On October 28, 2019 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the third quarter ended September 30, 2019 (Press release, CRISPR Therapeutics, OCT 28, 2019, View Source [SID1234549924]).

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"In 2019, we’ve made significant progress across several development programs, including ongoing enrollment of our CTX001 trials in beta thalassemia and severe sickle cell disease, with preliminary data expected from these programs later this year. We also began treating patients in our clinical trial for CTX110, our allogeneic CAR-T therapy, and are advancing additional CAR-T candidates toward clinical development," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "Looking forward, we expect a robust 2020, with continued focus on execution as we anticipate conducting five clinical trials in parallel."

Recent Highlights and Outlook

Beta Thalassemia and Sickle Cell Disease

Enrollment is ongoing at six clinical trial sites in the U.S., Canada and Europe for the Phase 1/2 study of CTX001 in patients with transfusion-dependent beta thalassemia (TDT) and at ten clinical trial sites in the U.S., Canada and Europe for the study in patients with severe sickle cell disease (SCD).

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion for orphan drug designation (ODD) of CTX001 for the treatment of TDT. In addition, we are expanding the TDT patient population for CTX001 to include beta zero/beta zero subtypes.

The Company expects to release preliminary safety and efficacy data from the ongoing Phase 1/2 clinical trials in late 2019.

Immuno-Oncology

The Company has begun treating patients in a Phase 1/2 trial to assess the safety and efficacy of CTX110, its wholly-owned allogeneic CAR-T cell therapy targeting CD19+ malignancies. The multi-center, open label trial is designed to enroll up to 95 patients and investigate several dose levels of CTX110. The study is currently enrolling at five clinical trial sites in the U.S. and Australia. In addition, the Company obtained approval from Health Canada for its Clinical Trial Application (CTA). The Company believes its CRISPR-based allogeneic CAR-Ts may have a superior product profile compared to current autologous therapies and allow accessibility to broader patient populations.

The Company expects to initiate a Phase 1/2 clinical trial of CTX120, targeting B-cell maturation antigen (BCMA) for the treatment of multiple myeloma, in the first half of 2020. CRISPR Therapeutics continues to advance additional allogeneic CAR-T candidates toward clinical development including CTX130, targeting CD70 for the treatment of solid tumors and hematologic malignancies. The Company continues to scale its capabilities to enable rapid advancement of these programs into and through the clinic.

The Company recently announced it entered into a license agreement with KSQ Therapeutics whereby CRISPR Therapeutics gained access to KSQ intellectual property (IP) for editing certain novel gene targets in its allogeneic oncology cell therapy programs, and KSQ gained access to CRISPR Therapeutics’ IP for editing novel gene targets identified by KSQ as part of its current and future eTILTM (engineered tumor infiltrating lymphocyte) cell programs. The agreement further strengthens the Company’s proprietary allogeneic CAR-T platform.

The Company will present a poster at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference on November 9, 2019 related to single-cell RNA sequencing and functional assessment of healthy donor and cancer patient-derived T and CAR-T cells (#P187).

Regenerative Medicine

On September 17, 2019, CRISPR Therapeutics, in collaboration with ViaCyte, presented positive in vitro data for a potential immune-evasive cell replacement therapy for diabetes at the 55th Annual Meeting of the European Association for the Study of Diabetes (EASD) in Barcelona, Spain. The oral presentation included new data that demonstrate the successful differentiation of CRISPR-edited human pluripotent stem cells to pancreatic precursor cells.

Other Corporate Matters

Vertex has exercised the options granted under the collaboration it established with CRISPR Therapeutics in 2015 to in-license three additional targets for the development of treatments using CRISPR-based gene editing. The targets include the cystic fibrosis transmembrane conductance regulator (CFTR) gene and two undisclosed targets. Under the terms of the agreement, CRISPR Therapeutics will receive an upfront payment of $30 million in connection with the option exercise and has the potential to receive up to $410 million in development, regulatory and commercial milestones and royalties on net product sales for each of the three targets, and Vertex will receive exclusive rights to develop and commercialize products related to these targets globally. The research term of the Company’s 2015 collaboration with Vertex has now expired, and Vertex no longer holds rights to in-license additional targets under that agreement.

The Company recently announced proposed plans that Casebia Therapeutics, previously a joint venture between CRISPR Therapeutics and Bayer, would operate under the direct management of CRISPR Therapeutics. Upon closing of the transaction, Casebia Therapeutics would focus on the development of its lead programs in hemophilia, ophthalmology and autoimmune diseases, with Bayer having opt-in rights for two products at IND submission. The transaction is subject to negotiation and execution of definitive agreements as well as certain customary conditions. The Company and Bayer are negotiating the definitive agreements and, subject to the finalization of the definitive agreements and satisfaction of closing conditions, anticipate to close the transaction in the fourth quarter of 2019.
Third Quarter 2019 Financial Results

Cash Position: Cash as of September 30, 2019, was $629.7 million, compared to $427.9 million as of June 30, 2019, an increase of $201.8 million as increased cash operating expenses were offset by $68.6 million net proceeds from financing activities and $175 million upfront payments received from Vertex related to the 2019 collaboration agreement announced in June.

Revenues: Total collaboration revenues were $211.9 million for the third quarter of 2019 compared to $0.6 million for third quarter of 2018 with the increase primarily driven by the collaboration agreement with Vertex.

R&D Expenses: R&D expenses were $57.2 million for the third quarter of 2019 compared to $39.8 million for the third quarter of 2018. The increase was driven by increased headcount and services expense supporting the advancement of the hemoglobinopathies program, the broadening of the Company’s wholly-owned immuno-oncology portfolio, as well as increased investment in the Company’s CRISPR/Cas9 platform research and some non-cash expense related to the Company’s collaboration with Vertex.

G&A Expenses: General and administrative expenses were $15.5 million for the third quarter of 2019 compared to $10.2 million for the third quarter of 2018. The increase was driven by increased headcount-related expense and external professional and consulting service expense.

Net Income (Loss): Net Income was $138.4 million for the third quarter of 2019 compared to a loss of $50.7 million for the third quarter of 2018, driven predominantly by increased revenue recognized in connection with the Company’s collaboration with Vertex.

On October 28, 2019 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") reported key interim findings from the pivotal Phase 3 SIERRA trial of Iomab-B, including feasibility and safety data, at 50% of total patient enrollment (Press release, Actinium Pharmaceuticals, OCT 28, 2019, View Source [SID1234549923]). The SIERRA trial (Study of Iomab-B in Elderly Relapse/Refractory Acute Myeloid Leukemia) is a 150-patient, 1:1 randomization Phase 3 pivotal trial that is studying Iomab-B (Iodine-131 apamistamab) compared to physician’s choice of salvage chemotherapy in patients age 55 and above with active, relapsed or refractory AML (Acute Myeloid Leukemia). These interim data provide information on safety and feasibility of using Iomab-B to enable a BMT (Bone Marrow Transplant), the only curative treatment option in this patient population that is not typically considered eligible for BMT.

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Key findings from the first 75 patients enrolled (50% of total enrollment) in the trial include:

– Patients enrolled in the SIERRA trial were a median age of 64 (55-76) and had active disease with a median bone marrow blast percentage at randomization of 28 percent (5-97%). All patients were intermediate or poor, adverse risk groups. Two-thirds (68%) of the patients enrolled were in the poor prognostic, adverse cytogenetic and molecular risk group.

– All patients receiving a therapeutic dose of Iomab-B (31/31) in the study arm received a bone marrow transplant and engrafted rapidly without delay in blood count recovery.

– Only 7/38 patients (18%) randomized to the control arm achieved an initial complete response (CR) and were able to proceed to a conventional bone marrow transplant.

– Of the 31 patients who failed the primary endpoint in the control arm (82% failure), 20 (64%) were considered eligible for potential rescue by treatment with Iomab-B followed by a BMT. All 20 patients (100%) who crossed over and received a therapeutic dose of Iomab-B followed by a BMT achieved engraftment without delay in blood count recovery despite high blast count burden prior to transplant (median 35%).

– 12/38 patients (32%) randomized to the control arm received targeted agents all of which were recently approved. 11/12 patients (92%) received the Bcl-2 inhibitor venetoclax with either an hypomethylating agent or low dose cytarabine. Of these, only 3/11 patients (27%) achieved a CR.

– 100-day Non-Relapse Transplant Related Mortality remains lower in the Iomab-B arm (1/31 patients or 3%) compared to patients in the control arm who received conventional transplants (2/7 or 29%). Of the 31 patients who received Iomab-B in the study arm, 30 are potentially evaluable for the primary endpoint compared to 5 in the control arm. This difference between study arm and control arm remains consistent with that reported at the interim update at 25% of enrollment.

– The SIERRA trial remains the only randomized Phase 3 clinical trial to offer BMT as an option for patients age 55 and above with active, relapsed or refractory AML.

Sergio Giralt, M.D., Chief of Adult BMT, Memorial Sloan Kettering Cancer Center; Chair, Myeloma Service, said, "The results from the first 50% of patients to be enrolled in the SIERRA trial continue to be highly encouraging, particularly Iomab-B’s ability to enable a transplant in this patient population that would otherwise be ineligible. Despite eight new therapies having been approved for patients with AML, better outcomes for patients with AML are needed, in particular for the large number of relapsed and refractory patients. Newly approved targeted agents are not curative and as seen in the SIERRA trial do not enable a high rate of potentially curative BMT."

*Footnotes for table can be found below Conference Call and Webcast information

Mark Berger, M.D., Actinium’s Chief Medical Officer, said, "We are thrilled that data from the halfway point of the SIERRA trial validate the promising interim safety and feasibility results from the first 25% of patients in this trial. It is particularly gratifying for me to see that these relapsed, refractory patients with heavy disease burden who received a therapeutic dose of Iomab-B were successfully transplanted. In addition, it is heartening that Iomab-B can be an effective pathway to transplant even when the recently approved targeted therapies unfortunately fail, as shown by our control arm and crossover data. With the first half of the trial behind us and with strong data in hand, we are focused on taking this message to hematologists and transplant physicians. We look forward to continuing to execute on our strategies to bring Iomab-B to patients as quickly as possible."

Conference Call and Webcast Information

Time and Date: 12:00 PM ET on Monday, October 28th
Registration Link: https://platform.cinchcast.com/ses/kSO55A9SLNGFoAvv1hgHpg~~
U.S./Canada Toll Free Dial-in: (855) 698-6739
Participant Dial-in: (646) 402-9440
Conference ID: 0417

A replay of the call will be available on the Investor Relations page of the Company’s website.

Table Footnotes:

Data available on 31 patients per arm
1 patient with circulating blasts and bone marrow <5% not included in median/range
No therapeutic dose (6) due to: declining KPS (3), infusion reaction (1), unfavorable biodistribution (1), post-randomization eligibility (1)
Ineligible for crossover (9) due to: hospice care/progression (4), declined/ineligible for BMT (2), died pre-crossover (3). Eligible for crossover (2), received dosimetry but not Iomab-B therapy due to declining status
Crossed-over and received therapeutic dose of Iomab-B
ANC engraftment data not available (1), platelet engraftment data not available (5)
ANC and platelet engraftment data not available (1), engraftment failure (1)
ANC engraftment data not available (2), platelet engraftment data not available (4)
1 patient at 161 days had delayed transplant due to infection and respiratory failure, received Iomab-B and BMT when stable
Data available on: Iomab-B study arm (30), CR after salvage therapy (7) and no CR after salvage crossed-over received Iomab-B and transplanted (16)
About the SIERRA Trial

The SIERRA trial (Study of Iomab-B in Elderly Relapse/Refractory Acute Myeloid Leukemia) is the only randomized Phase 3 trial that offers BMT (Bone Marrow Transplant) as an option for older patients with active, relapsed or refractory AML or acute myeloid leukemia. BMT is the only potentially curative treatment option for older patients with active relapsed or refractory AML and there is no standard of care for this indication other than salvage therapies. Iomab-B is an ARC (Antibody Radiation-Conjugate) comprised of the anti-CD45 antibody apamistamab and the radioisotope I-131 (Iodine-131). The 20 active SIERRA trial sites in the U.S. and Canada represent many of the leading bone marrow transplant centers by volume.

On October 28, 2019 Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage oncology company targeting the epigenetic causes of cancers, reported that it has entered into a $10.9 million common stock purchase agreement, including a $1.0 million initial common stock purchase, with Aspire Capital Fund, LLC, a Chicago-based institutional investor (Press release, Salarius Pharmaceuticals, OCT 28, 2019, View Source [SID1234549922]).

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With the investment by Aspire plus non-dilutive funding from the Cancer Prevention Research Institute of Texas (CPRIT) and ongoing financial support from the National Pediatric Cancer Foundation, Salarius believes it is well-capitalized to advance its current clinical programs through a number of near-term, value creating milestones, including early safety and efficacy data readouts from the ongoing Phase 1/2 clinical trial of Seclidemstat, Salarius’ lead drug candidate, in Ewing sarcoma and the Phase 1 clinical trial of Seclidemstat in advanced solid tumors.

Under the agreement, Aspire Capital is committed to purchase up to $10.9 million of Salarius’ common stock over a 30-month span extending into 2022, subject to certain terms and conditions. Immediately upon execution of the agreement, Aspire Capital made an initial purchase of 210,526 shares for $1.0 million, a per share purchase price of $4.75, which is equal to the closing sale price of Salarius’ shares on October 24, 2019, the date of the agreement. Any additional sales to Aspire Capital under the agreement will occur at the sole discretion of Salarius and at prices based on the market price of Salarius’ common stock at the time of each sale.

"This common stock purchase agreement with Aspire Capital provides Salarius with additional access to capital and financing flexibility allowing Salarius to further advance its lead drug candidate, Seclidemstat," commented David Arthur, Chief Executive Officer of Salarius. "We believe our non-dilutive financial support from both CPRIT and the National Pediatric Cancer Foundation, now combined with funding from Aspire Capital, puts Salarius in a good financial position as we continue our work targeting the epigenetic causes of cancer."

Steven G. Martin, the Managing Member of Aspire Capital, commented, "We are very pleased to announce this investment in Salarius as we recognize LSD1 as a novel and exciting target with ever increasing clinical validation in a broad range of cancers, including Ewing sarcoma and other advanced solid tumors, as well as hematologic cancers such as AML. Seclidemstat has demonstrated promising and potentially best-in-class characteristics driven by its differentiated reversible binding profile and inhibition of both the enzymatic and scaffolding functions of LSD1. Furthermore, we’re intrigued by recent evidence showing that the inhibition of LSD1 stimulates interferon production highlighting Seclidemstat’s potential as an important new immuno-oncology agent. Aspire Capital is confident that Salarius offers a good opportunity for near and long-term value creation."

Under the terms of the agreement with Aspire Capital, Salarius retains full control over the timing of any stock sales made under the agreement and the amount of stock sold to Aspire Capital. There are no warrants, options, financing swaps, derivatives or other securities associated with the agreement. The agreement contains no restrictions on the use of proceeds, financial covenants or restrictions on future financings and no rights of first refusal, participation rights, penalties or liquidated damages. Lastly, Salarius maintains the right to terminate the agreement at any time, at its discretion, without any additional cost or penalty. The proceeds from this agreement will be used for working capital and general corporate purposes.

In addition to the Common Stock Purchase Agreement, Salarius also entered into a Registration Rights Agreement with Aspire Capital. Additional details regarding the transaction, including the issuance of commitment fee shares, the Common Stock Purchase Agreement and related Registration Rights Agreement is available in Salarius’ Current Report on Form 8-K, filed today with the SEC. Salarius has filed a prospectus supplement to its Form S-3 shelf registration statement (File No. 333-231010), which was declared effective on May 17, 2019 by the U.S. Securities and Exchange Commission, qualifying the offer and sale of common shares to Aspire Capital. A copy of the Prospectus Supplement is available on EDGAR at www.sec.gov or may be obtained upon request to Salarius. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such jurisdiction.

On October 28, 2019, Celsion Corporation (the "Company"), reported that it entered into a common stock purchase agreement (the "Purchase Agreement") with Aspire Capital Fund, LLC, an Illinois limited liability company ("Aspire Capital") which provides that, upon the terms and subject to the conditions and limitations set forth therein, Aspire Capital is committed to purchase up to an aggregate of $10.0 million of shares of the Company’s common stock over the 24-month term of the Purchase Agreement (Filing, 8-K, Celsion, OCT 28, 2019, View Source [SID1234549921]). Concurrently with entering into the Purchase Agreement, the Company also entered into a registration rights agreement with Aspire Capital (the "Registration Rights Agreement"), in which the Company agreed to file one or more registration statements, as permissible and necessary to register under the Securities Act of 1933, as amended (the "Securities Act"), registering the sale of the shares of the Company’s common stock that have been and may be issued to Aspire Capital under the Purchase Agreement.

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Under the Purchase Agreement, after the Securities and Exchange Commission (the "SEC") has declared effective the registration statement referred to above, on any trading day selected by the Company, the Company has the right, in its sole discretion, to present Aspire Capital with a purchase notice (each, a "Purchase Notice"), directing Aspire Capital (as principal) to purchase up to 100,000 shares of the Company’s common stock per business day, up to $10.0 million of the Company’s common stock in the aggregate at a per share price (the "Purchase Price") equal to the lesser of:

the lowest sale price of the Company’s common stock on the purchase date; or
the arithmetic average of the three (3) lowest closing sale prices for the Company’s common stock during the ten (10) consecutive trading days ending on the trading day immediately preceding the purchase date.

In addition, on any date on which the Company submits a Purchase Notice to Aspire Capital in an amount of 100,000 shares, the Company also has the right, in its sole discretion, to present Aspire Capital with a volume-weighted average price purchase notice (each, a "VWAP Purchase Notice") directing Aspire Capital to purchase an amount of stock equal to up to 30% of the aggregate shares of the Company’s common stock traded on its principal market on the next trading day (the "VWAP Purchase Date"), subject to a maximum number of shares the Company may determine. The purchase price per share pursuant to such VWAP Purchase Notice is generally 97% of the volume-weighted average price for the Company’s common stock traded on its principal market on the VWAP Purchase Date.

The Purchase Price will be adjusted for any reorganization, recapitalization, non-cash dividend, stock split, or other similar transaction occurring during the period(s) used to compute the Purchase Price. The Company may deliver multiple Purchase Notices and VWAP Purchase Notices to Aspire Capital from time to time during the term of the Purchase Agreement, so long as the most recent purchase has been completed.

The Purchase Agreement provides that the Company and Aspire Capital shall not effect any sales under the Purchase Agreement on any purchase date where the closing sale price of the Company’s common stock is less than $0.25. There are no trading volume requirements or restrictions under the Purchase Agreement, and the Company will control the timing and amount of sales of the Company’s common stock to Aspire Capital. Aspire Capital has no right to require any sales by the Company, but is obligated to make purchases from the Company as directed by the Company in accordance with the Purchase Agreement. There are no limitations on use of proceeds, financial or business covenants, restrictions on future fundings, rights of first refusal, participation rights, penalties or liquidated damages in the Purchase Agreement. In consideration for entering into the Purchase Agreement, concurrently with the execution of the Purchase Agreement, the Company issued to Aspire Capital 100,000 shares of the Company’s common stock (the "Commitment Shares"). The Purchase Agreement may be terminated by the Company at any time, at its discretion, without any cost to the Company. Aspire Capital has agreed that neither it nor any of its agents, representatives and affiliates shall engage in any direct or indirect short-selling or hedging of the Company’s common stock during any time prior to the termination of the Purchase Agreement. Any proceeds from the Company receives under the Purchase Agreement are expected to be used for working capital and general corporate purposes.

The foregoing is a summary description of certain terms of the Purchase Agreement and the Registration Rights Agreement and, by its nature, is incomplete. Copies of the Purchase Agreement and Registration Rights Agreement are filed herewith as Exhibits 10.1 and 4.1, respectively, to this Current Report on Form 8-K and are incorporated herein by reference. All readers are encouraged to read the entire text of the Purchase Agreement and the Registration Rights Agreement.

The issuance of the Commitment Shares and all other shares of common stock that may be issued from time to time to Aspire Capital under the Purchase Agreement is exempt from registration under the Securities Act, pursuant to the exemption for transactions by an issuer not involving any public offering under Section 4(a)(2) of the Securities Act.

On October 28, 2019 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported the first clinical results from its Phase I/2 trial evaluating MRTX849, an investigational KRAS G12C inhibitor, in patients with solid tumors expressing KRAS G12C mutations (Press release, Mirati, OCT 28, 2019, View Source [SID1234549920]). MRTX849 demonstrated clinical activity, including objective responses, in patients with non-small cell lung cancer (NSCLC) and colorectal cancer (CRC). While the maximum tolerated dose (MTD) has not yet been established, dose expansion is underway, and the trial continues to enroll patients. These data were presented in an oral presentation at the 2019 AACR (Free AACR Whitepaper)-NCI-EORTC 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) in Boston.

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"Patients whose tumors carry the KRAS G12C mutation have a poor prognosis, are resistant to standard of care treatment and have no available targeted therapeutic options," said Charles M. Baum, M.D., Ph.D., President and Chief Executive Officer, Mirati Therapeutics. "Early efficacy and safety data from this Phase 1/2 trial demonstrate the potential of a potent and effective KRAS therapy. We look forward to investigating MRTX849 in patients with a variety of KRAS G12C mutated cancers and bringing the hope of a targeted therapy to them."

The ongoing Phase 1/2, first-in-human, open-label multicenter trial has enrolled 17 patients, including 10 patients with NSCLC, 4 patients with CRC, and 3 patients with other tumor types. Five dose cohorts have been evaluated: 150 mg, 300 mg, 600 mg, and 1200 mg, taken orally once daily, and 600 mg, taken orally twice daily. The trial enrolled single patient dose escalation cohorts in an accelerated titration design. Trial objectives include evaluation of safety, tolerability, pharmacodynamics (PD), pharmacokinetics (PK) and tumor response evaluated using RECIST v1.1 criteria.

As of the data cut-off date of October 11, 2019, 12 patients across all dose levels were evaluable for response with at least one radiographic scan.

At the highest dose (600 mg BID), three of five (3/5) evaluable patients with NSCLC and one of two (1/2) evaluable patients with CRC achieved a partial response (PR); the remaining patients experienced stable disease (SD).

Across all dose levels, three of six (3/6) patients with NSCLC and one of four (1/4) patients with CRC achieved a PR. Two responding patients (1 NSCLC and 1 CRC) achieved confirmed PRs, both with continuing tumor shrinkage following their first scan. The other two patients with PRs (both NSCLC) remain on study but have not yet had confirmatory scans.

Clinical PK data demonstrated that the dose of 600 mg BID results in drug levels that meet or exceed those likely to lead to full inhibition of KRAS G12C signaling.

Treatment duration across all dose levels ranged from 6.7- 38.6 weeks for patients with NSCLC and 9.9-30.1 weeks for patients with CRC as of the data cut-off.

Treatment-related adverse events (AEs) were primarily grade 1 events. One patient experienced a dose-limiting toxicity (DLT) at the 1200 mg QD dose (capsule burden intolerance [12 capsules]) and one patient experienced a DLT at the 600 mg BID dose (grade 3/4 isolated amylase/lipase increase). The MTD has not yet been established and further dose escalation may be explored. Enrollment into dose expansion at the 600 mg BID dose is underway.

"There are currently no effective targeted therapies for patients with KRAS-mutant cancers," said Pasi A. Jänne, M.D., Ph.D., Director of The Lowe Center for Thoracic Oncology at the Dana Farber Cancer Institute and MRTX849-001 investigator. "KRAS mutations are the most common oncogenic alteration in all of human cancers, and as such, finding a therapeutic approach for this subset of cancers would have tremendous clinical impact for cancer patients."

In addition, data demonstrating the efficacy of MRTX849 in preclinical studies were published simultaneously with the oral presentations at AACR (Free AACR Whitepaper)-NCI-EORTC 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), in Cancer Discovery, a journal of the American Association of Cancer Researchers (AACR) (Free AACR Whitepaper). "The KRAS G12C Inhibitor, MRTX849, Provides Insight Toward Therapeutic Susceptibility of KRAS Mutant Cancers in Mouse Models and Patients," by lead author Jill Hallin, Principal Scientist at Mirati and corresponding author James G. Christensen, Ph.D. Executive Vice President and Chief Scientific Officer at Mirati, describes the challenging research path leading to a novel therapy that directly targets KRAS. Cancer Discovery publishes high-impact, peer-reviewed articles describing major advances in research and clinical trials.

About MRTX849

MRTX849 is an investigational, orally-available small molecule that is designed to potently and selectively inhibit a form of KRAS which harbors a substitution mutation (G12C). KRAS G12C mutations are present in approximately 14% of NSCLC adenocarcinoma patients, 4% of colorectal cancer patients, and subsets of other types of cancer. Tumors characterized by KRAS G12C mutations are commonly associated with poor prognosis and resistance to therapy, and patients with these mutations have few treatment options. MRTX849 is being evaluated in a Phase 1/2 trial treating patients with molecularly-identified, KRAS G12C-positive advanced solid tumors.