BCG Live(Organon & Co.)

Since yesterday, Protara Therapeutics\' stock has skyrocketed, jumping from $3.54 per share to about $6.75.\n At six months, Protara Therapeutics’ investigational cell therapy elicited a complete response (CR) rate of 72% among 18 patients with bladder cancer, data that sent the biotech’s shares surging.After nine months, two of out of three evaluable patients maintained their CRs, according to a Dec. 5 company release. In the phase 2 trial, no severe treatment-related adverse events had been reported by the data cutoff of Nov. 19.The open-label study, known as ADVANCED-2, enrolled patients with high-risk non-muscle invasive bladder cancer (NMIBC) who had not received or were unresponsive to Bacillus Calmette-Guérin (BCG), an immunotherapy used as the standard of care in the indication.The trial is testing Protara’s lead asset TARA-002, a cell therapy designed to activate immune cells within the tumor and generate a pro-inflammatory response. The data show that 13 of 18 patients (72%) across BCG exposures experienced CRs at six months, findings that are being shared today at the Society of Urologic Oncology’s annual meeting in Dallas. When zooming in on the patient population by BCG exposure, the pivotal cohort of BCG-unresponsive patients saw a 100% (4/4) CR rate at six months and an 80% (4/5) CR rate at any time. The cohort is designed to be registrational and aligns with FDA draft guidance for the specific patient population, according to Protara.In the proof-of-concept arm of BCG-naive patients, the CR rate was 64% (9/14) at six months and 67% (10/15) at any time.No patients discontinued due to adverse events, and there weren’t any treatment-related adverse events at or above a grade 2 level, the company said. In general, most adverse events were grade 1 and transient, according to the New York-based biotech, with the most common symptoms in line with typical responses to bacterial immunopotentiation. “These impressive TARA-002 results demonstrate meaningful activity in a difficult to treat patient population,” Brian Mazzarella, M.D., study investigator and vice president of research for Urology America, said in the release. “The activity of TARA-002 across BCG exposures, coupled with its ease of use and low procedural burden for physicians, make it an exciting potential treatment option for NMIBC patients.” The biotech is expected to report 12-month data from the trial in mid-2025, according to Protara CEO Jesse Shefferman.Since yesterday, Protara’s stock has skyrocketed, jumping from $3.54 per share at market close to $6.76 at 2:13 p.m. ET today.NMIBC makes up about 80% of bladder cancer cases, with around 65,000 Americans diagnosed with the form of bladder cancer every year.Earlier this year, ImmunityBio landed an FDA approval for its immunotherapy Anktiva in conjunction with BCG for BCG-unresponsive patients.Pharma data intelligence outlet Evaluate recently listed the interleukin-15 superagonist as one of the top 10 drug launches expected this year, forecasting that the immunotherapy could reach nearly $900 million in 2028 sales. The standard-of-care BCG is a vaccine supplied to the U.S. by Merck & Co. The med is used as to protect against tuberculosis and to stop or slow bladder cancer\'s growth.

In recent developments, ImmunityBio has gained FDA approval for its immunotherapy drug, Anktiva, in combination with Bacillus Calmette-Guérin (BCG) for the treatment of non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS). This approval sets the stage for ImmunityBio to directly compete with Merck's heavyweight drug Keytruda and Ferring Pharmaceuticals' gene therapy drug Adstiladrin, both of which are administered without BCG. However, the global shortage of BCG, with Merck being the sole supplier in the United States, poses a significant challenge. The Global BCG Market: According to a research report by GlobalInfoResearch, there are currently seven companies worldwide that produce BCG: Merck, Sanofi Pasteur, Japan BCG Laboratory, China National Biotec Group, Serum Institute of India, Intervax, and GSBPL. Among them, Merck, Japan BCG Laboratory, China National Biotec Group, Serum Institute of India, and GSBPL are the remaining manufacturers of intravesical BCG. After facing production issues in 2012, Sanofi officially exited the market, leaving Merck as the global primary supplier of BCG. Merck's BCG Supply Strategy: Since 2019, Merck has been implementing a controlled supply of BCG, allocating limited quantities to distributors and wholesalers based on historical purchasing patterns rather than directly supplying individual healthcare providers. In October 2020, Merck announced plans to construct a new TICE BCG production facility in North Carolina, with an expected completion date between late 2025 and late 2026. Once operational, Merck anticipates doubling BCG production capacity to meet future demand. ImmunityBio's Alternative Approach: Although ImmunityBio faces direct competition from established rivals, there is a silver lining. The FDA approval for Anktiva does not specify or restrict the type of BCG used in conjunction with the drug. This allows ImmunityBio to explore alternative options to Merck's TICE BCG. The California-based biotechnology company has entered into an exclusive agreement with the Serum Institute of India (SII), the world's largest manufacturer of BCG, to produce BCG globally. The details of the agreement, including pricing and terms, have not been disclosed. SII's BCG Products: SII offers two BCG products for cancer treatment. The first is a traditional BCG known as Onco-BCG, which has already been commercialized outside the United States. The second product is a recombinant BCG currently undergoing mid-term trials in Europe. This version incorporates two gene modifications to enhance its immunogenicity under CD8+ and CD4+ stimulation, while improving safety compared to standard BCG. Future Prospects: Both ImmunityBio and SII are working diligently to expedite the second phase of clinical trials. However, the timeline for commercial supply remains uncertain in both cases. ImmunityBio has expressed confidence that once the trial protocol is submitted to the FDA, a clearer timeline will emerge. The collaboration between ImmunityBio and SII marks a significant step towards addressing the chronic shortage of BCG supply. By diversifying the sources of BCG production, ImmunityBio aims to ensure a long-term solution for the treatment of NMIBC with Anktiva. With SII's expertise and global reach, the partnership holds promise in meeting the demand for BCG and advancing cancer therapies.