On March 30, 2022 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 that financial results for the fourth quarter and year ended December 31, 2021 (Press release, Crinetics Pharmaceuticals, MAR 30, 2022, View Source [SID1234611191]).

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"We are excited to have started 2022 with the momentum that we generated in 2021 across all facets of the company," said Scott Struthers, Ph.D., founder and chief executive officer of Crinetics. "Last year was transformative for Crinetics as the company achieved multiple key milestones in our discovery and clinical programs, raised significant additional capital, and continued to add world-class talent to the company from the bench to the board. Since inception, we have had a strategy of investing in innovative drug discovery programs to build a diverse endocrine pipeline. We advance this pipeline by following a uniquely efficient development paradigm that leverages endocrine biomarkers from preclinical experiments through patient studies. I am proud of how this strategy came together over the past year to move us significantly closer to our vision of building the world’s leading endocrine company."

FULL YEAR 2021 AND RECENT HIGHLIGHTS
Reported positive proof-of-concept data from two Phase 1 programs of CRN04777 and CRN04894. In September 2021 and via a separate press release issued today, Crinetics announced positive data from a Phase 1 single- and multiple-ascending dose (MAD) study of CRN04777, a somatostatin receptor type 5 (SST5) agonist being developed as a treatment for congenital and syndromic hyperinsulinisms. The results supported clinical proof-of-concept, showing strong dose-dependent suppression of fasting insulin as well as dose-dependent suppression of glucose- and sulfonylurea-induced insulin secretion. In August 2021, Crinetics announced positive preliminary data from the single ascending dose (SAD) cohorts of an ongoing Phase 1 study of CRN04894, its adrenocorticotropic hormone (ACTH) antagonist being developed for the treatment of conditions of ACTH excess, including Cushing’s disease and congenital adrenal hyperplasia. The data supported clinical proof-of-concept by providing evidence of clinically relevant cortisol suppression as well as showing dose-dependent reductions in basal cortisol levels and suppression of cortisol following ACTH challenge. The data from the Phase 1 studies for CRN04777 and CRN04894 suggested that the molecules are both orally bioavailable and support once daily dosing schedules. Preliminary data from the MAD cohorts of the CRN04894 Phase 1 study
Initiated Phase 3 PATHFNDR program evaluating paltusotine in acromegaly. In the second quarter of 2021, Crinetics initiated its Phase 3 PATHFNDR program, which consists of two Phase 3 trials assessing the safety and efficacy of once-daily oral paltusotine. Together these trials are designed to evaluate paltusotine in a wide cross section of acromegaly patients. If successful, Crinetics believes these trials could support registration of paltusotine for all acromegaly patients who require pharmacotherapy, including untreated patients and those switching from standard of care. Topline data from both of these trials (PATHFNDR-1 and PATHFNDR-2) are expected to be available in 2023.
Entered into strategic licensing agreement with Sanwa Kagaku Kenkyusho Co., Ltd. ("Sanwa") for the development and commercialization of paltusotine in Japan. Per the agreement, Crinetics received $13 million upfront and is eligible to receive development, regulatory, and commercial milestones. In addition, Crinetics will be eligible to receive tiered royalties on net product sales should paltusotine receive marketing approval in Japan. In exchange, Sanwa was granted an exclusive right to develop and commercialize paltusotine in Japan and will assume all costs associated with clinical trials and regulatory applications in the territory. Crinetics retains all rights to develop and commercialize paltusotine outside of Japan.
Co-founded Radionetics Oncology. In October 2021, Crinetics, together with 5AM Ventures and Frazier Healthcare Partners, founded Radionetics Oncology, Inc., an independently operated company that aims to develop a deep pipeline of novel, targeted, nonpeptide radiopharmaceuticals for the treatment of a broad range of oncology indications. In conjunction with formation of the company, Radionetics received an exclusive world-wide license to Crinetics’ radiotherapeutics technology platform and associated intellectual property in exchange for equity, milestones in excess of $1 billion, and single-digit royalties on net sales. Radionetics launched with $30 million from a private financing with 5AM Ventures and Frazier Healthcare Partners as the sole investors.
Announced data from an open-label extension trial of paltusotine in acromegaly. Results showed that oral paltusotine maintained serum IGF-1 at levels previously achieved with injected somatostatin receptor ligands for up to 51 weeks. The results were featured in a poster presentation at the Society for Endocrinology BES congress, which can be found here.
Unveiled a parathyroid hormone receptor antagonist program. In September 2021, Crinetics announced its intent to develop a nonpeptide oral parathyroid hormone (PTH) receptor antagonist for the treatment of hypercalcemia associated with hyperparathyroidism (HPT) and other diseases of PTH receptor type 1 (PTHR1) over-activation. Crinetics is in the late stages of selecting a lead candidate from this family of compounds and anticipates initiation of IND-enabling studies in 2022. If successful, PTHR1 antagonists could represent a viable treatment option to improve the outcomes and experience of patients with primary hyperparathyroidism. Details on the preclinical efforts supporting the program were presented in a late-breaking poster at the annual meeting of the American Society for Bone and Mineral Research (ASBMR). More information on the program and a copy of the poster can be found here.
Strengthened balance sheet with successful common stock offerings. In April 2021, Crinetics completed an underwritten follow-on offering of 4,562,044 shares of its common stock at a price to the public of $16.44 per share, raising gross proceeds of approximately $75.0 million. In July 2021, Crinetics entered into a securities purchase agreement with Frazier Healthcare Partners for the private placement of 851,306 shares at $17.62 per share, raising gross proceeds of $15.0 million. In October 2021, Crinetics completed an underwritten public offering of 8,712,400 shares of its common stock at a price to the public of $19.80 per share, raising gross proceeds of approximately $172.5 million.
Strengthened company leadership with appointments to management team and Board of Directors. Throughout 2021 and in early 2022, Crinetics built upon its strong leadership and scientific expertise by appointing Garlan Adams to the role of general counsel, Jeff Knight to the role of chief operating officer, James Hassard to the role of chief commercial officer, Christopher Robillard to the role of chief business officer, and Dr. Rogério Vivaldi Coelho and Caren Deardorf to the Board of Directors.

FOURTH QUARTER AND FULL YEAR 2021 FINANCIAL RESULTS
Research and development expenses were $24.6 million and $84.3 million for the three months and full year ended December 31, 2021, respectively, compared to $16.8 million and $57.0 million for the same periods in 2020. The increases were primarily attributable to increased spending on manufacturing and development activities associated with our clinical and nonclinical activities for paltusotine, CRN04777, CRN04894, and our other preclinical research programs.
General and administrative expenses were $7.4 million and $24.5 million for the three months and full year ended December 31, 2021, respectively, compared to $5.0 million and $18.0 million for the same periods in 2020. The increases were primarily due to personnel-related costs.
Net loss for the three months ended December 31, 2021, was $30.8 million, compared to a net loss of $21.6 million for the same period in 2020. For the year ended December 31, 2021, the company’s net loss was $107.6 million compared to a net loss of $73.8 million for the year ended December 31, 2020.
Unrestricted cash, cash equivalents and investments totaled $333.7 million as of December 31, 2021, compared to $193.3 million as of September 30, 2021, and $170.9 million as of December 31, 2020.
Revenues were $1.1 million for the three months and full year ended December 31, 2021, consisting of non-cash upfront consideration recognized upon the transfer of intellectual property from Crinetics to Radionetics Oncology.
The company had 47,784,611 common shares outstanding as of March 25, 2022.

WEBCAST AND CONFERENCE CALL ON CRN04777 MULTIPLE-ASCENDING DOSE DATA
Crinetics will hold a conference call and live audio webcast today, March 30, 2022, at 4:30 p.m. Eastern Time to discuss results from the multiple-ascending dose cohorts of the Phase 1 trial of CRN04777. These results were announced in a separate press release issued earlier today, which is available on the company’s website. To participate, please dial 1-877-407-0789 (domestic) or 1-201-689-8562 (international) and refer to conference ID 13727857. To access the webcast, click here. Following the live event, a replay will be available on the Events page of the Company’s website.

On March 30, 2022 Acacia Pharma Group plc ("Acacia Pharma" or the "Company") (EURONEXT: ACPH), reported that it will not publish its results for the full year to end December 2021 on 31 March 2022 as originally scheduled (Press release, Acacia Pharma, MAR 30, 2022, View Source [SID1234611190]).

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This comes as a result of the changes in regulatory requirements following Brexit. The Company is required under Belgian law to have its accounts audited by either an EU audit firm or a third country audit firm registered in Belgium. Following Brexit, the Company’s statutory auditor PwC UK ceased to be an EU audit firm on 30 June 2021.

The Company has been informed that there is currently no legal mechanism for PwC UK to register as a third country audit firm in Belgium; the Company is therefore required to also have its accounts audited by PwC Belgium. A new date for the publication of its results will be set and these will be released before the end of April 2022 as required under the applicable rules.

On March 30, 2022 BioVaxys Technology Corp. (CSE:BIOV, FRA:5LB, OTCQB:BVAXF) ("BioVaxys" or "Company"), reported the expansion of its cancer vaccine platform with BVX-0922, its autologous haptenized tumor vaccine for colorectal cancer ("CRC") (Press release, BioVaxys Technology, MAR 30, 2022, View Source [SID1234611189]). BioVaxys plans to advance an Investigator-Sponsored Clinical Trial Application ("CTA") in the EU with the European Medicines Agency ("EMEA") this year for BVX-0922. An Investigator Sponsored CTA is submitted to regulatory authorities by a clinical investigator who both initiates and conducts an initial clinical study of a new drug or procedure, and under whose immediate direction the investigational drug is administered.

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CRC is the third most common malignancy and the second most deadly cancer world-wide, with an estimated 1.9 million new CRC cases diagnosed and 0.9 million deaths globally in 2020. The incidence of CRC is higher in highly developed countries, with global new CRC cases predicted to reach 3.2 million in 2040. When diagnosed early, the five-year relative survival rate for stage I and stage II colon cancer is 90%; however, CRC patients often experience no signs or symptoms associated with the disease. The 5-year survival rate for patients diagnosed at Stage IV is only 14% (Journal of Translational Oncology, Global Colorectal Cancer Burden in 2020 And Projections to 2040, Vol 14, Issue 10, October 2021).

A major benefit of the Company’s autologous haptenized tumor vaccine technology platform is the rapid scalability into a range of tumor types, especially those where the standard of care for these cancer patients typically involves surgical excision of tumor tissue. Access to these tumor cells is necessary for BioVaxys to manufacture autologous haptenized tumor cell vaccines, such as BVX-0918 for late-stage ovarian cancer or BVX-0922 for CRC.

The BioVaxys vaccine platform is based on the established immunological concept that modifying surface proteins—whether they are viral or tumor—with haptens makes them more visible to the immune system. This process of haptenization "teaches" a patient’s immune system to recognize and make target proteins more "visible" as foreign, thereby stimulating a T-cell mediated immune response. BioVaxys’ cancer vaccines are created by extracting a patient’s own (i.e., autologous) cancer cells, chemically linking with a hapten, and re-injecting them into the patient to induce an immune response to proteins which are otherwise not immunogenic.

Haptenization is a well-known and well-studied immunotherapeutic approach in cancer treatment, and has been evaluated in both regional and disseminated metastatic tumors. BioVaxys has a significant advantage over many other companies looking at cancer therapies in that it already has extensive promising clinical data for its cancer vaccines.

First-generation single-hapten vaccines invented by BioVaxys Co-Founder and Chief Medical Officer David Berd, MD, achieved positive immunological and clinical results in his previous FDA-approved Phase I and Phase II human trials in over 600 patients with different tumor types, as well as having no observed toxicity in years of clinical study.

For example, two studies of patients with metastatic melanoma were completed and published. The first trial tested the activity of the autologous, DNP-modified vaccine in 83 evaluable patients with incurable, metastatic melanoma (Berd et al, Int J Cancer 2001; 94: 531-539). Following vaccine administration there were 11 responses—2 complete, 4 partial, and 5 mixed; 2 patients were judged to have stable disease. Both complete responses and two of the four partial responses occurred in patients with lung metastases. Response durations were as follows: partial responses— 5, 6, 8, and 47+ months; complete responses—12, 29 months. In a second trial 214 patients with advanced stage III melanoma (lymph node metastases) underwent excision of large lymph node masses and then were administered the haptenized vaccine. With a median follow-up time of 5.1 years the 5-year overall survival rate was 46%, which is considerably higher than survival times reported with surgery alone (Berd et al, J. Clin. Oncology, 1997, 15:2359)

A first generation autologous, haptenized vaccine was also tested in two clinical trials conducted by Dr. Berd in women with advanced ovarian cancer who had ceased to respond to conventional chemotherapy. In the first trial 13 evaluable patients with bulky, chemotherapy-refractory disease were treated. The patients exhibited complete regression of a residual peritoneal mass by computed tomography (CT) and a concomitant fall in serum CA-125 (an ovarian cancer serum marker) from 65 to 6. Both the CT and CA-125 responses were maintained for 6 months.

In a second study (Taha et al, Gynecol Oncol 2014; 134, Abstract 25: 428-437), 26 subjects with recurrent platinum resistant ovarian cancer were enrolled. Vaccine was prepared for, but not administered to, 25 additional subjects. In six vaccinated subjects, CA125 levels became normal following surgery plus vaccine and remained normal throughout the 9-month duration of the protocol. Median overall survival by Kaplan–Meier method after surgery in the vaccinated group was 25.4 months compared to 6.5 months in the vaccine prepared but not administered group.

Finally, preliminary studies of a first generation, autologous, single-hapten vaccine have been performed in kidney cancer, non-small cell lung cancer, breast cancer, and acute myelogenous leukemia. The results indicated that preparation and administration of vaccine was feasible.

BioVaxys has enhanced the first-generation approach in these previous studies of using a single-hapten to now utilizing two haptens ("bi-haptenization"), which the Company believes will yield superior results. The global colorectal cancer therapeutics market is projected to reach $16.5 billion by 2026 (fortunebusinessinsights.com).

On March 30, 2022 Nextera AS, a drug and target discovery company built on the unique and novel NextCore protein engineering and discovery platform, reported a strategic collaboration with Zelluna Immunotherapy AS on optimizing T cell receptors (TCRs) for subsequent implementation into the "off-the-shelf" TCR based Natural Killer (TCR-NK) cell approach of Zelluna Immunotherapy AS for the treatment of cancer (Press release, Nextera, MAR 30, 2022, View Source [SID1234611188]).

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Nextera’s aim is to engage into strategic commercial partnerships focused on the huge unmet medical demand in autoimmunity and immuno-oncology by leveraging its NextCore platform. This partnership represents the first collaboration on engineering TCRs, which will complement the company’s focus on TCR-Like antibodies and HLA peptidome target deconvolution.

TCR-NK products are a novel and proprietary class of allogeneic cellular therapies that combine the inherent pan cancer killing mechanism, efficiency, and the allogeneic nature of NK cells with the targeting capabilities of TCRs. Arming NK cells with TCRs may unlock the potential to treat a wide range of currently untreatable cancers.

"We are delighted to engage into this collaboration with Zelluna, which is pioneering the TCR-NK field with its innovative and proprietary allogenic "off-the-shelf" approach. Engineered TCR therapy has very recently entered the mature clinical pipeline and further expansion of this field has the outlook to make a transformational difference for patients across multiple cancer indications. Our differentiated NextCore platform is uniquely well situated to harness the new generation of TCR targeting modules in these approaches to maximize efficacy and safety" said Dr. Geir Åge Løset, co-founder and CEO/CSO of Nextera.

On March 30, 2022 Alpine Immune Sciences, Inc. (Nasdaq: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune and inflammatory diseases, reported that Alpine will host a virtual investor event on April 12th, 2022 at 6:00pm ET, to coincide with the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (Press release, Alpine Immune Sciences, MAR 30, 2022, View Source [SID1234611171]).

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During the investor event, Alpine will provide a corporate update on the development strategy for davoceticept (ALPN-202) and review the company’s oral presentation at the AACR (Free AACR Whitepaper) Annual Meeting ("Monotherapy dose escalation of davoceticept (ALPN-202), a conditional CD28 costimulator and dual checkpoint inhibitor, in advanced malignancies (NEON-1)"). Following the presentation, Alpine will hold a question-and-answer discussion.

Currently, abstract titles are now live on the AACR (Free AACR Whitepaper) meeting website and a copy of Alpine’s presentation will be available on April 12th on the Scientific Publications page of Alpine’s website.

Investor Event – Conference Call and Webcast Details:

To access the investor event by phone, dial (800) 816-3005 (domestic) or (857) 770-0069 (international) and reference conference ID: 4238059.

A live webcast of the investor event will be available online in the investor relations section of the company’s website at View Source A replay will be available on the company website for 90 days following the webcast.

About Davoceticept (ALPN-202)

Davoceticept (ALPN-202) is a first-in-class, conditional CD28 costimulator and dual checkpoint inhibitor intended for the treatment of cancer. Preclinical studies of davoceticept have successfully demonstrated superior efficacy in tumor models compared to checkpoint inhibition alone. Completion of dose escalation and initiation of expansion cohorts of NEON-1 (NCT04186637), a Phase 1 monotherapy dose escalation and expansion trial in patients with advanced malignancies, is anticipated in the first half of 2022. NEON-2 (NCT04920383), a combination study of davoceticept (ALPN-202) and pembrolizumab was initiated in June 2021 and is currently on partial clinical hold.