On February 28, 2022 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported that management will be participating in the following investor conferences (Press release, Sangamo Therapeutics, FEB 28, 2022, View Source [SID1234609297]):

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Cowen 42nd Annual Health Care Conference
Date: Monday, March 7th at 12:50 p.m. Eastern Time
Barclays Global Healthcare Conference
Date: Thursday, March 17th at 9:00 a.m. Eastern Time
H.C. Wainwright & Co. Gene Editing and Therapy Conference 2022
Date: Wednesday, March 30th at 7:00 a.m. Eastern Time
Access links for presentations, panels and fireside chats will be available on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The presentations will also be available on the Sangamo Therapeutics website after the event.

On March 28, 2022 Merus N.V. (Nasdaq: MRUS) ("Merus", "we", or "our"), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics and Triclonics), reported financial results for the fourth quarter and full year ended December 31, 2021 and provided a business update (Press release, Merus, FEB 28, 2022, View Source [SID1234609296]).

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"We made significant progress in 2021, advancing our clinical programs, further developing our discovery and research pipeline and strengthening our balance sheet," said Bill Lundberg, M.D., President and Chief Executive Officer of Merus. "Our most advanced product candidate, zenocutuzumab remains on track for a clinical update on the eNRGy trial in the first half of 2022, and, if the rate of enrollment and efficacy remain consistent, we believe a sufficient number of patients will be enrolled in the eNRGy trial and Early Access Program, with sufficient follow up, by mid-2022, that could form the basis of a potential registrational data set. We also plan to provide clinical updates on our second most-advanced candidate, petosemtamab, and on MCLA-129 in the second half of this year."

Clinical Programs and Business Update

Zenocutuzumab, or "Zeno" (MCLA-128: HER3 x HER2 Biclonics)
NRG1+ Cancer: Phase 1/2 eNRGy trial clinical data and program update planned for first half of 2022

Zeno is currently being investigated in the phase 1/2 eNRGy trial to assess the safety and anti-tumor activity of Zeno monotherapy in NRG1+ cancer.

As of February 2022, more than 100 patients with NRG1+ cancer have been treated with Zeno monotherapy in our phase 1/2 eNRGy trial and Early Access Program ("EAP"). Merus plans to provide a clinical program update in the first half of 2022. In June 2021, Merus shared interim clinical data from an April 2021 data cutoff, demonstrating encouraging early clinical activity, with confirmed partial response rates observed to be 42% in NRG1+ pancreatic cancer (5 of 12), and 29% across all NRG1+ tumor types treated (13 of 45).

In November 2021, Merus reported that it met with the U.S. Food and Drug Administration (FDA) in an end-of-phase Type B meeting to discuss interim results from the ongoing phase 1/2 eNRGy trial and EAP in NRG1+ cancer, and to discuss the development plan for Zeno. Based on feedback received from the FDA, Merus believes that the trial design and planned enrollment will be appropriate to potentially support a Biologics License Application ("BLA") submission seeking a tumor agnostic indication for Zeno in patients with previously treated NRG1+ cancer.

In January 2021, the FDA granted Fast Track Designation to Zeno for the treatment of patients with metastatic solid tumors harboring NRG1 gene fusions (NRG1+ cancers) that have progressed on standard of care therapy.

Details of the eNRGy trial, including current trial sites, can be found at clinicaltrials.gov and Merus’ trial website at www.nrg1.com, or by calling 1-833-NRG-1234.

Petosemtamab, or "Peto" (MCLA-158: Lgr5 x EGFR Biclonics): Solid Tumors
Dose expansion continues in the phase 1 trial: update planned for second half of 2022

Peto is currently enrolling in a phase 1 open-label, multicenter study, and is in the expansion phase, in patients with solid tumors.

In October 2021, Merus reported early interim clinical data of our Peto program in patients with advanced head and neck squamous cell carcinoma (HNSCC) at the 2021 AACR (Free AACR Whitepaper)-NCI-EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). Among 10 patients with previously treated advanced HNSCC, as of the August 9, 2021 safety and efficacy data cutoff, the median age was 65 and the median number of prior lines of therapy was two. Seven patients were evaluable for an interim efficacy analysis by investigator assessment (three patients were enrolled <8 weeks before the data cutoff date). Three of seven patients achieved a partial response, with one of these three achieving complete response after the data cutoff date. Tumor reduction was observed in the target lesions of all seven patients. The safety results presented for Peto were based on 29 patients with advanced solid tumors who were treated at the recommended phase 2 dose across the phase 1 trial, and, as of the data cutoff, the most frequent adverse events (AEs) were infusion related reactions with 72% any grade and 7% grade 3 or greater. Mild to moderate skin toxicity (3% grade ≥3) was also observed.

MCLA-145 (CD137 x PD-L1 Biclonics): Solid Tumors
Phase 1 trial continues

MCLA-145 is currently enrolling a global, phase 1, open-label, single-agent clinical trial evaluating MCLA-145 in patients with solid tumors. In December 2021, Merus reported interim clinical data on MCLA-145 from the phase 1 trial in patients with solid tumors at the ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress 2021. As of the data cutoff date of July 14, 2021, 34 patients with advanced or metastatic solid tumors with median age of 60.5 years had been treated at 8 dose levels ranging from 0.4-75mg Q2W. The median (range) duration of treatment was approximately 6 (1-74) weeks. AEs observed were consistent with the mechanism of action of MCLA-145 and were generally managed with drug interruption and/or steroids in some patients. Preliminary evidence of antitumor activity was observed at doses ≥ 25 mg. Robust T-cell activation was observed across the 10 to 75 mg biweekly dosing range, including activation of cytotoxic CD8+ cells and elevation of cytokines.

Following Incyte’s (Nasdaq: INCY) election to opt-out of its ex-U.S. development of MCLA-145, announced earlier this year, Merus holds global rights to this program. Further clinical evaluation of MCLA-145 is planned, both as monotherapy and in combination with a PD-1 blocking antibody.

MCLA-129 (EGFR x c-MET Biclonics): Solid Tumors
Dose escalation continues in the phase 1 trial: update planned for the second half of 2022

MCLA-129 is currently enrolling in a phase 1/2, open-label clinical trial consisting of dose escalation followed by a planned dose expansion. Primary objectives of phase 1 are to determine the maximum tolerated dose and/or the recommended phase 2 dose, and the objectives of phase 2 are to evaluate safety, tolerability and potential clinical activity of the recommended phase 2 dose in patients with advanced solid tumors. MCLA-129 is subject to a collaboration and license agreement with Betta Pharmaceuticals Co. Ltd. (Betta), which permits Betta to exclusively develop MCLA-129 in China, while Merus retains global rights outside of China. In October 2021, Betta announced that the first patient was dosed in a phase 1/2 trial in China sponsored by Betta, of MCLA-129 in patients with advanced solid tumors.

Collaborations Update

Incyte

In the third quarter of 2021 Merus received a milestone payment for achieving pre-clinical candidate nomination of a novel bispecific antibody (target pair program) under the global collaboration and license agreement with Incyte. Candidate nomination has triggered this program’s next phase of IND-enabling studies by Incyte.

Merus receives reimbursement for research activities related to the collaboration and is eligible to receive potential development, regulatory and commercial milestones and sales royalties for any products, if approved.

Loxo Oncology at Lilly

In January 2021 Merus and Loxo Oncology at Lilly, a research and development group of Eli Lilly and Company (Lilly) announced a research collaboration and exclusive license agreement to develop up to three CD3-engaging T-cell re-directing bispecific antibody therapies. The collaboration is progressing well with active research programs underway.

Corporate Activities

Completed two Public Offerings in 2021

In January and November 2021, Merus closed two public offerings, with the cumulative gross proceeds from the offerings of approximately $250 million, inclusive of underwriters’ 30-day option to purchase additional common shares at the public offering price. All of the shares in the offering were sold by Merus. Proceeds from these offerings are planned to be used to continue to fund Merus’ product candidates in clinical and preclinical development, as well as for general corporate purposes.

Shannon Campbell Appointed as Chief Commercial Officer

In February 2022, Merus appointed Ms. Campbell as Executive Vice President and Chief Commercial Officer to lead the commercial strategy for the most advanced clinical candidate, Zeno, as well as Merus’ robust pipeline of multispecific product candidates in development. Ms. Campbell brings over 25 years of pharmaceutical commercialization experience and joins Merus from Novartis Pharmaceuticals Corporation, where she led Novartis’s U.S. Oncology Solid Tumor Franchise and was responsible for a broad portfolio of therapies in oncology and rare diseases. Prior to Novartis, Ms. Campbell was with Bayer HealthCare Pharmaceuticals, where she was instrumental in helping to build, launch and lead Bayer’s U.S. Oncology business.

Cash Runway projected to be beyond 2024

Based on the Company’s current operating plan, Merus expects our existing cash, cash equivalents and marketable securities will fund Merus’ operations beyond 2024.

Full Year 2021 Financial Results

Collaboration revenue for the year ended December 31, 2021 increased $19.2 million as compared to the year ended December 31, 2020, primarily as a result of $17.3 million in Lilly reimbursement revenue that did not occur in 2020, increase of $3.0M in Incyte reimbursement, partially offset by a decrease in other collaboration revenue of $1.2M. The change in exchange rates did not materially impact collaboration revenue.

Research and development expense for the year ended December 31, 2021 increased $28.1 million as compared to the year ended December 31, 2020, primarily as a result of an increase in manufacturing related costs, and higher research and development-related costs related to our programs, particularly increases in costs for zenocutuzumab and for MCLA-129.

General and administrative expense for the year ended December 31, 2021 increased $5.1 million as compared to the year ended December 31, 2020, primarily as a result increases in stock-based compensation, insurance, facilities, and personnel costs, partially offset by a decrease in consulting.

Other income, net consists of interest earned on our cash and cash equivalents held on account, accretion of investment earnings and net foreign exchange gains or losses on our foreign denominated cash, cash equivalents and marketable securities.

Merus ended 2021 with cash, cash equivalents and marketable securities of $430.7 million as compared to $207.8 million at December 31, 2020. The increase was primarily the result of closing of the collaboration and license agreement and a share purchase agreement with Eli Lilly in January 2021 for a total of $60.0 million, the aggregate net proceeds from the January 2021 follow-on offering of $129.4 million and the aggregate net proceeds from the November 2021 follow-on offering of $118.7 million.

On February 28, 2022 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, reported financial results for the fourth quarter and year ended December 31, 2021, and provided operational updates (Press release, Turning Point Therapeutics, FEB 28, 2022, View Source [SID1234609241]).

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"We are encouraged by the progress made during 2021 and into the first two months of 2022," said Athena Countouriotis, M.D., President and CEO. "We look forward to sharing topline ORR and DOR data for our lead investigational therapy repotrectinib in patients with ROS1-positive NSCLC and completing a pre-NDA meeting with the FDA in the second quarter. Supported by our strong financial position, with approximately $1 billion in cash at the end of 2021, we continue to invest in our clinical programs and our research engine, while we also consider opportunities to bring in external innovation where there’s a strategic fit."

Fourth quarter and recent operational highlights include:

REPOTRECTINIB, ROS1/TRK INHIBITOR

Remain on track to report topline blinded independent central review (BICR) data from all of the ROS1-positive non-small cell lung cancer (NSCLC) cohorts from TRIDENT-1 and discuss the BICR data with the FDA at a pre-NDA meeting, as well as share topline ORR and DOR BICR data prior to the pre-NDA meeting, in the second quarter of 2022.
The Chinese Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted the company’s Greater China partner, Zai Lab, Breakthrough Therapy Designation for repotrectinib for the treatment of patients with ROS1-positive metastatic NSCLC who have not been treated with a ROS1 tyrosine kinase inhibitor (TKI).
Strong enrollment progress in the Phase 2 registration enabling TRIDENT-1 study across all cohorts during the fourth quarter of 2021 with expansion cohort 4 (EXP-4 — ROS1-positive advanced NSCLC population pretreated with one prior TKI without chemotherapy) now fully enrolled with the targeted 60 patients. Enrollment across all six cohorts of the study remains open and continues to progress steadily.
Updated durability data from ROS1-positive TKI-naïve NSCLC patients in the Phase 1 portion of the TRIDENT-1 trial continues to demonstrate best-in-class potential. Among a total of 7 patients treated at or above the recommended Phase 2 dose (RP2D), 3 patients had a DOR of greater than 30 months, using blinded independent central review (BICR) assessments as of the data cut-off date of July 22, 2019 followed by physician assessments as of the data cut-off date of November 29, 2021. 4 out of 7 patients remained on treatment for greater than 3 years.
Held a Type B meeting with the FDA to discuss potential next steps for repotrectinib in NTRK-positive TKI-pretreated advanced solid tumor patients treated within expansion cohort 6 (EXP-6) of the registrational TRIDENT-1 study.
The FDA guided that a pre-NDA meeting should be requested to discuss the topline BICR results from the Phase 2 TKI-pretreated EXP-6 and TKI-naïve EXP-5 patients, when responders have been followed for at least six months past onset of response.
The FDA noted that data from EXP-5 may be used to support the efficacy data for EXP-6, or potentially could be pooled with data from EXP-6 to support a broader indication.
The company plans to provide guidance on the timing of the pre-NDA meeting for repotrectinib in patients with NTRK-positive advanced solid tumors after completion of enrollment of the targeted 40 EXP-6 patients is achieved.
ELZOVANTINIB (TPX-0022), MET/SRC/CSF1R INHIBITOR

Patient enrollment continues in the SHIELD-1 study at 40 mg QD to 40 mg BID in Phase 1 dose expansion and in parallel elzovantinib is being studied at an intermediate dose level (60 mg QD to 60 mg BID) in Phase 1 dose escalation.
FDA clearance of the Investigational New Drug (IND) Application for the combination of elzovantinib and aumolertinib in EGFR mutant MET-amplified NSCLC. The combination of elzovantinib and aumolertinib will be studied in the SHIELD-2 Phase 1b/2 trial in patients with EGFR mutant MET-amplified advanced NSCLC who have progressed following treatment with osimertinib.
TPX-0046, RET INHIBITOR

Ongoing evaluation of multiple doses and schedules to further characterize the pharmacokinetics, safety, and efficacy profile before determining the RP2D.
TPX-0131, ALK INHIBITOR

Ongoing patient dosing in the Phase 1/2 FORGE-1 study of TPX-0131 in locally advanced or metastatic TKI-pretreated ALK-positive NSCLC. The study endpoints include safety and tolerability, determination of the RP2D, pharmacokinetics, and any early signals of efficacy.
DISCOVERY

Continued advancement of internal discovery programs targeting aberrant GTPase signaling known to drive genomically defined cancers with significant unmet medical need. The most advanced programs target KRAS G12D and the p21 activated kinase, or "PAK" family. The company is targeting nomination of two development candidates in the second half of 2022 with a goal to achieve at least one new IND per year beginning in 2023.
Upcoming Milestones
Key milestones anticipated in 2022 include:

Repotrectinib

Provide topline BICR data from all the ROS1-positive NSCLC cohorts from TRIDENT-1 and discuss the BICR data with the FDA at a pre-NDA meeting in the second quarter of 2022
Provide a clinical data update from the NTRK+ advanced solid tumor cohorts from TRIDENT-1 in the second half of 2022
Elzovantinib

Initiate the Phase 1b/2 SHIELD-2 study of elzovantinib in combination with aumolertinib in mid-2022
Initiate the Phase 2 portion of the SHIELD-1 study in the second half of 2022, pending FDA feedback on data from the intermediate dose level
Provide a clinical data update from the Phase 1 SHIELD-1 study in the second half of 2022
TPX-0131

Provide early interim data from initial patients treated in the dose-finding portion of the FORGE-1 study in the fourth quarter of 2022 or early 2023
Discovery

Nominate 2 development candidates in the second half of 2022
Provide details on the other 2 GTPase signaling discovery programs in the second half of 2022
Fourth Quarter and Full-Year 2021 Financial Results

Revenue: Revenue of $30.8 million for the year was attributable to the company’s licensing agreements with Zai Lab for repotrectinib and elzovantinib in Greater China.

R&D Expenses: Research and development expenses were $58.2 million for the fourth quarter compared to $34.3 million for the fourth quarter of 2020, and $193.0 million for the year compared to $113.4 million for the year ended December 31, 2020. Primary drivers of the year-over-year increase were investments made to develop repotrectinib, elzovantinib, TPX-0046, TPX-0131, discovery efforts and personnel expenses.

G&A Expenses: General and administrative expenses were $20.5 million for the fourth quarter compared to $13.7 million for the fourth quarter of 2020, and $75.9 million for the year compared to $73.4 million for the year ended December 31, 2020. G&A expenses in 2020 included a one-time non-cash stock-based compensation charge of $31.4 million associated with the modification of the vesting and expected term of the outstanding stock options pursuant to a transition agreement with our scientific founder.

Net Loss: Net loss was $78.4 million for the fourth quarter compared to net loss of $47.4 million for the fourth quarter of 2020, and $236.6 million for the year compared to $157.3 million for the year ended December 31, 2020.

Cash position: Cash, cash equivalents and marketable securities at December 31 totaled $981.6 million, reflecting a net decrease of $140.9 million from December 31, 2020. Turning Point projects its cash position is sufficient to fund current operations into the second half of 2024.

On February 28, 2022, Rocket Pharmaceuticals, Inc. (the "Company") reported that it entered into a sales agreement (the "Sales Agreement"), with Cowen and Company, LLC ("Cowen"), with respect to an at-the-market offering program pursuant to which the Company may offer and sell, from time to time at its sole discretion, shares of its common stock, par value $0.01 per share, having an aggregate offering price of up to $200,000,000 (the "Shares") through Cowen as its sales agent (Filing, 8-K, Rocket Pharmaceuticals, FEB 28, 2022, View Source [SID1234609212]). The Shares to be offered and sold under the Sales Agreement, if any, will be offered and sold pursuant to the Company’s shelf registration statement on Form S-3 (File No. 333-253756), which was filed with the Securities and Exchange Commission ("SEC") on March 2, 2021 and which became effective on September 10, 2021. The Company filed a prospectus supplement with the SEC on February 28, 2022 in connection with the offer and sale of the Shares pursuant to the Sales Agreement.

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Under the Sales Agreement, the Company will set the parameters for the sale of Shares, including the number of Shares to be issued, the time period during which sales of Shares are requested to be made, limitations on the number or dollar value of Shares that may be sold in any one trading day and any minimum price below which sales may not be made. Subject to the terms of the Sales Agreement, Cowen may sell the Shares by any method that is deemed to be an "at-the-market offering" as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended (the "Securities Act"), including sales made directly on The Nasdaq Global Market ("Nasdaq") or any other trading market for the Shares.

The Company or Cowen may suspend or terminate the offering of Shares upon notice to the other party and subject to other conditions. The offering of Shares pursuant to the Sales Agreement will terminate upon the earlier of (i) the sale of all Shares subject to the Sales Agreement or (ii) termination of the Sales Agreement in accordance with the terms and conditions set forth therein. Cowen will act as sales agent on a commercially reasonable efforts basis consistent with its normal trading and sales practices and applicable state and federal law, rules and regulations and the rules of Nasdaq.

The Company will pay Cowen a cash commission of up to 3.0% of gross proceeds from the sale of the Shares pursuant to the Sales Agreement. The Company has also agreed to provide Cowen with customary indemnification and contribution rights. The Company will also reimburse Cowen for certain expenses incurred in connection with the Sales Agreement.

The foregoing description of the Sales Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Sales Agreement, a copy of which is attached as Exhibit 10.1 hereto and is incorporated herein by reference.

Goodwin Procter LLP, counsel to the Company, has issued a legal opinion relating to the Shares. A copy of such legal opinion, including the consent included therein, is attached as Exhibit 5.1 hereto.

This Current Report on Form 8-K shall not constitute an offer to sell or solicitation of an offer to buy any Shares, nor shall there be any sale of the Shares in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities law of such state or jurisdiction.

On February 28, 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, and Sanwa Kagaku Kenkyusho Co., Ltd. ("Sanwa"), an established, fully integrated pharmaceutical company headquartered in Nagoya, Japan, reported that the parties have entered into a strategic partnership to exclusively develop and commercialize paltusotine in Japan (Press release, Crinetics Pharmaceuticals, FEB 28, 2022, View Source [SID1234609209]). Paltusotine is Crinetics’ investigational, orally available nonpeptide somatostatin receptor type 2 (SST2) agonist being evaluated as a treatment for acromegaly and neuroendocrine tumors (NETs), including NETs complicated by carcinoid syndrome.

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Under the terms of this agreement, Crinetics will receive $13.0 million upfront and will be eligible to receive milestone payments related to the achievement of certain development, regulatory and commercial goals. In addition, upon market approval of paltusotine in Japan, Crinetics will be eligible to receive tiered royalties based on net product sales. Sanwa will have an exclusive right to develop and commercialize the product in Japan and will be responsible for leading the development and commercialization of paltusotine for acromegaly and NETs in Japan. Also, Sanwa will assume all costs associated with clinical trials and regulatory applications associated with these processes. Crinetics retains all rights to develop and commercialize paltusotine outside Japan.

There are approximately 10,000 acromegaly patients and 11,000 NETs patients in Japan and, as in the United States, somatostatin analogues are the first-line medical therapy for individuals for whom surgery is either not prescribed or is not curative. Shusaku Isono, President and Chief Executive Officer of Sanwa Kagaku Kenkyusho Co., Ltd. said "Through this license agreement, we will make our best effort with Crinetics to provide a new oral treatment option for acromegaly and NETs patients in Japan."

"In Sanwa, we met a company that shares our vision of developing, as the first indication for paltusotine, a once-daily oral therapy for acromegaly that will establish a new class of medicine to allow patients to live full lives free of the burden of painful monthly injections," added Scott Struthers, Ph.D., founder and Chief Executive Officer of Crinetics. "With the promise of additional indications for NETs, we look forward to a long and productive relationship with our colleagues at Sanwa and are pleased to have the external validation that such a high-quality partnership provides."

Crinetics is currently enrolling patients in its Phase 3 PATHFNDR program, which is evaluating the safety and efficacy of once-daily oral paltusotine in a wide cross section of acromegaly patients in the United States and Europe. In Japan, Sanwa expects to initiate Phase 1 development with paltusotine in 2022.

ABOUT ACROMEGALY
Acromegaly is a serious disease generally caused by a pituitary adenoma, a benign tumor in the pituitary that secretes growth hormone. Excess GH secretion causes excess secretion of IGF-1 from the liver. Together, excess of these hormones leads to the symptoms and physical manifestations of acromegaly, including abnormal growth of hands and feet, alteration of facial features, arthritis, carpal tunnel syndrome, joint aches, deepening of voice due to enlarged vocal cords, fatigue, sleep apnea, enlargement of heart, liver and other organs, and changes in glucose and lipid metabolism. Surgical removal of pituitary adenomas, if possible, is the preferred initial treatment for most acromegaly patients. Pharmacological treatments are used for patients that are not candidates for surgery, or when surgery is unsuccessful in achieving treatment goals. Approximately 50% of patients with acromegaly prove to be candidates for pharmacological treatment. Long-acting somatostatin-receptor ligands (SRLs) are the most common initial pharmacologic treatment; however, these drugs require monthly depot injections with large gauge needles that are commonly associated with pain, injection site reactions, and increased burden of therapy on the lives of patients.

ABOUT NEUROENDOCRINE TUMORS AND CARCINOID SYNDROME
Carcinoid syndrome is a group of symptoms that presents in some individuals with neuroendocrine tumors (NETs). NETs are a rare, slow-growing type of cancer that arises most often in the digestive tract. Carcinoid syndrome is most common in patients with NETs that develop in the lung and gastrointestinal tract and metastasize to the liver. In these cases, the liver is unable to filter the hormones secreted by the NETs, causing them to be circulated systemically and inducing the symptoms of carcinoid syndrome which commonly include diarrhea and flushing.

ABOUT PALTUSOTINE
Paltusotine is an investigational, orally available nonpeptide agonist that is designed to be highly selective for the somatostatin receptor type 2 (SST2). It was designed by the Crinetics discovery team to provide a once-daily option for patients with acromegaly and neuroendocrine tumors. A previously completed Phase 1 trial of paltusotine showed clinical proof of concept by providing evidence of potent suppression of the growth hormone axis in healthy volunteers. In Phase 2 trials, paltusotine maintained IGF-1 levels in acromegaly patients who switched from injectable depot medications to once-daily oral paltusotine. IGF-1 is the primary biomarker endocrinologists use to manage their acromegaly patients.