2 months ago
On August 13, Karyopharm Therapeutics (NASDAQ:KPTI) held its second-quarter earnings call, and the story split cleanly in two. The company laid out a myelofibrosis combination therapy with data strong enough to earn an accelerated approval pathway from the FDA, alongside a cash position that funds operations only into September. A $15.8 million loan payment lands September 10, and management admitted that paying it without new financing could trip a liquidity covenant and trigger default. Investors are being asked to weigh a genuinely differentiated drug against a genuinely short clock.
The bull case rests on selinexor paired with ruxolitinib, which Karyopharm plans to submit for FDA review this month under the Accelerated Approval Pathway for myelofibrosis. The Phase III SENTRY study found statistically significant, rapid, deep, and sustained spleen volume reductions, along with a preliminary overall survival signal and hints of disease modification. At week 24, the combination nearly doubled the spleen response rate versus ruxolitinib alone, with responses showing up as early as week 12 and holding through week 36 across patient subgroups. Even when ruxolitinib was dosed below 15 milligrams a day, the combination produced SVR35 rates as high as 50%, compared with zero for ruxolitinib alone, suggesting doctors could trim ruxolitinib without losing efficacy.
The overall survival hazard ratio at the time of the topline ******* ysis was 0.43. If cleared, this would be the first approved combination therapy in frontline myelofibrosis, a disease affecting roughly 20,000 people in the US with about 4,000 newly treated each year and no combination option on the market. Karyopharm pegs the peak US revenue opportunity at up to $1 billion and plans to lean on the commercial infrastructure it already runs for XPOVIO, including its KaryForward patient support program, to move quickly if approved. On the existing business, XPOVIO generated $30.8 million in U.S. net product revenue in the quarter, with demand holding roughly flat from a year earlier even as the treatment landscape gets more crowded.
The financial picture is where the caution belongs. Total revenue fell to $33.4 million from $37.9 million a year earlier, largely because Menarini's reimbursement of development costs ended in late 2025, a roughly $6.5 million swing. Net loss widened to $67 million from $37.3 million, and while some of that reflects non-cash mark-to-market adjustments tied to the company's financing structure, the cash balance tells its own story: $65.4 million in cash, equivalents, and investments at quarter end. Management said that funds current plans only through September 2026. A $15.8 million term loan payment is due September 10, and if it goes out without additional financing or a lender waiver, cash would fall below the $10 million minimum liquidity covenant, an event of default.
#september #cash #karyopharm #myelofibrosis
The bull case rests on selinexor paired with ruxolitinib, which Karyopharm plans to submit for FDA review this month under the Accelerated Approval Pathway for myelofibrosis. The Phase III SENTRY study found statistically significant, rapid, deep, and sustained spleen volume reductions, along with a preliminary overall survival signal and hints of disease modification. At week 24, the combination nearly doubled the spleen response rate versus ruxolitinib alone, with responses showing up as early as week 12 and holding through week 36 across patient subgroups. Even when ruxolitinib was dosed below 15 milligrams a day, the combination produced SVR35 rates as high as 50%, compared with zero for ruxolitinib alone, suggesting doctors could trim ruxolitinib without losing efficacy.
The overall survival hazard ratio at the time of the topline ******* ysis was 0.43. If cleared, this would be the first approved combination therapy in frontline myelofibrosis, a disease affecting roughly 20,000 people in the US with about 4,000 newly treated each year and no combination option on the market. Karyopharm pegs the peak US revenue opportunity at up to $1 billion and plans to lean on the commercial infrastructure it already runs for XPOVIO, including its KaryForward patient support program, to move quickly if approved. On the existing business, XPOVIO generated $30.8 million in U.S. net product revenue in the quarter, with demand holding roughly flat from a year earlier even as the treatment landscape gets more crowded.
The financial picture is where the caution belongs. Total revenue fell to $33.4 million from $37.9 million a year earlier, largely because Menarini's reimbursement of development costs ended in late 2025, a roughly $6.5 million swing. Net loss widened to $67 million from $37.3 million, and while some of that reflects non-cash mark-to-market adjustments tied to the company's financing structure, the cash balance tells its own story: $65.4 million in cash, equivalents, and investments at quarter end. Management said that funds current plans only through September 2026. A $15.8 million term loan payment is due September 10, and if it goes out without additional financing or a lender waiver, cash would fall below the $10 million minimum liquidity covenant, an event of default.
#september #cash #karyopharm #myelofibrosis
4 months ago
Ipsen agreed to acquire Kartos Therapeutics, a Redwood City, California-based clinical-stage biopharmaceutical company developing a treatment for myelofibrosis, a rare blood cancer, for $450 million upfront and up to $1.3 billion in additional milestone payments, bringing the total potential deal value to $1.75 billion.
According to Reuters, the acquisition centers on navtemadlin, an oral MDM2 inhibitor being evaluated as an add-on therapy to ruxolitinib, the current standard of care for myelofibrosis, in patients who have a suboptimal response to that treatment. The drug is currently in a Phase III trial called POIESIS, which is enrolling more than 600 patients across more than 250 sites globally. Top-line data from that trial are expected in 2027, the company said.
Ipsen CEO David Loew said in a statement that the deal "further strengthens our late-stage oncology pipeline" and that navtemadlin has the potential to become a new treatment option as early as 2028.
Myelofibrosis is a rare blood cancer in which the bone marrow becomes scarred, forcing blood production to shift to the spleen and causing significant symptoms including fatigue, night sweats, and progressive splenomegaly. Approximately 50% to 75% of patients on ruxolitinib discontinue treatment within three years, and median overall survival after discontinuation is roughly one to two years, the company said.
The milestone payments to Kartos shareholders include a significant regulatory approval payment and sales-based milestones, Ipsen said. The deal is expected to close by the end of the third quarter, subject to antitrust clearance under the Hart-Scott-Rodino Antitrust Improvements Act.
According to Reuters, the acquisition centers on navtemadlin, an oral MDM2 inhibitor being evaluated as an add-on therapy to ruxolitinib, the current standard of care for myelofibrosis, in patients who have a suboptimal response to that treatment. The drug is currently in a Phase III trial called POIESIS, which is enrolling more than 600 patients across more than 250 sites globally. Top-line data from that trial are expected in 2027, the company said.
Ipsen CEO David Loew said in a statement that the deal "further strengthens our late-stage oncology pipeline" and that navtemadlin has the potential to become a new treatment option as early as 2028.
Myelofibrosis is a rare blood cancer in which the bone marrow becomes scarred, forcing blood production to shift to the spleen and causing significant symptoms including fatigue, night sweats, and progressive splenomegaly. Approximately 50% to 75% of patients on ruxolitinib discontinue treatment within three years, and median overall survival after discontinuation is roughly one to two years, the company said.
The milestone payments to Kartos shareholders include a significant regulatory approval payment and sales-based milestones, Ipsen said. The deal is expected to close by the end of the third quarter, subject to antitrust clearance under the Hart-Scott-Rodino Antitrust Improvements Act.
4 months ago
Wilmington, Delaware-based Incyte Corporation (INCY) is a biopharmaceutical company that engages in the discovery, development, and commercialization of therapeutics in the United States and internationally. The company has a market cap of $19.6 billion and offers JAKAFI for the treatment of myelofibrosis, ICLUSIG, a kinase inhibitor for the treatment of chronic myeloid leukemia, and other products.
Companies with a market cap of $10 billion or more are typically referred to as "big-cap stocks." INCY fits right into that category, with its market cap exceeding this threshold, reflecting its substantial size and influence in the biotechnology industry.
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Companies with a market cap of $10 billion or more are typically referred to as "big-cap stocks." INCY fits right into that category, with its market cap exceeding this threshold, reflecting its substantial size and influence in the biotechnology industry.
Dear Microsoft Stock Fans, Mark Your Calendars for June 30
Dear Walmart Stock Fans, Mark Your Calendars for June 22
Nothing Seems to Be Going Right for Meta Platforms. How to Play META Stock Here.
4 months ago
Eli Lilly and Company (NYSE:LLY) is among the stocks with the best earnings growth for the next 3 years. On June 15, Eli Lilly and Company (NYSE:LLY) shared new data from the Phase 1 AJX-101 study. The study showed that an investigational type II JAK2 inhibitor delivered an "encouraging" safety profile, demonstrating clinical activity in patients living with myelofibrosis who have failed a type I JAK2 inhibitor.
The therapy is aimed at selectively binding the type II conformation of the JAK2 kinase, thus providing improved efficacy over current solutions. It will also offer a new option for patients who develop resistance to type I JAK2 inhibitors. This addition follows the completion of the acquisition.
Copyright: kadmy / 123RF Stock Photo
Earlier on June 9, Jefferies lifted the price target on Eli Lilly and Company (NYSE:LLY) to $1,350 from $1,330 and reiterated a Buy rating. This comes after the company reported full data from retatrutide's TRIUMPH-1 obesity study and TRANSCEND-T2D-1 study. Despite some safety concerns, which the firm believes are manageable, the retatrutide's overall profile "will be difficult to beat," the firm said. Indeed, LLY is among the stocks with the best earnings growth for the next 3 years.
Eli Lilly and Company (NYSE:LLY) is an Indiana-based company specializing in human pharmaceutical products. Founded in 1876, the company offers products for cardiometabolic health, oncology, immunology, and migraine prevention.
The therapy is aimed at selectively binding the type II conformation of the JAK2 kinase, thus providing improved efficacy over current solutions. It will also offer a new option for patients who develop resistance to type I JAK2 inhibitors. This addition follows the completion of the acquisition.
Copyright: kadmy / 123RF Stock Photo
Earlier on June 9, Jefferies lifted the price target on Eli Lilly and Company (NYSE:LLY) to $1,350 from $1,330 and reiterated a Buy rating. This comes after the company reported full data from retatrutide's TRIUMPH-1 obesity study and TRANSCEND-T2D-1 study. Despite some safety concerns, which the firm believes are manageable, the retatrutide's overall profile "will be difficult to beat," the firm said. Indeed, LLY is among the stocks with the best earnings growth for the next 3 years.
Eli Lilly and Company (NYSE:LLY) is an Indiana-based company specializing in human pharmaceutical products. Founded in 1876, the company offers products for cardiometabolic health, oncology, immunology, and migraine prevention.
