Scholar Rock Reports Third Quarter 2023 Financial Results and Highlights Business Progress

On November 7, 2023 Scholar Rock (NASDAQ: SRRK), a Phase 3 clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results and corporate updates for the third quarter ended September 30, 2023 (Press release, Scholar Rock, NOV 7, 2023, View Source [SID1234637161]).

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"We have made significant progress across our pipeline over the last quarter, including completing enrollment of our Phase 3 SAPPHIRE trial of apitegromab, which was designed to build on the positive Phase 2 TOPAZ results. Based on the sustained improvement in motor function that was observed after 36 months of treatment in the TOPAZ trial, the favorable safety and tolerability profile, and the high continued participation rate in our long-term extension study, we believe apitegromab has the potential to be a transformative therapy for SMA patients," said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock. "Further, we are excited to leverage our expertise in selective myostatin inhibition and expand into cardiometabolic disorders, including obesity. We believe our highly selective myostatin inhibitor SRK-439 has the potential to help patients retain lean muscle mass, which has been a key obstacle for many on GLP-1 receptor agonist therapy, and we plan to file an IND in 2025 to initiate clinical testing of SRK-439 in combination with GLP-1 receptor agonists."

"Additionally, we recently presented new clinical and biomarker data from the Phase 1 DRAGON trial that demonstrated the therapeutic potential of SRK-181 in heavily pretreated patients with anti-PD-1 resistant clear cell renal cell carcinoma. We believe that the DRAGON trial has achieved the objective of establishing proof of concept that our highly selective approach to blocking latent TGFβ1 can restore sensitivity to a checkpoint inhibitor, most notably in those with anti-PD-1 resistant ccRCC and supports further development of SRK-181," he added.

Recent Company Highlights and Upcoming Milestones

Spinal Muscular Atrophy (SMA) Program

Apitegromab is an investigational fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle and is being developed as a potential first muscle-targeted therapy for the treatment of SMA.

Completed enrollment of its pivotal Phase 3 SAPPHIRE clinical trial. The Company announced that it completed enrollment in September 2023, with topline data expected in the fourth quarter of 2024. If successful and if approved, the Company expects to initiate a commercial product launch in 2025.
Presented encore 36-month extension data from the Phase 2 TOPAZ trial at the 28th Annual Congress of the World Muscle Society in October. The Company presented encore data from its Phase 2 TOPAZ trial extension period evaluating patient outcomes in SMA after 36 months of treatment of apitegromab, which showed long-term sustained improvements in motor function and in patient-reported outcome measures in patients with nonambulatory Types 2 and 3 SMA receiving survival motor neuron (SMN) therapy.
ONYX long-term extension study for patients from both the TOPAZ and SAPPHIRE studies remains ongoing.
Immuno-Oncology Program

SRK-181 is an investigational selective inhibitor of latent TGFβ1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy in patients with advanced cancer.

Presented clinical and biomarker data from Phase 1 DRAGON proof-of-concept trial at the SITC (Free SITC Whitepaper) 38th Annual Meeting. The Company presented new data from its Phase 1 DRAGON proof-of-concept trial. As of the August 29, 2023 data cutoff, SRK-181 data showed favorable tolerability and promising anti-tumor activity in heavily pretreated patients with clear cell renal cell carcinoma (ccRCC) resistant to anti-PD-1. Of 28 evaluable patients in the ccRCC cohort, six patients had confirmed partial responses and achieved a best tumor reduction of 33% to 93%. The objective response rate (ORR) was 21.4% and the disease control rate was 57%. Circulating granulocytic myeloid-derived suppressor cell (gMDSC) levels correlated with better clinical responsiveness in ccRCC patients treated with SRK-181 in combination with pembrolizumab. The Company believes these data from the Phase 1 DRAGON trial supports proof of concept and that the trial has met its primary objectives. The Company is completing enrollment of the trial in December 2023, while continuing to treat patients who remain on study.
Cardiometabolic Program

SRK-439 is a novel, preclinical investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin, maintenance of myostatin specificity (i.e., no GDF11 or Activin-A binding), and is initially being developed for the treatment of obesity.

Plans to initiate a Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity in 2024. As part of the Company’s strategy to advance the development of SRK-439, it plans to initiate a Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 RA in 2024, subject to IND clearance. Data from the clinical trial are expected in mid-2025 and will be used to inform further clinical development of SRK-439. The Company plans to file an IND for SRK-439 for the treatment of obesity in 2025.
Third Quarter 2023 Financial Results

For the quarter ended September 30, 2023, net loss was $42.4 million compared to a net loss of $43.3 million for the quarter ended September 30, 2022.

The Company did not record any revenue for either the quarter ended September 30, 2023 or September 30, 2022.
Research and development expense was $30.3 million for the quarter ended September 30, 2023, compared to $33.4 million for the quarter ended September 30, 2022.
General and administrative expense was $13.3 million for the quarter ended September 30, 2023, compared to $10.5 million for the quarter ended September 30, 2022.
As of September 30, 2023, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $218.6 million, which in addition to approximately $92.5 million of net proceeds from the October 2023 equity offering, is projected to fund the Company’s anticipated operating and capital expenditure requirements into the second half of 2025.
"Our upsized public offering achieved two key objectives: it enables us to bring our expertise to the cardiometabolic and obesity space with SRK-439, a highly selective myostatin inhibitor, and it extends our cash runway well past our upcoming SAPPHIRE Phase 3 data read out in Q4 2024," said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. "We are executing against our plan and we are well positioned going into 2024."

Conference Call and Webcast
The Company’s earnings conference call for the third quarter will be held at 8:00 a.m. ET on November 7, 2023. To access the conference call by phone, participants may register here to receive the dial-in number and unique PIN. A live webcast of the conference call will be available on the Investors & Media section of the Scholar Rock website at View Source An archived replay of the webcast will be available for approximately 90 days following the call.

About the Phase 3 SAPPHIRE Trial
SAPPHIRE is an ongoing randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 SMA who are receiving SMN-targeted therapy (either nusinersen or risdiplam). SAPPHIRE targeted enrolling approximately 156 patients aged 2-12 years old in the main efficacy population. These patients were randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks. An exploratory population that targeted enrolling up to 48 patients aged 13-21 years old will also separately be evaluated. These patients were randomized 2:1 to receive either apitegromab 20 mg/kg or placebo. For more information about SAPPHIRE, visit www.clinicaltrials.gov. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established.

About SRK-181 & the Phase 1 DRAGON Proof-of-Concept Trial
SRK-181 is a selective inhibitor of TGFβ1 activation being developed to overcome primary resistance to checkpoint inhibitor therapy, such as anti-PD-(L)1 antibodies, in advanced cancer. TGFβ1 is the predominant TGFβ isoform expressed in many human tumor types. Based on analyses of various human tumors that are resistant to anti-PD-(L)1 therapy, data suggest that TGFβ1 is a key contributor to the immunosuppressive tumor microenvironment, excluding and preventing entry of cytotoxic T cells into the tumor, thereby inhibiting anti-tumor immunity. 1

SRK-181 specifically targets the latent TGFβ1 isoform in a context-independent manner, designed to enable complete inhibition of TGFβ1 in all compartments within the tumor microenvironment. Scholar Rock believes that SRK-181 has the potential to overcome this immune cell exclusion and induce tumor regression when administered in combination with anti-PD-(L)1 therapy while potentially avoiding toxicities associated with non-selective TGFβ inhibition. The Phase 1 DRAGON proof-of-concept clinical trial (NCT04291079) in patients with locally advanced or metastatic solid tumors is ongoing. The trial is currently enrolling and dosing patients in multiple proof of concept cohorts conducted in parallel, including urothelial carcinoma (UC), cutaneous melanoma (MEL), non-small cell lung cancer (NSCLC), head and neck squamous cell carcinoma (HNSCC), and clear cell renal cell carcinoma (ccRCC). SRK-181 is an investigational product candidate, and its efficacy and safety have not been established. SRK-181 has not been approved for any use by the FDA or any other regulatory agency.

Rigel Reports Third Quarter 2023 Financial Results and Provides Business Update

On November 7, 2023 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported financial results for the third quarter ended September 30, 2023, including sales of TAVALISSE (fostamatinib disodium hexahydrate) tablets for the treatment of adults with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment and sales of REZLIDHIA (olutasidenib) capsules for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test (Press release, Rigel, NOV 7, 2023, View Source [SID1234637160]).

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"The third quarter of 2023 was marked by strong momentum from our commercial hematology-oncology portfolio driven by record TAVALISSE net product sales and growing REZLIDHIA awareness among leukemia treaters," said Raul Rodriguez, Rigel’s president and CEO. "These strong net sales combined with our expense discipline have allowed us to make substantial progress on our plan to reach financial breakeven."

Business Update

· In the third quarter of 2023, a total of 2,551 bottles of TAVALISSE were sold in the U.S. During the quarter, 2,412 bottles were shipped directly to patients and clinics, representing the highest number of bottles shipped to patients and clinics in a quarter since launch.

· During the third full quarter of launch, a total of 210 bottles of REZLIDHIA were sold in the U.S. During this quarter, 221 bottles were shipped directly to patients and clinics.

· Last week, Rigel announced four poster presentations highlighting data from the Company’s commercial and clinical-stage hematology-oncology portfolio at the upcoming 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. Included is a poster, Abstract #2888, reporting post hoc analyses in a subset of patients with mIDH1 R/R AML or MDS that were R/R to hematopoietic stem cell transplant (HSCT), ivosidenib (IVO), or venetoclax (VEN). The analyses suggest that olutasidenib alone or in combination with azacitidine may induce complete remissions in these patients. To learn more about Rigel’s clinical and commercial hematology-oncology portfolio visit Booth #2805 during ASH (Free ASH Whitepaper) 2023.

· Rigel continues to advance its open-label, Phase 1b clinical trial of R2891, an investigational, potent, and selective IRAK1/4 inhibitor, in patients with lower-risk myeloid dysplastic syndrome (LR-MDS) who are refractory/resistant to prior therapies. Target enrollment in the second cohort of the trial has been completed and Rigel is currently enrolling patients in the third cohort. Preliminary data results are expected by mid-year 2024.

· In September, the Data and Safety Monitoring Board (DSMB) recommended that the fostamatinib study arm of the ACTIV-4 Host Tissue (NECTAR) platform cease enrollment. No safety concerns were identified. The National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health, concurs with the DSMB’s recommendations and has asked the trial investigators to cease enrollment, complete follow-up for participants already enrolled, and complete study closeout. The full study data will be analyzed and disseminated as previously planned.

Financial Update

For the third quarter of 2023, Rigel reported a net loss of $5.7 million, or $0.03 per basic and diluted share, compared to a net loss of $19.0 million, or $0.11 per basic and diluted share, for the same period of 2022.

For the third quarter of 2023, total revenues were $28.1 million, consisting of $24.5 million in TAVALISSE net product sales, $2.7 million in REZLIDHIA net product sales, and $1.0 million in contract revenues from collaborations. TAVALISSE net product sales of $24.5 million increased by $5.3 million, or 27%, compared to $19.2 million in the same period of 2022. Contract revenues from collaborations for the third quarter of 2023 consisted primarily of royalty revenue from Grifols S.A. (Grifols) of $0.8 million.

For the third quarter of 2023, total costs and expenses were $32.6 million, compared to $40.8 million for the same period of 2022. The decrease in costs and expenses was partly due to decreased research and development costs due to the completion of activities related to the Phase 3 clinical trial of fostamatinib in wAIHA and the Phase 3 clinical trial of fostamatinib in high-risk hospitalized patients with COVID-19. Also contributing to the decrease, were lower facility-related costs and an upfront payment to Forma Therapeutics Inc. (Forma, now Novo Nordisk) recorded as in-process research and development (IPR&D) and included within cost and expenses in the third quarter of 2022. These decreases were partially offset by increased research and development costs due to the timing of activities related to the IRAK 1/4 inhibitor program.

For the nine months ended September 30, 2023, Rigel reported a net loss of $25.8 million, or $0.15 per basic and diluted share, compared to a net loss of $60.0 million, or $0.35 per basic and diluted share, for the same period of 2022.

For the nine months ended September 30, 2023, total revenues were $81.1 million, consisting of $68.1 million in TAVALISSE net product sales, $6.7 million in REZLIDHIA net product sales, $5.3 million in contract revenues from collaborations, and $1.0 million in government contract revenue. TAVALISSE net product sales of $68.1 million increased by $14.1 million, or 26%, compared to $53.9 million in the same period of 2022. Contract revenues from collaborations for the nine months ended September 30, 2023, consisted primarily of revenue from Grifols related to the delivery of drug supplies of $2.8 million and a royalty of $2.3 million. Government contract revenue for the nine months ended September 30, 2023, was related to income recognized in the second quarter of 2023 pursuant to the agreement with the U.S. Department of Defense to support Rigel’s Phase 3 clinical trial of fostamatinib in high-risk hospitalized patients with COVID-19.

For the nine months ended September 30, 2023, total costs and expenses were $103.5 million, compared to $126.6 million for the same period of 2022. The decrease in costs and expenses was partly due to decreased research and development costs due to the completion of trial activities related to the Phase 3 clinical trial of fostamatinib in wAIHA and the Phase 3 clinical trial of fostamatinib in high-risk hospitalized patients with COVID-19, as well as timing of activities related to the IRAK 1/4 inhibitor program. Also contributing to the decrease were lower facility-related costs, and an upfront payment to Forma (now Novo Nordisk) recorded as IPR&D and included within cost and expenses in the third quarter of 2022.

As of September 30, 2023, Rigel had cash, cash equivalents and short-term investments of $62.4 million, compared to $58.2 million as of December 31, 2022.

Conference Call and Webcast with Slides Today at 4:30pm Eastern Time

Rigel will hold a live conference call and webcast today at 4:30pm Eastern Time (1:30pm Pacific Time).

Participants can access the live conference call by dialing (877) 407-3088 (domestic) or (201) 389-0927 (international). The conference call will also be webcast live and can be accessed from the Investor Relations section of the company’s website at www.rigel.com. The webcast will be archived and available for replay after the call via the Rigel website.

Replimune Reports Fiscal Second Quarter 2024 Financial Results and Provides Corporate Update

On November 7, 2023 Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of a novel portfolio of oncolytic immunotherapies, reported financial results for the fiscal second quarter ended September 30, 2023 and provided a business update (Press release, Replimune, NOV 7, 2023, View Source [SID1234637159]).

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"We look forward to presenting the topline data from our registration-directed CERPASS clinical trial of RP1 in combination with Libtayo in cutaneous squamous cell carcinoma (CSCC) as well as sharing an initial snapshot from the full patient population in the IGNYTE clinical trial cohort of RP1 combined with Opdivo in anti-PD1 failed melanoma at an investor call in a few weeks time," said Philip Astley-Sparke, CEO of Replimune. "We are also excited to present the design of a confirmatory study agreed with the FDA to support a potential approval of RP1 in anti-PD1 failed melanoma via the accelerated approval pathway. For RP2 and RP3, we are continuing to see anti-tumor activity in our Phase 1 program and look forward to providing a full update in early 2024."

Program Highlights & Milestones

RP1

CERPASS clinical trial of RP1 combined with Libtayo (cemiplimab-rwlc) in CSCC
The trigger for the primary analysis from the registration-directed CERPASS clinical trial occurred in late June and data collection activities are now complete. The independent review process is expected to complete shortly, triggering a defined process timeline to disclosure in early December.
Assuming positive data demonstrating overall clinical benefit, the Company plans to submit a Biologics License Application (BLA) for RP1 in Q2 2024.
RP1 combined with Opdivo (nivolumab) in anti-PD1 failed melanoma
The Company will present initial snapshot data for all patients on its conference call in early December by which point all patients will have had at least 6 months follow up. The Company also plans to provide a regulatory update including the design of the intended confirmatory clinical trial to support a potential approval under the accelerated pathway. Planning for the confirmatory study is underway to ensure it has commenced ahead of any BLA submission. The per protocol primary analysis will take place 12 months post the last patient enrolled. Accordingly, the Company plans to submit the BLA in Q3 2024.
RP1 combined with Opdivo in anti-PD1 failed non-melanoma skin cancers (NMSC)
Recruitment remains ongoing into the cohort of patients with anti-PD1 failed NMSC, including CSCC. The Company plans to provide a data update of the first 30 patients with at least 6 months follow up on its conference call in early December.

RP1 in solid organ transplant recipients with skin cancers
Presented initial data from the ARTACUS clinical trial of RP1 monotherapy in solid organ transplant recipients with skin cancers at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 38th Annual Meeting in November 2023. The data included 23 evaluable patients with CSCC (n=20) and Merkel cell carcinoma (n=3).
The data demonstrated an overall response rate (ORR) of 34.5% and a confirmed complete response (CR) rate of 21%.
RP1 monotherapy was well tolerated, and the safety profile was similar to non-immunocompromised patients with advanced skin cancers (i.e. from the IGNYTE study). No immune-mediated adverse events or evidence of allograft rejection were observed.
RP2 and RP3

RP2 and RP3 Phase 1 program
Accrual in the Phase 1 program is now substantially complete. Any additional Phase 2 development programs not already announced which are driven by data from the full Phase 1 data and other opportunistic considerations are expected to be announced in early 2024.
The Company will present updated data from a cohort of metastatic uveal melanoma patients enrolled in the open-label, multicenter Phase 1 study of RP2 as a single agent and in combination with nivolumab during a Plenary Session at the 20th Annual International Society for Melanoma Research Congress on November 8, 2023.
RP2 and RP3 Phase 2 program
RP2 and RP3 in combination with atezolizumab and bevacizumab in third-line colorectal cancer (CRC)
Two signal finding cohorts of 30 patients each are being enrolled in collaboration with Roche. Patients in the first cohort will be treated with atezolizumab combined with bevacizumab and RP2 and the second cohort with atezolizumab and bevacizumab and RP3. This clinical trial is ongoing.
RP2 and RP3 in combination with atezolizumab and bevacizumab in second-line (2L) hepatocellular carcinoma (HCC)
Two signal finding cohorts of 15 patients each will be enrolled in collaboration with Roche. The first cohort will enroll 2L patients treated with standard of care atezolizumab combined with bevacizumab and RP3, and the second cohort will enroll 2L patients treated with atezolizumab combined with bevacizumab and RP2. This clinical trial has recently initiated.
RP3 in combination with standard of care therapy in squamous cell carcinoma of the head and neck (SCCHN)
Initiation has been delayed due to the global shortage of cisplatin and carboplatin.
Corporate Update

Announced the appointment of Emily Hill as Chief Financial Officer (CFO). Ms. Hill was most recently CFO of the commercial stage biotech company PTC Therapeutics and has more than 15 years of experience in the biotechnology and life sciences industry having held senior financial management and investor relations roles at several leading public biotechnology and pharmaceutical companies.
Due to the timing of expected data, the company will commence a quiet period on November 13, 2023 that will remain in effect until the planned conference call in early December.
Financial Highlights

Cash Position: As of September 30, 2023, cash, cash equivalents and short-term investments were $496.8 million, as compared to $583.4 million as of March 31, 2023. The decrease was primarily related to cash utilized in operating activities in advancing the Company’s expended clinical development plans.

Based on the current operating plan, the Company believes that existing cash, cash equivalents and short-term investments, as of September 30, 2023, will enable the Company to fund operations into the second half of calendar year 2025.
R&D Expenses: Research and development expenses were $49.1 million for the second quarter ended September 30, 2023, as compared to $28.8 million for the second quarter ended September 30, 2022. This increase was primarily due to increased clinical and manufacturing expenses driven by the Company’s lead programs and increased personnel expenses. Research and development expenses included $4.4 million in stock-based compensation expenses for the second quarter ended September 30, 2023.
S,G&A Expenses: Selling, general and administrative expenses were $14.7 million for the second quarter ended September 30, 2023, as compared to $12.7 million for the second quarter ended September 30, 2022. The increase was primarily driven by personnel related costs, including sales and marketing personnel associated with pre-launch planning and build of the Company’s commercial infrastructure. Selling, general and administrative expenses included $4.7 million in stock-based compensation expenses for the second quarter ended September 30, 2023.
Net Loss: Net loss was $60.0 million for the second quarter ended September 30, 2023, as compared to a net loss of $43.1 million for the second quarter ended September 30, 2022.
About CERPASS
CERPASS is Replimune’s registration-directed randomized, global Phase 2 clinical trial to compare the effects of Libtayo (cemiplimab-rwlc) alone versus a combination of Libtayo and Replimune’s investigational oncolytic immunotherapy RP1. The clinical trial enrolled 211 patients with locally advanced or metastatic cutaneous squamous cell carcinoma who are naïve to anti-PD-1 therapy. The clinical trial will evaluate complete response rate and overall response rate as its two independent primary efficacy endpoints as assessed by independent review, as well as secondary endpoints including duration of response, progression-free survival, and overall survival. The clinical trial is being conducted under a clinical trial collaboration agreement with Regeneron and full commercial rights retained by Replimune. Libtayo is a registered trademark of Regeneron.

About IGNYTE
IGNYTE is Replimune’s multi-cohort Phase 1/2 clinical trial of RP1 in combination with Opdivo (nivolumab). There are 3 tumor specific cohorts in this clinical trial including a cohort in anti-PD1 failed melanoma with registrational intent that has completed enrollment with 140 patients enrolled. This cohort was initiated after completing enrollment in a prior Phase 2 cohort in the same clinical trial of approximately 30 patients with melanoma. The additional cohorts currently enrolling and are in non-melanoma skin cancers which includes both naïve and anti-PD1 failed CSCC, and in anti-PD1 failed microsatellite instability high, or MSI-H/dMMR tumors. This trial is being conducted under a collaboration and supply agreement with Bristol-Myers Squibb. Opdivo is a registered trademark of Bristol-Myers Squibb.

About RP1
RP1 is Replimune’s lead product candidate and is based on a proprietary new strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF intended to maximize tumor killing potency, the immunogenicity of tumor cell death, and the activation of a systemic anti-tumor immune response.

About RP2 & RP3
RP2 and RP3 are derivatives of RP1 that express additional immune-activating proteins. RP2 expresses an anti-CTLA-4 antibody-like molecule and RP3 additionally expresses the immune co-stimulatory pathway activating proteins CD40L and 4-1BBL, but does not express GM-CSF. RP2 and RP3 are intended to provide targeted and potent delivery of these proteins to the sites of immune response initiation in the tumor and draining lymph nodes, with the goal of focusing systemic immune-based efficacy on tumors and limiting off-target toxicity.

Pyxis Oncology Announces Initiatives to Prioritize Lead ADC Program; Reports Financial Results for Third-Quarter 2023 and Provides Corporate Update

On November 7, 2023 Pyxis Oncology, Inc. (Nasdaq: PYXS), a clinical-stage company focused on developing antibody-drug conjugates (ADCs) and immune-oncology (IO) therapeutics to target difficult-to-treat cancers, reported initiatives to extend its cash runway, reported financial results for the quarter ended September 30, 2023, and provided a corporate update (Press release, Pyxis Oncology, NOV 7, 2023, View Source [SID1234637157]). The company ended the third quarter of 2023 with approximately $134.4 million in cash, cash equivalents, restricted cash and short-term investments.

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"We are excited to begin dosing in the fifth cohort of our Phase 1 trial of our lead asset PYX-201, a first-in-class and first-in-concept tumor stroma targeting ADC against EDB-fibronectin in tumor stroma. We believe the progress we are making through the dose escalation portion of our trial speaks to the potential safety profile of our ADC drug candidate. Moreover, based on data from Pfizer’s HER2 ADC previously under development with the same linker and payload and our preclinical experiments, we believe that at 3.6 mg/kg and above, we are approaching biologically active dose levels," said Lara S. Sullivan, M.D., President and Chief Executive Officer of Pyxis Oncology. "In order to maximize PYX-201’s chances of success, as well as those of PYX-106, we are announcing a number of initiatives to extend our cash runway into 2026, which we believe is the responsible thing to do in the current funding environment. We believe the actions we are taking best position Pyxis Oncology for future success, with our current cash resources now taking us beyond important near-term 2024 readouts for our ongoing clinical trials."

Pam Connealy, COO and CFO of Pyxis Oncology added, "In addition to the cost reductions announced today, which include a 40% decrease in headcount, we are also seeking additional non-dilutive funding through the potential monetization of our acquired Apexigen royalty streams. Additionally, we are evaluating partnerships of several assets that we have chosen not to move into the clinic and potential antibody and ADC platform technology collaborations. We believe this increased focus on our lead and secondary assets, in addition to the changes we are making to the PYX-106 program, gives us the best chance for success in bringing our novel ADC and IO candidates to the patients who need them. We appreciate the contributions from our dedicated team members that have enabled us to reach this point in our clinical programs."

Corporate Updates


PYX-201: Fifth dose cohort open for enrollment in Phase 1 trial. To date, 15 subjects have been dosed with PYX-201 in the PYX-201-101 trial. The fifth dose cohort is expected to begin enrolling shortly and is planned to evaluate a 3.6 mg/kg dose administered once every three weeks. Management believes the efficient progression through dose escalation to date speaks to the potential safety profile of PYX-201, with preliminary Phase 1 data expected in 1H 2024.

PYX-106: Phase 1 trial now focusing on NSCLC and other tumor types. Following analyses of data from a competing anti-Siglec-15 clinical trial and a review of internally generated preclinical results, Pyxis Oncology has decided to focus on enrolling additional patients with specific tumor types, including non-small cell lung cancer, in its ongoing Phase 1 trial. Currently, dosing is ongoing in the second cohort at a dose of 1.0 mg/kg. As a result of this repositioning of the trial, preliminary data from the PYX-106-101 trial is now anticipated in 2H 2024. Importantly, the repositioning of the trial has not increased the cost of the study.

Cash runway extended to early 2026 due to cost reductions and portfolio prioritization. Pyxis Oncology plans to focus its cash resources on and around its two clinical-stage programs, extending its cash runway past key Phase 1 data readouts for PYX-201 and PYX-106, expected in 2024. Following a portfolio and business review, the company is announcing a reduction in overall headcount by approximately 40% and pausing funding of certain early-stage research programs. These initiatives are anticipated to extend Pyxis Oncology’s cash runway into early 2026. In addition, the company has undertaken monetization efforts for its acquired Apexigen royalty streams and has also undertaken initiatives for possible partnerships of several assets that we have chosen not to move into the clinic, and antibody and ADC platform technologies, which may bring in additional non-dilutive funding.

Acquisition of Apexigen completed and further sotigalimab development. On August 23, 2023, Pyxis Oncology announced the successful completion of its acquisition of Apexigen, Inc., in an all-stock transaction valued at approximately $10.7 million. Opportunity to advance clinical development of sotigalimab (which Pyxis Oncology has renamed PYX-107) will be further assessed as part of portfolio evaluation following preliminary data for our ongoing Phase 1 trial of PYX-201.

Potential Upcoming Milestones


PYX-201 (ADC targeting various solid tumors): Report preliminary Phase 1 data and PK/PD results in 1H 2024.

PYX-106 (IO targeting various solid tumors): Report preliminary Phase 1 data and PK/PD results in 2H 2024.

Updates on additional non-dilutive funding efforts as appropriate.

Q3 2023 Financial Results


As of September 30, 2023, Pyxis Oncology had cash and cash equivalents, including restricted cash, and short-term investments of $134.4 million (unaudited), which is now expected to fund operations into early 2026 and reflects continued financial discipline.

Research and development expenses were $14.7 million for the three months ended September 30, 2023, compared to $19.0 million for the three months ended September 30, 2022. The period-over-period decline was primarily due to lower contract manufacturing and preclinical research costs, which were partially offset by increased clinical trial-related expenses for PYX-201 and PYX-106.

General and administrative expenses were $10.7 million for the three months ended September 30, 2023, compared to $9.4 million for the three months ended September 30, 2022. The period-over-period increase was primarily due to higher stock-based compensation expenses, which were partially offset by lower professional and consultant fees.

Net loss was $23.0 million, or $0.56 per common share, for the three months ended September 30, 2023, compared to $27.7 million, or $0.85 per common share, for the three months ended September 30, 2022. Net losses for the quarters ended September 30, 2023 and 2022 included $5.2 million and $4.4 million, respectively, related to non-cash stock-based compensation expense.

As of September 30, 2023, the outstanding number of shares of common stock of Pyxis Oncology was 44,294,092.

Propanc Biopharma Confirms PRP Enhances Chemosensitivity and Alters Tumor Microenvironment of Pancreatic Tumor Cells

On November 7, 2023 Propanc Biopharma, Inc. (OTC Pink: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that PRP enhances the sensitivity of resistant pancreatic tumor cells to standard chemotherapy treatment and alters the tumor microenvironment by decreasing the fibrotic tissue and its malignancy (ability to spread into surrounding tissues) (Press release, Propanc, NOV 7, 2023, View Source [SID1234637156]). The results were reported by the Company’s joint researcher, Mrs. Belén Toledo Cutillas, MSc, at the laboratory of Professor Macarena Perán, PhD, University of Jaén, Granada, Spain.

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Various methods, including apoptosis (programmed cell death) and viability analysis, live/dead assays, and RT-qPCR (real time quantification of gene expression) analyses confirmed PRP enhances the sensitivity of pancreatic tumor cells to standard chemotherapy treatment and decreases expression of genes related to chemoresistance. It was also proven that a major pathway, TGF-β, responsible for tumorigenesis (production or formation of tumors) and metastasis (cancer spread), is involved with the observed effects by inhibiting this pathway after PRP treatment. Similar analyses and an in vivo (in a living organism) study evaluating the effects on the tumor microenvironment confirmed transformational changes to the extracellular matrix components of the resistant tumors and according to Mrs. Cutillas, "seems to re-educate the malignant tumor cells by inducing apoptosis and reversing their malignant phenotype (cellular characteristics)."

Dr Julian Kenyon, MD, MB, ChB, Propanc’s Chief Scientific Officer said, "Results from the experiments conducted by Belen demonstrates the effects of PRP on reducing chemoresistance in pancreatic tumor cells, which is a significant issue among pancreatic adenocarcinoma (PDAC) patients, who have a poor 5-year survival rate of less than eight percent. We look forward to investigating the clinical effects in patients as we progress with future planned clinical trials, where an opportunity for a combinatorial therapeutic strategy may be uncovered to treat these resistant tumors."

PRP is a mixture of two proenzymes, trypsinogen and chymotrypsinogen from bovine pancreas, administered by intravenous injection. A synergistic ratio of 1:6 inhibits growth of most tumor cells. Examples include pancreatic, ovarian, kidney, breast, brain, prostate, colorectal, lung, liver, uterine, and skin cancers. Orphan Drug Designation status of PRP has been granted from the US Food and Drug Administration (FDA) for treatment of pancreatic cancer.