Gigantism

Pictured: Blood sample for insulin-like growth fac The biotech Tuesday reported that 56% of patients taking paltusotine in a late-stage trial achieved the primary endpoint of IGF-1 levels for their rare hormonal disorder. An NDA submission is slated for later this year with a launch anticipated in 2025. Pictured: Blood sample for insulin-like growth factor-1 (IGF-1)/iStock, Jarun011 Crinetics Pharmaceuticals has secured another late-stage win as its oral, once-daily investigational drug paltusotine aced a second Phase III study for acromegaly, a rare hormonal disorder. The biotech’s stock shot up over 10% Tuesday morning following the announcement . In the late-stage trial, 56% of patients taking paltusotine achieved the primary endpoint for the insulin-like growth factor 1 (IGF-1) level compared to 5% on placebo. The treatment also helped reduce symptoms of the disease—a secondary endpoint of the study. No serious adverse events were reported. Acromegaly, sometimes referred to as gigantism, is a serious hormonal disorder caused by a benign tumor in the pituitary gland that secretes growth hormone. Over 25,000 patients in the U.S. suffer from the rare disease, which results in enlargement of face, hands and feet. Left untreated, it can cause type 2 diabetes, hypertension, heart disease and other serious complications. Surgical removal of the tumor is the preferred treatment but many patients are not candidates for surgery. Current pharmacological options include painful monthly injections or Pfizer’s subcutaneous daily injection of Somavert. The Phase III data are the second positive trial results for Crinetics in acromegaly. In September 2023, the biotech shared data for a study of paltusotine in patients who switched from their current injections to Crinetics’ pill. Among treated patients, 83% were able to maintain their IGF-1 levels within the upper limit of normal, compared to only 4% of those on placebo. With these data in hand, Crinetics plans to submit its New Drug Application to the FDA in the second half of this year, while prepping for a 2025 launch. Jefferies analysts believe the market opportunity could be around $300 million for the company. Crinetics is also testing the drug candidate in carcinoid syndrome, reporting positive Phase II topline results last week. Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn .

Pictured: Blood sample for insulin-like growth factor-1 (IGF-1)/iStock, Jarun011 Crinetics Pharmaceuticals has secured another late-stage win as its oral, once-daily investigational drug paltusotine aced a second Phase III study for acromegaly, a rare hormonal disorder. The biotech’s stock shot up over 10% Tuesday morning following the announcement. In the late-stage trial, 56% of patients taking paltusotine achieved the primary endpoint for the insulin-like growth factor 1 (IGF-1) level compared to 5% on placebo. The treatment also helped reduce symptoms of the disease—a secondary endpoint of the study. No serious adverse events were reported. Acromegaly, sometimes referred to as gigantism, is a serious hormonal disorder caused by a benign tumor in the pituitary gland that secretes growth hormone. Over 25,000 patients in the U.S. suffer from the rare disease, which results in enlargement of face, hands and feet. Left untreated, it can cause type 2 diabetes, hypertension, heart disease and other serious complications. Surgical removal of the tumor is the preferred treatment but many patients are not candidates for surgery. Current pharmacological options include painful monthly injections or Pfizer’s subcutaneous daily injection of Somavert. The Phase III data are the second positive trial results for Crinetics in acromegaly. In September 2023, the biotech shared data for a study of paltusotine in patients who switched from their current injections to Crinetics’ pill. Among treated patients, 83% were able to maintain their IGF-1 levels within the upper limit of normal, compared to only 4% of those on placebo. With these data in hand, Crinetics plans to submit its New Drug Application to the FDA in the second half of this year, while prepping for a 2025 launch. Jefferies analysts believe the market opportunity could be around $300 million for the company. Crinetics is also testing the drug candidate in carcinoid syndrome, reporting positive Phase II topline results last week. Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.

March 9, 2015 By Riley McDermid , BioSpace.com Breaking News Sr. Editor Boston area rare disease startup Chiasma said late Monday that it will use a new round of funding to open new, larger headquarters in the Newton or Cambridge area in coming months, the Boston Business Journal reported. CEO Roni Mamluk told the paper that the $70 million Series E financing round it closed last week will now be used to expand its operations in Newton or Cambridge, while maintaining a development facility in Israel. Chiasma also said in a statement that it will use the funding “to build its sales and marketing capabilities and prepare for the U.S. commercial launch of its lead product,” an acromegaly drug that it hopes to submit to the U.S. Food and Drug Administration for approval by June. Last week’s round included investors Rock Springs Capital and Sofinnova Ventures , prior investors MPM Capital , F2 Capital , 7 Med Health Ventures , Abingworth and ARCH Venture Partners , and an undisclosed blue chip public investment fund. That came as welcome news less than a year after global behemoth Roche AG cancelled a commercial agreement with the Israeli startup for a product for treatment of acromegaly (gigantism) because the product did not meet its clinical trial criteria. That year-old deal should have generated $600 million for Chiasma’s investors, but only brought in about $65 million advance and some milestone payments before the cancellation. Roche ( RHHBY ) said in August that while the Phase III trial results were good, it would opt out of the deal “after receiving additional information about the trial, and after further consultation with the regulatory authorities.” Now, however, Chiasma is forging ahead. It said the money would immediately be ploughed into the U.S. commercial launch of its lead product, octreotide capsules for adults with acromegaly, a rare endocrine disease, which could happen as soon as 2016. “The commitment from both new and existing investors provide us with the resources to advance our regulatory efforts, prepare for a successful launch of octreotide capsules, test oral octreotide capsules for additional indications and further invest in earlier-stage TPE programs that can fuel our growth over the long term,” said Mamluk, chief executive officer of Chiasma. “With this financing in place, we are well positioned to advance a portfolio of oral drugs that address unmet needs in orphan indications.” It said it remains on track to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for octreotide capsules in the second quarter of 2015. If approved, the drug will be the first and only oral somatostatin analog, which will be a relief to patients who can currently only receive the therapy via injection. “Acromegaly typically develops when a benign tumor of the pituitary gland produces too much growth hormone (GH), ultimately leading to significant health problems and early death if untreated. Common features of acromegaly are facial changes, intense headaches, joint pain, impaired vision and enlargement of the hands, feet and tongue,” said Chiasma. Diabetes, hypertension, cardiac and cerebrovascular disease and respiratory disorders are all associated with the disease, which can sometimes require surgery or radiation to remove the tumor. “Medical treatments available currently include dopamine agonists, GH antagonists and injectable somatostatin analogs, the current standard of care,” said Chiasma. “Currently available somatostatin analogs require large-bore needles, for injection into muscle (octreotide) or deep into the tissue underlying the skin (lanreotide).” There is no question that Chiasma is a scrappy, long-term fighter. After a massive restructuring in 2006, the company was then hit hard by the recession, but managed to right itself after bringing in former BioMarin Pharmaceutical Inc. ( BMRN ) exec Fredric Price as chairman and CEO. Mamluk eventually replaced Price but kept the startup pointed in the right direction. It also has been no slouch in the venture capital raising department. Previous financings for the 10-year-old company include $38.5 million for a Series D in July 2012 from MPM , ARCH and Abingworth , three debt financings totaling 15.8 million from 2008 to 2011; a $44 million Series C from Ofer Hi-Tech, ARCH, MPM and F2; and a $7.7 million Series B from Israel investors Ofer Hi-Tech , Yissum Research Development , Jerusalem Global Ventures and F2 . BioSpace Temperature Poll Vertex Pharmaceuticals made news last week when it terminated leases on three properties in Cambridge, Mass, that freed up 313,000 square feet of space in the Genetown area . The company has spent a significant part of 2014 consolidating its operations on the South Boston waterfront, leasing 291,000 square feet of office space at West Kendall Street in Cambridge’s Kendall Square. So we wanted to ask the BioSpace community: Is Boston going to be getting more biotech leases anytime soon, or fewer tenants? var _polldaddy = [] || _polldaddy; _polldaddy.push( { type: "iframe", auto: "1", domain: "biospace.polldaddy.com/s/", id: "after-vertex-pulls-up-stakes-will-boston-see-a-biotech-exodus", placeholder: "pd_1425682894" } ); (function(d,c,j){if(!document.getElementById(j)){var pd=d.createElement(c),s;pd.id=j;pd.src=(' '==document.location.protocol)?' ':' ';s=document.getElementsByTagName(c)[0];s.parentNode.insertBefore(pd,s);}}(document,'script','pd-embed'));