and administrative expense for the year ended December 31, 2019 was $5.2 million, compared to $4.8 million for the year ended December 31, 2018. The increase of 8% in general and administrative costs was primarily related to an increase in personnel and consulting costs and an increase related to public company expenses, rent and depreciation. These costs were offset by a decrease in accounting fees and restructuring charges.

Net Loss: The net loss attributable to common stockholders for the year ended December 31, 2019 was ($14.1) million, or ($1.84) per share, compared to ($15.5) million, or ($5.23) per share, in 2018.

On March 9, 2020 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported financial results for the fourth quarter and year ended December 31, 2019 and provided an update on clinical and corporate developments (Press release, Deciphera Pharmaceuticals, MAR 9, 2020, View Source [SID1234555314]).

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"2019 was a year of outstanding execution for Deciphera," said Steve Hoerter, President and Chief Executive Officer. "In 2020, our top priority is preparing for the potential approval and launch of ripretinib now that the NDA has been accepted by the FDA for Priority Review. Patients with GIST are in need of a new treatment option, and we believe ripretinib has the potential to transform the treatment of this disease."

Mr. Hoerter continued, "Our broad development program for ripretinib is on track, and we look forward to the completion of enrollment in INTRIGUE, our pivotal Phase 3 study of ripretinib in second-line GIST, in the second half of this year. Beyond ripretinib, we remain focused on the balance of our wholly-owned, clinical-stage pipeline, with updated clinical data for both DCC-3014 and rebastinib, as well as an IND submission for DCC-3116, expected later this year."

Recent Program Highlights

Ripretinib
Announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) seeking approval for ripretinib for the treatment of patients with advanced gastrointestinal stromal tumors (GIST) who have received prior treatment with imatinib, sunitinib, and regorafenib, and assigned a Prescription Drug User Fee Act (PDUFA) target action date of August 13, 2020.

The NDA is being reviewed by the FDA under the Oncology Center of Excellence Real-Time Oncology Review (RTOR) pilot program. This pilot program aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible, while maintaining and improving review quality. Additional information about RTOR can be found at: View Source

Received Priority Review, under the Project Orbis initiative, for the marketing authorization applications in Canada and Australia. Project Orbis, an initiative of the FDA Oncology Center of Excellence, is designed to provide a framework for concurrent submission and review of oncology products among international partners. Additional information about the Project Orbis initiative can be found at: View Source
DCC-3014
Presented preliminary data from the ongoing Phase 1 study of DCC-3014, including anti-tumor activity in three initial patients with diffuse-type tenosynovial giant cell tumor (TGCT). These data, which provide clinical proof-of-concept for DCC-3014’s potential in diffuse-type TGCT, were presented at the Connective Tissue Oncology Society (CTOS) 2019 Annual Meeting. DCC-3014 was also shown to be generally well tolerated with no reported grade 3 or higher treatment-emergent adverse events in initial diffuse-type TGCT patients.
Rebastinib
Announced the selection of the Phase 2 dose for rebastinib in the Phase 1b/2 study in combination with carboplatin and activation of Part 2 of the study in patients with breast cancer, ovarian cancer, and mesothelioma.
Recent Corporate Updates

Announced the closing of an underwritten public offering of 3,659,090 shares of common stock at a public offering price of $55.00 per share in February 2020. Total net proceeds to Deciphera were approximately $188.4 million, after deducting underwriting discounts and commissions and other offering expenses.
Announced the appointment of Ron Squarer to its Board of Directors. Mr. Squarer served as Chief Executive Officer and a member of the Board of Directors of Array BioPharma, Inc. from 2012 until its acquisition by Pfizer Inc. in August 2019 following the successful commercial launches of both Braftovi and Mektovi and brings over two decades of experience in the biopharmaceutical industry.
Announced the appointment of Frank S. Friedman to its Board of Directors. Mr. Friedman recently served as the global Chief Operating Officer of Deloitte Touche Tohmatsu Limited, culminating a 40-year career at the organization.
Fourth Quarter 2019 Financial Results

Cash Position: As of December 31, 2019, cash, cash equivalents, and marketable securities were $579.6 million, compared to cash and cash equivalents of $293.8 million as of December 31, 2018. The increase was primarily due to the follow-on public offering in the third quarter of 2019 and did not include the proceeds from the Company’s follow-on public offering completed in February 2020. We expect our current cash, cash equivalents, and marketable securities, together with the proceeds from our recent follow-on public offering in February 2020, will enable us to fund our operating and capital expenditures into the second half of 2022.
R&D Expenses: Research and development expenses for the fourth quarter of 2019 were $46.6 million, compared to $27.4 million for the same period in 2018. The increase was primarily due to personnel costs as well as clinical trial costs related to ripretinib, DCC-3014, and rebastinib. Non-cash stock-based compensation was $2.5 million and $1.0 million for the fourth quarters of 2019 and 2018, respectively.
SG&A Expenses: Selling, general, and administrative expenses for the fourth quarter of 2019 were $23.7 million, compared to $6.5 million for the same period in 2018. The increase was primarily due to personnel costs as well as external spend associated with commercial readiness and moving to our new headquarters. Non-cash stock-based compensation was $2.9 million and $1.8 million for the fourth quarters of 2019 and 2018, respectively.
Net Loss: For the fourth quarter of 2019, Deciphera reported a net loss of $67.2 million, or $1.31 per share, compared with a net loss of $32.3 million, or $0.86 per share, for the same period in 2018. The increase in the net loss is primarily due to the increase in R&D expenses and G&A expenses discussed above.
Conference Call and Webcast

Deciphera will host a conference call and webcast to discuss this announcement today, March 9, 2020 at 4:30 PM ET. To access the live call by phone please dial (866) 930-5479 (domestic) or (409) 216-0603 (international); the conference ID is 1669368. A live audio webcast of the event may also be accessed through the "Investors" section of Deciphera’s website at www.deciphera.com. A replay of the webcast will be available for 30 days following the event.

On March 9, 2020 Numab Therapeutics reported the closing of its Series B financing round at a total volume of CHF 22M (approximately USD 22.6M) (Press release, Numab, MAR 9, 2020, View Source [SID1234555313]). New investors in this round included 3SBio/Sunshine Guojian, Mitsubishi UFJ Capital Co., Ltd. and Eisai Co., Ltd. as well as Numab’s board member Dr. Daniel Vasella . Existing shareholders that participated in Numab’s Series A round also contributed to today’s financing. With the financing secured, Numab plans to further broaden its proprietary pipeline and accelerate the development for a number of programs towards the clinic. The company also plans to initiate a clinical trial for its lead oncology program ND021 during the course of 2020.

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Mitsubishi UFJ Capital is one of Asia’s leading venture capital firm focusing on life science, information and communications technology and high technology investments. Numab and Eisai entered into a global research and option agreement to discover and develop a portfolio of multi-specific antibody immunotherapies for cancer in October 2019. In December 2019, Numab added a regional alliance with 3SBio’s subsidiary Sunshine Guojian Pharmaceutical Co., Ltd. to its growing roster of pharmaceutical partnerships.

"We are very pleased to have attracted a renowned institutional investor in Mitsubishi UFJ Capital to the Numab story and likewise appreciate the additional display of confidence in our platform and pipeline strategy by our partners as well as existing Series A investors and our board member Daniel Vasella," commented Dr. David Urech, Chief Executive Officer of Numab Therapeutics.

Multi-specific antibodies have the potential to unlock entirely novel modes-of-action aiming at superior benefit-to-risk profiles relative to conventional cancer immune therapies. Numab’s proprietary MATCH technology platform represents one of the most versatile and flexible sources for multi-specific antibodies. MATCH molecules can incorporate up to six binding specificities in true plug-and-play fashion. The individual antibody Fv building blocks are designed for maximum stability and developability

On March 9, 2020 Zentalis Pharmaceuticals reported set a preliminary $100 million target for its IPO, according to documents filed with the Securities and Exchange Commission (Press release, Zentalis Pharmaceuticals, MAR 9, 2020, View Source [SID1234555312]). The New York-based company has applied for a Nasdaq listing under the stock symbol "ZNTL." That company is joined by Ayala Pharmaceuticals and Lyra Therapeutics, both of which also filed IPO paperwork last Friday.

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Zentalis is developing small molecules to address biological pathways of cancers. The company says in its prospectus its small molecules are different than currently marketed therapies, and that they also have the potential to address large populations of cancer patients.

The Zentalis pipeline consists of four compounds, three of which are in early-stage clinical testing. Lead drug candidate, ZN-c5, is designed to target estrogen receptors (ER), proteins to which the hormone estrogen binds. Tumors that are ER positive and human epidermal growth factor receptor (HER2) negative rely on these proteins for their growth and survival. Zentalis’s ZN-c5 is an oral drug that’s a selective estrogen receptor degrader (SERD). The compound is in Phase 1/2 testing.

The Zentalis drug is intended to challenge fulvestrant (Faslodex), an AstraZeneca (NYSE: AZN) SERD. In 2018, the last year prior to the launch of generic competition, fulvestrant accounted for more than $1 billion in revenue for AstraZeneca. This drug is administered via two 5 mL intramuscular injections given once a month. In addition to being painful, injections of the drug into the muscle restricts how much ER degradation can be achieved, limiting its efficacy, Zentalis says.

As a once-daily pill, Zentalis believes its drug would offer patients more convenience. The drug might also be more effective. According to early data from a Phase 1/2 study, the drug was rapidly absorbed and achieved high exposure levels in the body. The Zentalis drug was well tolerated by patients and no dose-limiting toxicities were reported. Additional preliminary data from the dose escalation portion of the study are expected in the second half of this year.

The Zentalis drug is also being tested in combination with palbociclib (Ibrance), a Pfizer (NYSE: PFE) cancer drug that is FDA approved as a treatment for HR positive, HER2 negative breast cancer. Under an agreement with Pfizer, the pharmaceutical giant is supplying its drug for the study at no cost to Zentalis, according to the filing. Zentalis is responsible for conducting the study and paying for it. The agreement does not give Pfizer the right to participate in future Zentalis clinical trials, and each company keeps rights to their respective drugs.

The Zentalis pipeline also includes ZN-c3 and ZN-e4, drugs in early-stage development for solid tumors and non-small cell lung cancer respectively. Preliminary data for those tests are expected in 2021. Another Zentalis compound, ZN-d5, is an experimental treatment for B-cell lymphoma. In the first half of this year, the company plans to seek FDA clearance to begin clinical testing of that drug. Zentalis says in the prospectus that it will use the IPO proceeds to continue development of its three clinical-stage compounds and to advance its B-cell lymphoma drug to human testing.

Since its 2014 founding, Zentalis has raised $162.1 million, according to the filing. The most recent financing was an $85 million Series C round that closed in December. As of the end of last year, the company had $67.2 million in cash. Its largest shareholders include Recurium Equity, Matrix Capital Management Master Fund, and Viking Global Investors. The percentage of Zentalis held by those shareholders is more than 5 percent, but the exact amount was not disclosed.