On August 30, 2023 BioLineRx, a pre-commercial stage biopharmaceutical company pursuing life-changing therapies for certain cancers and rare diseases, reported its unaudited financial results for the second quarter ended June 30, 2023, and provided corporate and portfolio updates (Press release, BioLineRx, AUG 30, 2023, View Source [SID1234634868]).

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"We had a very productive second quarter across all areas of the company, including our focused pre-launch preparation activities tied to the potential U.S. approval of motixafortide in the next few weeks, as well as the formation of a new strategic partnership, announced today, to develop and commercialize motixafortide in Asia," said Philip Serlin, Chief Executive Officer of BioLineRx. "The partnership, which is subject to certain closing conditions, provides a pathway forward to pursue potential indications for motixafortide in stem cell mobilization and pancreatic cancer in Asia, as well as a source of substantial funding to the company.

Additionally, we advanced our second major development program for motixafortide in pancreatic cancer through the initiation of a randomized Phase 2 clinical trial with Columbia University in first line metastatic pancreatic cancer based on promising data from a single-arm pilot phase.

"Finally, our clinical trial collaboration with Washington University School of Medicine in St. Louis to evaluate motixafortide as monotherapy and in combination with natalizumab for stem cell mobilization for gene therapies in sickle cell disease continues to progress, and we anticipate clinical trial initiation this year. I am extremely pleased with our progress to date and look forward to a fruitful second half of the year, including our potential transition to a commercial stage company," Mr. Serlin concluded.

Corporate Updates

On track for September 9, 2023 PDUFA target action date on NDA for motixafortide in stem cell mobilization for autologous transplantation in multiple myeloma
Signed exclusive license agreement to develop and commercialize motixafortide in Asia with concurrent equity investment; license agreement includes $15 million upfront payment, plus potential development, regulatory and sales milestones, and tiered double-digit royalties, as well as various development obligations for the licensee, including the planned initiation in China of a registrational study in stem-cell mobilization and a randomized Phase 2/3 study in first-line pancreatic cancer; straight common equity investment of $14.6 million in BioLineRx at $2.136 per ADS with no warrants; effectiveness and closing of transactions is contingent upon approval by Israeli Innovation Authority of license agreement within four months of execution, and other closing conditions
Clinical Portfolio Updates

Motixafortide (selective inhibitor of CXCR4 chemokine receptor)

Multiple Myeloma

Announced publication in Nature Medicine of GENESIS Phase 3 clinical trial data evaluating motixafortide and G-CSF in stem cell mobilization for autologous transplantation in multiple myeloma
Pancreatic Ductal Adenocarcinoma

Announced initiation of randomized, investigator-initiated Phase 2 clinical trial in collaboration with Columbia University, with joint funding of the study by Regeneron and BioLineRx, assessing motixafortide in combination with the PD-1 inhibitor cemiplimab and standard-of-care chemotherapy as first-line treatment in patients with mPDAC. Anticipate initial patient data in 2023. A poster of the amended clinical trial design was presented at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June
Sickle Cell Disease & Gene Therapy

Continued to advance plans for a clinical trial in collaboration with Washington University School of Medicine in St. Louis to evaluate motixafortide as monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell mobilization for gene therapies in sickle cell disease. Anticipate trial initiation later this year
AGI-134 (synthetic alpha-Gal glycolipid)

Solid Tumor Immunotherapy

Evaluating next development pathways for AGI-134 program in consultation with scientific advisory board. Results from Phase 1/2a first-in-human, single-agent study announced in Q4 2022. Study met primary endpoint for safety and tolerability and demonstrated immune activity across multiple biomarkers
Second Quarter 2023 Financial Results

Research and development expenses for the three months ended June 30, 2023 were $3.0 million, a decrease of $2.4 million, or 44.3%, compared to $5.4 million for the three months ended June 30, 2022. The decrease resulted primarily from lower expenses related to NDA supporting activities related to motixafortide as well as lower expenses associated with the completed AGI-134 clinical trial
Sales and marketing expenses for the three months ended June 30, 2023 were $5.6 million, an increase of $4.4 million, or 383.9% compared to $1.2 million for the three months ended June 30, 2022. The increase resulted primarily from the ramp-up of pre-launch activities related to motixafortide
General and administrative expenses for the three months ended June 30, 2023 were $1.3 million, an increase of $0.3 million, or 24.4% compared to $1.0 million for the three months ended June 30, 2022. The increase resulted primarily from an increase in payroll and related expenses due to a small increase in headcount and share-based compensation, as well as small increases in a number of G&A expenses
Net loss for the three months ended June 30, 2023 was $18.5 million, compared to $7.4 million for the three months ended June 30, 2022. The Company’s net loss for the six months ended June 30, 2023 amounted to $30.7 million, compared to $12.4 million for the six months ended June 30, 2022. The increases in net loss for both the three and six months ended June 30, 2023 were due primarily to a non-operating expense of approximately $7.8 million and $10.8 million respectively, related to the revaluation of outstanding warrants resulting from an increase in the Company’s share price over the preceding three and six months
As of June 30, 2023, the Company held cash, cash equivalents, and short-term bank deposits of $32.8 million and anticipates this will be sufficient to fund operations, as currently planned, into the first half of 2024. This amount does not include $29.6 million in total funding from the exclusive license agreement and equity investment announced today, which the Company anticipates closing in Q3 subject to formal transaction approval by the Israeli Innovation Authority and other closing conditions

On August 30, 2023 BioInvent reported its interim report for January-June 2023 (Presentation, BioInvent, AUG 30, 2023, https://www.bioinvent.com/sites/bioinvent/files/pr/20230830-cb906e7c-ce8a-4f6e-8240-2cd14a254fc7-1.pdf?ts=1693375214 [SID1234634867]).

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On August 30, 2023 Serina Therapeutics, Inc. ("Serina"), a privately-held, clinical-stage biotechnology company developing a pipeline of therapies for the treatment of Parkinson’s Disease and other neurological diseases, entered into a merger agreement with AgeX Therapeutics, Inc. (NYSE American: AGE) ("AgeX") on August 29, 2023, under which Serina will merge with a wholly-owned subsidiary of AgeX in an all-stock transaction (Press release, Serina Therapeutics, AUG 30, 2023, View Source [SID1234634788]). The combined company will continue under the Serina Therapeutics name and will focus on advancing Serina’s pipeline of small molecule drug candidates targeting central nervous system ("CNS") indications, enabled by the company’s proprietary POZ Platform delivery technology. In addition to advancing the company’s wholly-owned pipeline assets, Serina is working with companies in the pharmaceutical industry currently advancing pre-clinical studies exploring POZ polymer lipid-nanoparticles ("LNPs") in next generation RNA vaccines.

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"The merger with AgeX positions Serina to advance our CNS pipeline assets and expand our platform partnering opportunities," said Milton Harris, PhD, Co-Founder and Chair of the Board of Serina. "We believe it represents the best path forward for Serina in accessing transformative capital to advance our platform technology. As a board director of the combined company, I look forward to collaborating with our new partners AgeX and Juvenescence, as we continue the work of translating our science into innovative therapeutics."

"We are delighted to announce the proposed merger with Serina," said Joanne M. Hackett, PhD, Chairperson and Interim Chief Executive Officer of AgeX. "The AgeX team thoroughly reviewed and evaluated numerous strategic alternatives for creating stockholder value, and we believe this transaction with Serina presented the most compelling option for our stockholders. We see exciting potential to generate novel drug candidates with the POZ Platform delivery technology."

"Serina merging with AgeX is an important step towards recognizing the potential to develop the POZ Platform to deliver novel medicines and treatment modalities," said Richard Marshall, Chief Executive Officer of Juvenescence Limited ("Juvenescence"). "We plan to leverage our deep pharmaceutical expertise and network to assist the combined company to reach its goal in maximizing value for stockholders."

The combined company will focus on advancing Serina’s lead drug candidate (SER-252, POZ-apomorphine) for the treatment of advanced Parkinson’s Disease through pre-clinical studies, with the goal of submitting an investigational new drug submission ("IND") to the Food and Drug Administration for the initiation of a Phase I clinical trial during the fourth quarter of 2024. Serina has two other pipeline assets that are positioned to enter IND enabling studies, SER-227 (POZ-buprenorphine) for certain post-operative pain indications and SER-228 (POZ-cannabidiol) for refractory epilepsy indications. Additionally, the combined company will focus on expanding Serina’s LNP and antibody drug conjugate ("ADC") partnering collaborations.

About the Transaction, Management and Organization

Under the terms of the merger agreement, pending stockholder approval of the transaction, Serina will merge with a wholly-owned subsidiary of AgeX, and stockholders of Serina will receive shares of AgeX common stock ("merger"). AgeX following the merger is referred to herein as the "combined company." The merger has been approved by the boards of directors of both companies and is expected to close in the first quarter of 2024, subject to customary closing conditions.

Upon completion of the merger, pre-merger AgeX stockholders are expected to own approximately 25% of the newly combined company while pre-merger Serina stockholders are expected to own approximately 75% of the newly combined company. The final percentage of the combined company owned by pre-merger Serina stockholders and pre-merger AgeX stockholders upon completion of the merger may be subject to certain adjustments and assumptions. As part of the merger, pre-merger AgeX stockholders will be issued Post-Merger Warrants. The Post-Merger Warrants issued to AgeX stockholder, Juvenescence, have a cash exercise requirement that will provide an additional $15 million in capital to the combined company in three equal tranches over the term of the warrant, which expires July 31, 2025. AgeX stockholders that exercise the Post-Merger Warrants will additionally receive Incentive Warrants that expire four (4) years after the merger closing date. The terms and conditions for each type of warrant will be further detailed in the forms of warrant agreements that will be negotiated between the parties prior to the merger closing date.

Prior to the execution of the merger agreement, AgeX invested $10 million in Serina through the purchase of a Senior Convertible Loan Note ("CLN") described on the Current Report on Form 8-K that AgeX filed with the U.S. Securities and Exchange Commission ("SEC") on March 15, 2023. Immediately prior to completion of the merger, the CLN will be converted into Serina capital stock as a capital contribution. It is expected that the funds provided by the CLN, together with the additional $15 million of proceeds from the Juvenescence required warrant cash exercises, will provide working capital for the combined company to help fund operations into calendar year 2026.

Following the merger, it is anticipated that the combined company will be led by a new Chief Executive Officer ("CEO"). Current members of the executive team of Serina are expected to continue in key leadership roles, including Dr. Randall Moreadith as the Chief Science Officer, and Dr. Tacey Viegas as Chief Operating Officer and Secretary. Serina’s current Chief Financial Officer ("CFO") Steve Ledger is expected to serve as the interim CEO of the combined company until such time as the new CEO is hired. AgeX’s current CFO, Andrea Park, is expected to serve as the interim CFO and Chief Accounting Officer of the combined company until such time as the new CFO is hired, and is then expected to continue in the role of Chief Accounting Officer. The board of directors will be comprised of seven directors and are expected to include AgeX director Dr. Gregory Bailey, Juvenescence CEO Dr. Richard Marshall, Serina’s directors Dr. J. Milton Harris and Steve Ledger, and the Buck Institute for Aging’s Vice President of Business and Technology Advancement Remy Gross III. Two additional directors will be appointed in accordance with the merger agreement to fill the remaining seats on the board of directors.

Upon completion of the transaction, the combined company will operate under the Serina Therapeutics name, and the combined company’s common stock is expected to trade on the NYSE American under the ticker symbol "SER." The corporate headquarters will be in Huntsville, Alabama.

Gibson, Dunn & Crutcher LLP is providing legal counsel to AgeX. Bradley Arant Boult Cummings LLP is legal counsel to Serina.

On August 30, 2023 Diverse Biotech reported that it has been granted USPTO patent approval of its novel CUSP synthesis platform creating an entirely new class of drugs that are part-antibody and part-cannabinoid in one new molecule (Press release, Diverse Biotech, AUG 30, 2023, View Source [SID1234634787]). Starting with DBT-50201, its novel PEMBROLIZUMAB compound, Diverse expects to develop multiple treatments for many diseases with highly unmet needs. According to John Patrick, M.D., Executive Vice President and Chief Medical Officer for Diverse Biotech, "We are very excited about being granted our first patent for our monoclonal antibody class of drugs with our flagship drug DBT-50201 which represents a significant advancement for PEMBROLIZUMAB. We expect that our monoclonal drug DBT-50201 will mirror the results we have seen thus far in trials with our Temozolomide drug used to treat Glioblastomas (brain cancer), DBT-30180. We have observed significant increases in potency and efficacy with our CUSP technology averaging about 2,000% when compared to standard chemotherapy alone. Coupled with other potential decreases in toxicity, these results represent a major step forward in the treatment and cure of multiple cancers."

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On August 30, 2023 Sirnaomics reported positive Clinical Readouts Validate Potential of Proprietary Delivery Platforms and Cement Global Leadership in RNAi Therapeutics (Press release, Sirnaomics, AUG 30, 2023, View Source [SID1234634786]).

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Business Highlights

Promising progress in clinical development

After an End of Phase-II meeting and with guidance from the U.S. FDA, the Group is advancing the late-stage clinical development of STP705 for the treatment of isSCC, a significant milestone that has cemented the Group’s global leadership in RNAi therapeutics for cancer treatment.
Based on promising interim STP707 data, the Group is actively exploring collaborations for a Phase II combination trial to investigate the potential of combining STP707 with novel approved cancer therapies.
The Group’s first GalAhead product STP122G has received regulatory clearance from the U.S. FDA and commenced the Phase I clinical trial.
The Group’s subsidiary RNAimmune, Inc. has received regulatory clearance from the U.S. FDA to initiate Phase I clinical trial for RV-1730, a SARS-CoV-2 vaccine booster candidate.
Production capacity enhancement

Having produced a full GMP batch of STP707 for human injection in the first quarter of 2023, the Guangzhou Facility is expected to achieve full GMP-compliant manufacturing of Sirnaomics’s pipeline products. The Group also started expanding capacity of the filling line to include liquid dose fill in 2R vial to support the GalAhead platform.
Recognition by HKSTP

Sirnaomics received grants of HKD8 million from the HKSTP Clinical Translational Catalyst Programme, and HKD2 million from the HKSTP MedTech Co-create Programme.
HONG KONG, GERMANTOWN, Md., SUZHOU, China, Aug. 30, 2023 /PRNewswire/ — Sirnaomics Ltd. (the "Company"; together with its subsidiaries, "Sirnaomics" or the "Group"; stock code: 2257), a leading biopharmaceutical company in discovery and development of RNAi therapeutics, has announced its interim results for the six months ended 30 June 2023 (the "Period").

In the first half of 2023, Sirnaomics made significant progress in pipeline and business development. To ensure it had a sufficient cash runway amid global economic uncertainties, the Group focused resources on high-potential programs and optimized its internal structure, solidifying its international team specializing in RNAi therapeutics, with a strong focus on the U.S. and Asia markets. With extensive expertise in RNA therapeutics and innovative delivery platform technologies, the Group effectively implemented its clinical development strategy, which paved the way for it to start clinical trials in the U.S. for its leading clinical drug candidates, namely STP705, STP707 and STP122G.

The Group’s loss for the Period narrowed to US$ 41.1 million from US$46.1 million for the six months ended 30 Jun 2023. The lesser loss was primarily attributable to decrease in research and development expenses; and decrease in loss on changes in fair value of financial liabilities at FVTPL. As at 30 June 2023, the Group was in a healthy financial position with cash and cash equivalents and current time bank deposits amounting to US$77.3 million.

Clinical trials of leading product candidates have yielded promising results, giving impetus to the commercialization process

Sirnaomics has been advancing a prioritized drug product pipeline of innovative RNA-based medicines to improve the lives and well-being of patients worldwide. The following milestones and achievements exemplify the Group’s ongoing clinical execution across its pipeline.

STP705

Sirnaomics has used human data from STP705 to expand its clinical trials. With the data from STP705 proving its effectiveness in treating squamous cell carcinoma in situ (isSCC), the Group moved on to explore its use in a broader range of oncology indications including basal cell carcinoma (BCC), liver cancer, and medical aesthetics. Following the discussions with the U.S. FDA in early 2023, the Group was ready to proceed with a confirmatory clinical study for the treatment of isSCC, based on positive Phase IIa and IIb results. It is preparing to move forward to conduct a single dosage study as a sub-group of subjects in a larger Phase III clinical trial. Moreover, the final data of the Phase II study of STP705 for the treatment of BCC is expected to be available in the latter half of 2023, and the Group aims to take its STP705 skin cancer franchise into late-stage development by the end of the year. To support these programs and advance clinical development, Sirnaomics’s clinical teams in the U.S. and Asia are conducting multi-center global trials. By including subject populations from different regions, they aim to gather comprehensive and diverse data for various indications, including isSCC and liver cancer.

In addition, in June 2023, the Group announced interim data of its proof-of-concept Phase I STP705 trial, studying fat remodeling in abdominoplasty patients. All tissue samples examined in the review using variable doses of STP705 showed histological evidence suggestive of fat remodeling. The Group plans to use the information from the study to expand into the treatment of submental fat and other areas of noninvasive fat remodeling. The development program is expected to open up a new therapeutic area in medical aesthetics for the Group’s pipeline and the market has thus far responded positively.

STP707

The Group is actively advancing clinical trials for STP707 and expanding its therapeutic reach through systemic administration, pushing to open more opportunities to treat indications that could not be addressed by STP705. STP707 stands as proof of the safety and efficacy of the Group’s proprietary PNP delivery systems in IV administration. Encouraged by the promising interim data, the Group plans to explore collaborations for a Phase II combination trial, combining STP707 with novel approved cancer therapies such as immune check point inhibitors as well as traditional chemotherapy. Such potential combination therapies may include CCA, HCC, melanoma, and pancreatic cancer. The Group will also continue to explore other indications for Phase II trials and expand its clinical development programs. Given the significant market potential of STP707 through IV administration and partnership possibility, the Group believes allocating capital and corporate resources into advancing those valuable assets is the optimal growth strategy.

Additionally, Sirnaomics has announced the Group completed all dosing regimens for its Phase I study of STP707 for the treatment of multiple solid tumors in patients with various types of late-stage cancers who did not respond to multiple rounds of other oncology treatments. Approximately 74% of evaluable patients demonstrated a best response of stable disease (SD) and patients exhibited reduction in tumor burden per Response Evaluation Criteria in Solid Tumors.

Accelerating advancing additional ground-breaking first-in-class preclinical assets into clinical stage

During the Period, the Group advanced STP122G, the first representative candidate for the GalAhead delivery platform, into clinical stage, marking the first time Sirnaomics use its proprietary GalNAc RNAi platform technology, GalAhead, in one of its siRNA-based candidates and kicking off a trial for addressing the unmet need of a large patient population suffering from anticoagulation disorders. The Phase I clinical study commenced in April, with the first participant receiving a dose for anticoagulant treatment in June. The study aims to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of STP122G.

The Group plans to accelerate research and development of the next-generation GalAhead platform. Its robust pipeline comprises an array of nine GalAhead preclinical candidates. Subsequent to approval gained for STP122G, the Group expects to file IND in the U.S. for STP125G and STP144G, the good line up of assets from the GalAhead delivery platform, in the fourth quarter of 2023 and the first half of 2024.

Laying the foundation for future development with the Fill and Finish (F&F) Plant Facility in Guangzhou set up

After a successful first full year of operation, the Group’s Guangzhou F&F Facility has continued to optimize its clinical supplies strategy in Asia. The facility has continuously improved in GMP compliance and operational assurance for aseptic processing. Having produced recently a full GMP batch of STP707 for human injection, the facility is expected to be in full GMP-compliant manufacturing of the Group’s pipeline products, including formulation, fill and finish for both liquid and solid dose production, as well as testing and release. Expected to put out annually around 50,000 vials of lyophilized solid dose and 150,000 to 200,000 vials of liquid dose for human injectables, the facility will be able to support all clinical trials currently planned and future clinical development of the Group. In the first half of 2023, the Guangzhou Facility primarily backed the Group’s efforts with STP707 and initiated expanding filling line capacity for the GalAhead platform. Further expansions are planned to support the Group’s growing GalAhead product line.

Strategically reinforcing business presence in Hong Kong, aspiring to turn the city into a leading RNA medicine harbor in Asia

After Sirnaomics became listed in Hong Kong at end of 2021, it made a strategic decision to establish a meaningful R&D presence in the city. Today, Sirnaomics, its subsidiaries RNAimmune and EDIRNA have all set up Hong Kong offices and been admitted into the Hong Kong Science and Technology Park (HKSTP). Moreover, Sirnaomics received grants of HKD8 million from the HKSTP Clinical Translational Catalyst Program and HKD2 million from the HKSTP MedTech Co-create Program. The Group’s objectives are to pursue collaborations in RNA medicine-related research and development and clinical studies, as well as to establish a commercial-scale manufacturing plan in the Lok Ma Chau Loop.

Dr. Patrick Lu, Founder, Chairman of the Board, Executive Director, President and Chief Executive Officer of Sirnaomics said, "Capitalizing on our dual proprietary technology delivery platforms, PNP and GalAhead, leading clinical programs, and well-established R&D and manufacturing facilities, we have advanced drug candidates through various stages of development and achieved positive results. In addition, our Guangzhou Facility has operated smoothly and effectively, not only speaking well to our manufacturing capabilities, but also to our smooth transition from a biotech company into a biopharma corporation. We are also delighted to have secured funding from HKSTP, representing its endorsement of the unwavering commitment of our team to innovation and our mission of enhancing patients’ lives. Looking ahead, we aim to reinforce our competitive advantages, pursue global and local partnerships, and accelerate development of preclinical and clinical assets, so as to enlarge market coverage and ultimately improve the well-being and quality of life of patients worldwide."