On May 4, 2021 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported its financial results for the first quarter 2021 and reaffirmed its financial guidance for 2021 (Press release, Ultragenyx Pharmaceutical, MAY 4, 2021, View Source [SID1234579114]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to have strong Crysvita and Dojolvi launches and execute on our development priorities across our pipeline," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In 2021, we will have ongoing pivotal trials for three gene therapies, one pivotal monoclonal antibody study for osteogenesis imperfecta, our first mRNA program for GSDIII, and an antisense oligonucleotide for Angelman with our partner GeneTx. These six clinical programs along with our commercial and early-stage programs make ours one of the most diversified pipelines in rare disease."

First Quarter 2021 Financials

In the first quarter 2021, Crysvita revenue in Ultragenyx territories1 increased 46% versus the first quarter 2020. This increase is driven by demand from pediatric and adult patients with X-linked hypophosphatemia (XLH) and patients with tumor induced osteomalacia (TIO), which became Crysvita’s second approved indication in the United States in June 2020.

Dojolvi revenue in the first quarter 2021 continued to build on the launch momentum after the U.S. Food and Drug Administration (FDA) approval in June 2020. As of the end of the first quarter 2021 the company has received approximately 230 completed start forms from approximately 115 unique prescribers. This has led to approximately 180 patients on reimbursed therapy as of the end of March 2021.

First quarter 2021 revenue included $42.8 million related to the technology transfer as part of the Daiichi Sankyo strategic manufacturing partnership around the HeLa PCL and HEK293 technologies. This revenue is expected to taper significantly through the end of this year as these activities come to a close.

Total operating expenses of $206.0 million in the first quarter 2021 increased 31% or $49.0 million versus the first quarter 2020, primarily driven by a $50.0 million expense for the upfront payment on the closing of the license and collaboration agreement with Mereo Biopharma. Excluding this upfront payment, total operating expenses for the year are expected to increase modestly as the company prepares for and enrolls six clinical programs, including four registrational studies.

Net cash used in operations for the quarter ended March 31, 2021 was $159.3 million, compared to net cash used of $95.2 million for the same period in 2020. Cash, cash equivalents, and marketable debt securities were $1.0 billion as of March 31, 2021.

2021 Financial Guidance

Crysvita Guidance in Ultragenyx Territories
The company reaffirms the 2021 guidance range for Crysvita that was provided at the beginning of the year. This range is $180 million to $190 million and includes the North American profit share region and the other regions where product sales are recognized.

Program Updates and Upcoming Milestones

Dojolvi for the treatment of Long-chain Fatty Acid Oxidation Disorders, or LC-FAOD

Health Canada approved Dojolvi for the treatment of adult and pediatric patients with LC-FAOD in February 2021 and the therapy was made commercially available to patients in Canada beginning in April 2021.
Recently published data2 under France’s nominative Authorization for Temporary Use program , or Authorisations Temporaires d’Utilisation (ATU), of 18 pediatric and adult patients with LC-FAOD showed that Dojolvi (triheptanoin) led to reductions in LC-FAOD manifestations and was well-tolerated, with median follow-up duration of 22 months (range 9-228 months). When comparing the year prior to treatment to the first year receiving Dojolvi, annual emergency hospital care visits decreased from a mean of 1.12 to 0.17, or an 85% reduction, and the mean number of emergency home care events decreased from 16.82 to 2.83, an 83% reduction. Similarly, the cumulative annual number of days of emergency home care were reduced from 286 in the year prior to receiving Dojolvi to 51 in the first year receiving Dojolvi, an 82% reduction. Further improvements in the cumulative annual number of days of emergency home care were seen during the second year receiving Dojolvi.
The efficacy and safety profile reported under the ATU is consistent with the results from prior reported data from the company-sponsored studies.
GTX-102 for the treatment of Angelman Syndrome, partnered with GeneTx

A pre-application meeting with a national regulatory agency in Europe was held in which the company reviewed the available efficacy and safety data as well as its proposal for dosing and administration of GTX-102. Based on our discussion, the authority agreed in principle on the expansion of the trial to Europe using the proposed modified study design, dosing and administration strategy, pending review of the application. The application to enroll the clinical study in this region was recently submitted.
In Canada, an amendment to the open clinical trial agreement was submitted and included the additional data and updated protocol and is pending review.
Discussions with the FDA are continuing and a request for a meeting has been granted and will take place in the second quarter 2021. The meeting will review additional clinical data requested by the FDA regarding the previously treated patients that confirm that the serious adverse event in the previously treated patients has fully reversed as well as an amended dosing plan for the previously treated patients.
The Phase 1/2 study is expected to resume in 2021, with clinical data on some patients still available before the end of 2021.
DTX401 for the treatment of Glycogen Storage Disease Type Ia, or GSDIa

Ultragenyx completed the Scientific Advice process with the European Medicines Agency (EMA) and an End-of-Phase 2 meeting with the FDA.
The Phase 3 study design has been submitted and endpoints are being finalized with regulators.
The Phase 3 study is expected to initiate early in the second half of 2021.
DTX301 for the treatment of Ornithine Transcarbamylase, or OTC, Deficiency

Late in the first quarter 2021, Ultragenyx met with the FDA in an End-of-Phase 2 meeting where the Phase 3 design and endpoints were finalized. The Phase 3 study will include a 64-week primary efficacy analysis period and enroll approximately 50 patients 12 years of age and older, randomized 1:1 to DTX301 (1.7 x 10^13 GC/kg dose) or placebo. The co-primary endpoints are the change in 24-hour plasma ammonia levels and the percentage of patients who achieve a response as measured by discontinuation or reduction in baseline disease management.
The final Phase 3 study design incorporates Scientific Advice feedback from the EMA.
The Phase 3 study is expected to initiate in the second half of 2021.
UX701 for the treatment of Wilson Disease

During the first quarter 2021, the Investigational New Drug (IND) application cleared FDA review. A seamless, single-protocol Phase 1/2/3 study is expected to begin early in the second half of 2021.
UX053 for the treatment of Glycogen Storage Disease Type III, or GSDIII

The IND application for UX053 for the treatment of GSDIII, the company’s most advanced mRNA program, was cleared by the FDA in March 2021. Enrollment in a Phase 1/2 study is expected to begin in the second half of 2021.
1: Ultragenyx territories include the collaboration revenue from the North American profit share territory and other regions where revenue from product sales are recognized by Ultragenyx. This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.

2: View Source

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Tuesday, May 4, 2021, at 2 p.m. PT/ 5 p.m. ET to discuss the first quarter 2021 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 6883837. The replay of the call will be available for one year.

On May 4, 2021 DURECT Corporation (Nasdaq: DRRX) reported financial results for the three months ended March 31, 2021 and provided a corporate update (Press release, DURECT, MAY 4, 2021, https://www.prnewswire.com/news-releases/durect-corporation-announces-first-quarter-2021-financial-results-and-update-of-programs-301283764.html [SID1234579131]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Q1 2021 Accomplishments:

Initiated patient dosing in the Phase 2b AHFIRM clinical study of DUR-928 in severe Alcohol-associated Hepatitis (AH)
FDA approval of POSIMIR in adults to produce post-surgical analgesia for up to 72 hours following Arthroscopic Subacromial Decompression
Publication of DUR-928’s mechanism of action
Appointment of two highly experienced biopharmaceutical board members
Further strengthened our financial position by raising $47.8 million in equity and ending Q1 with $97.2 million in cash and investments
"We are pleased with progress made in opening clinical trial sites in the U.S. and early enrollment in the AHFIRM trial, our Phase 2b clinical trial designed to evaluate the potential life-saving capability of DUR-928 in patients with severe Alcohol-associated Hepatitis," stated James E. Brown, D.V.M, President and CEO of DURECT. "We are also in discussions with multiple potential POSIMIR partners with a goal of enabling the partner to launch the product this year."

Financial highlights for Q1 2021:

Total revenues were $2.2 million and net loss was $10.1 million for the three months ended March 31, 2021 as compared to total revenues of $1.6 million and net loss of $9.9 million for the three months ended March 31, 2020.
At March 31, 2021, cash and investments were $97.2 million, compared to cash, cash held in escrow and investments of $56.9 million at December 31, 2020. Debt at March 31, 2021 was $21.0 million, compared to $20.8 million at December 31, 2020.
Update on Selected Programs:

Epigenetic Regulator Program. DUR-928, the lead product candidate in the Company’s Epigenetic Regulator Program, is an endogenous, orally bioavailable, first-in-class small molecule, which may have broad applicability in acute organ injuries such as alcohol-associated hepatitis (AH) as well as in chronic liver diseases such as non-alcoholic steatohepatitis (NASH).

Clinical Development

Alcohol-associated Hepatitis (AH)

Enrollment is ongoing in our Phase 2b study in subjects with severe acute AH to evaluate saFety and effIcacy of DUR-928 treatMent (AHFIRM). AHFIRM is a randomized, double-blind, placebo-controlled, international, multi-center Phase 2b study to evaluate the safety and efficacy of DUR-928 in approximately 300 patients with severe AH. The study is comprised of three arms targeting approximately 100 patients each: (1) Placebo plus standard of care (SOC, which may include the use of methylprednisolone, a corticosteroid, at the discretion of the treating physician); (2) DUR-928 (30 mg); and (3) DUR-928 (90 mg). All patients in the trial receive supportive care. Patients receive an intravenous (IV) dose of DUR-928 or placebo (sterile water) on day 1 and a second IV dose on day 4 if they are still hospitalized. The primary outcome measure is 90-day survival rate for patients treated with DUR-928 compared to those treated with placebo plus SOC. Secondary endpoints include 28-day survival, the rate of adverse events, Lille and MELD (prognostic scores) and time in the intensive care unit. The Company is targeting approximately 50-60 clinical trial sites in the U.S., U.K., Europe and Australia.
Reflecting the life-threatening nature of AH and the lack of therapeutic options for this devastating condition, the FDA granted DUR-928 Fast Track Designation for the treatment of AH in December 2020. The FDA grants Fast Track Designation to facilitate development and expedite the review of therapies with the potential to treat a serious condition where there is an unmet medical need. A therapeutic that receives Fast Track Designation may benefit from early and frequent communication with the agency in addition to a rolling submission of the marketing application, with the objective of getting important new therapies to patients more quickly.
Given the high mortality rate in severe AH patients and the absence of an approved therapeutic, we believe demonstration of a robust survival benefit in the AHFIRM trial may support an NDA filing.
In March 2021, a peer-reviewed research paper describing the binding sites and proposed mechanism of action of DUR-928 was published in The Journal of Lipid Research. The publication shows that DUR-928 (referred to in the paper as 25HC3S) binds to and inhibits the activity of DNA methyltransferases (DNMTs) DNMT-1, 3a and 3b, epigenetic regulating enzymes that add methyl groups to DNA (a process called DNA methylation). As such, by inhibiting DNMT activity, DUR-928 inhibits DNA methylation, thereby regulating the expression of genes that modulate crucial cellular activities, including those associated with cell death, stress response, and lipid biosynthesis. These modulations may lead to improved cell survival, and reduced lipid accumulation and inflammation, as has been observed in various in vivo animal models and in results from DURECT’s completed clinical trials in alcohol-associated hepatitis (AH) and non-alcoholic steatohepatitis (NASH).
During 2019, we completed a Phase 2a clinical trial of DUR-928 in patients with AH. All 19 patients treated with DUR-928 survived the 28-day follow-up period, 74% of patients (14/19) were discharged in ≤ 4 days after receiving a single dose of DUR-928, and there were no drug-related serious adverse events.
Alcohol-associated hepatitis (also called alcoholic hepatitis or AH) is an acute form of alcoholic liver disease (ALD) associated with long-term heavy intake of alcohol, and often occurs after a recent period of increased alcohol consumption. AH is typically characterized by a recent onset of jaundice and hepatic failure. According to the most recent data provided by the Agency for Healthcare Research and Quality (AHRQ), a part of the US Department of Health and Human Services (HHS), there were approximately 132,000 hospitalizations for patients with AH in 2018. From a 2018 publication analyzing the mortality and costs associated with AH, the cost per patient is estimated at over $50,000 in the first year. ALD is one of the leading causes of liver transplants in the U.S., costing over $875,000 per patient. An analysis of 77 studies published between 1971 and 2016, which included data from a total of 8,184 patients, showed the overall mortality from AH was 26% at 28 days and 29% at 90 days after admission.
Non-Alcoholic Steatohepatitis (NASH)

In May 2020, we reported positive topline results from a Phase 1b randomized and open-label clinical study conducted in the U.S. to evaluate safety, pharmacokinetics and signals of biological activity (including clinical chemistry and biomarkers as well as liver fat content and liver stiffness by imaging) of DUR-928 in NASH patients with stage 1-3 fibrosis. In this 65-patient study, reductions from baseline (pre-treatment) levels were seen in liver enzymes, liver stiffness as measured by imaging, serum lipids and biomarkers. Many of these reductions were statistically significant and DUR-928 was well tolerated at all three doses evaluated.
We will present additional data from this trial at an upcoming medical conference and we are working with a number of disease experts to determine next steps for DUR-928 in NASH.
Non-alcoholic fatty liver disease (NAFLD) is the most common form of chronic liver disease in both children and adults. It is estimated that NAFLD affects approximately 30% to 40% of adults and 10% of children in the United States. NASH, a more severe and progressive form of NAFLD, is one of the most common chronic liver diseases worldwide, with an estimated prevalence of 3-5% globally. No drug is currently approved for NAFLD or NASH.
POSIMIR (bupivacaine solution) Post-Operative Pain Relief Depot. POSIMIR is DURECT’s post-operative pain relief depot that uses the Company’s patented SABER technology that delivers bupivacaine to provide up to 3 days of post-surgical analgesia.

In February 2021, POSIMIR was granted U.S. FDA approval in adults for administration into the subacromial space under direct arthroscopic visualization to produce post-surgical analgesia for up to 72 hours following arthroscopic subacromial decompression. POSIMIR is the only approved sustained-release bupivacaine product indicated for up to 72 hours of post-surgical analgesia from a single application.
The approval was based on positive data from a randomized, multicenter, assessor-blinded, placebo–controlled clinical trial in patients undergoing arthroscopic subacromial decompression surgery with an intact rotator cuff. The primary outcome measures were mean pain intensity and total opioid rescue analgesia administered, both evaluated over the first 72 hours after surgery versus placebo. POSIMIR demonstrated a statistically significant improvement in both primary outcome measures: a 1.3 point, or 20%, reduction in mean pain intensity on a 0-10 point pain scale (p=0.01), and a 67% reduction in I.V. morphine-equivalent rescue opioid use, from a median of 12 mg in the placebo group to 4 mg in the POSIMIR group (p=0.01). In connection with this approval, the Company or its licensee, will be required to conduct two postmarketing non-clinical studies. Full Prescribing Information, including the Boxed Warning, is available at www.POSIMIR.com.
DURECT is in discussions with potential licensees of commercialization rights to POSIMIR, for which DURECT currently holds worldwide rights. Our goal is to put a commercial license in place in time for the licensee to launch the product in Q4 2021.
Subacromial decompression is a type of shoulder surgery used to treat impingement syndrome, a common repetitive-use injury that causes pain when the arm is raised over the head. The procedure is typically performed arthroscopically, meaning that several small incisions are made in the skin and muscle of the shoulder through which a camera lens (arthroscope) and surgical instruments are inserted during surgery. Arthroscopic subacromial decompression is generally considered outpatient surgery, and most patients go home within a few hours of surgery. The recovery period may extend from weeks to months, but the most intense pain typically occurs during the first 3 days after surgery and is often managed with oral opioids. There are over 600,000 surgeries involving arthroscopic subacromial decompression performed each year in the U.S.
Conference Call

We will host a conference call today at 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time to discuss first quarter 2021 results and provide a corporate update:

The conference call will also be available by webcast on DURECT’s homepage at www.durect.com under the "Investors" tab. If you are unable to participate during the webcast, the call will be archived on DURECT’s website under "Event Calendar" in the "Investors" section.

On May 4, 2021 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported that the European Commission (EC) has approved an additional indication for the oral once-daily therapy XTANDI (enzalutamide) for adult men with metastatic hormone-sensitive prostate cancer (mHSPC, also known as metastatic castration-sensitive prostate cancer or mCSPC) (Press release, Astellas, MAY 4, 2021, View Source [SID1234579030]). Men diagnosed with mHSPC tend to have a poor prognosis, with a median survival of approximately 3-4 years,1 underscoring the need for new treatment options.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

With this indication, enzalutamide is now the only oral treatment approved by the EC to treat three distinct types of advanced prostate cancer — non-metastatic and metastatic castration-resistant prostate cancer (CRPC) and mHSPC.2 The EC approval is based on results from the pivotal Phase 3 ARCHES trial which evaluated enzalutamide in men with mHSPC.3

"Metastatic hormone-sensitive prostate cancer patients have limited options and, unfortunately, there is a poor prognosis for many men," said Andrew Armstrong, M.D., Professor of Medicine, Surgery, Pharmacology and Cancer Biology, Director of Research in the Duke Cancer Institute’s Center for Prostate and Urologic Cancers and lead investigator of ARCHES. "The research supporting this approval provides clinical evidence showing how enzalutamide can help improve outcomes for men with mHSPC, which gives healthcare professionals in Europe the option to offer the treatment across the advanced prostate cancer disease continuum."

Data from the ARCHES trial showed enzalutamide plus androgen deprivation therapy (ADT) significantly reduced the risk of radiographic progression or death by 61% versus placebo plus ADT in men with mHSPC (n=1,150; hazard ratio [HR]=0.39 [95% confidence interval (CI): 0.30-0.50]; P<0.0001).3

"Enzalutamide has been an established standard of care for men with advanced prostate cancer and has been prescribed to more than 610,000 patients worldwide since it was first approved in 2012," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Global Therapeutic Area Head, Oncology Development, Astellas. "This new indication for enzalutamide provides men with mHSPC a much-needed, additional therapy option earlier in their treatment journey. We look forward to working with health authorities across Europe to ensure men with mHSPC have access to enzalutamide as soon as possible."

The safety analyses of the ARCHES trial appear consistent with the safety profile of enzalutamide in previous clinical trials in CRPC. In ARCHES, Grade 3 or greater adverse events (AEs) (defined as severe/disabling or life-threatening) were similar for patients receiving both enzalutamide plus ADT and those who received placebo plus ADT (24.3% vs. 25.6%).3

The EC marketing authorization for enzalutamide in men with mHSPC is applicable to European Union (EU) member countries, and is also valid in Iceland, Norway and Liechtenstein.4

This approval will have no impact on the financial forecasts of the current fiscal year ending March 31, 2022.

About metastatic Hormone-Sensitive Prostate Cancer (mHSPC)
In men with prostate cancer, the disease is considered metastatic once the cancer has spread outside of the prostate gland to other parts of the body.5 Men are considered hormone- (or castration-) sensitive if their disease still responds to medical or surgical treatment to lower testosterone levels.6 mHSPC has a median survival of approximately 3-4 years for men starting treatment with ADT.1

About XTANDI (enzalutamide)
Enzalutamide is an androgen receptor signaling inhibitor indicated in the EU for the treatment of adult men with:2

Metastatic hormone-sensitive prostate cancer (mHSPC, also known as metastatic castration-sensitive prostate cancer or mCSPC) in combination with ADT.
High-risk non-metastatic castration-resistant prostate cancer (CRPC).
Metastatic CRPC who are asymptomatic or mildly symptomatic after failure of androgen deprivation therapy in whom chemotherapy is not yet clinically indicated. It is also indicated in adult men with metastatic CRPC whose disease has progressed on or after docetaxel therapy.
Important Safety Information
For important Safety Information for enzalutamide please see the full Summary of Product Characteristics at: View Source

About ARCHES
The company-sponsored, Phase 3, randomized, double-blind, placebo-controlled, multinational ARCHES trial (NCT02677896) enrolled 1,150 patients with mHSPC at sites in the U.S., Canada, Europe, South America, and the Asia-Pacific region. Patients in the trial were randomized to receive enzalutamide 160 mg daily or placebo and continued on a luteinizing hormone-releasing hormone (LHRH) agonist or antagonist or had a history of bilateral orchiectomy. The primary endpoint of the trial was radiographic progression-free survival (rPFS) assessed by blinded independent central review. rPFS was defined as the time from randomization to radiographic disease progression at any time or death within 24 weeks after study drug discontinuation. Radiographic disease progression was defined by identification of two or more new bone lesions on a bone scan with confirmation (Prostate Cancer Working Group 2 criteria) and/or progression in soft tissue disease. Patients were stratified by volume of disease (low vs high) and prior docetaxel therapy for prostate cancer (no prior docetaxel, 1-5 cycles, or 6 prior cycles).3

On May 4, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that it will release its first quarter 2021 financial results on Tuesday, May 11, after the close of the U.S. financial markets (Press release, Syndax, MAY 4, 2021, View Source [SID1234579045]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET on Tuesday, May 11, to discuss the Company’s financial results and provide a general business update.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website, www.syndax.com.

On May 4, 2021 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing novel, targeted therapies for rare and difficult to treat cancers, reported two collaboration agreements to support its process development and analytical chemistry activities for the cGMP manufacturing of Rhenium NanoLiposome (RNL), the Company’s lead investigational asset in clinical development for recurrent glioblastoma (Press release, Cytori Therapeutics, MAY 4, 2021, View Source [SID1234579073]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Plus Therapeutics signed a pre-clinical, clinical, and process development agreement with Invicro LLC (Invicro), a Konica Minolta company, a global provider of imaging biomarkers, core lab services, advanced analytics and software solutions for drug discovery and development with best-in-class expertise in radiochemistry. Under this agreement, Invicro will characterize the current manufacturing process and develop in-process manufacturing controls for the RNL active pharmaceutical ingredient (API) and final drug product and provide future clinical trial imaging support and drug development consulting.

In addition, Plus Therapeutics entered into an agreement with Eurofins BioPharma Inc. ("Eurofins"), a market leader in analytical chemistry for discovery pharmacology and advanced materials sciences. Eurofins will develop and validate test methods for purity, composition, and identity of Re-BMEDA, the API in RNL. These test methods will support release testing and compliance with cGMP requirements for new drug substances.

"Process optimization and appropriate quality controls of investigational compounds are very critical aspects in bringing novel drugs to markets," said Marc H. Hedrick M.D., President and Chief Executive Officer of Plus Therapeutics. "We have identified two best-in-class partners in Invicro and Eurofins to help us get one step further in bringing RNL to a registrational clinical trial and ultimately commercial supply."

As previously disclosed, Plus Therapeutics entered into a master services agreement with Piramal Pharma Solutions for the development, manufacture, and supply of RNL intermediate of the drug product.