Pro­teon trig­gers a stock im­plo­sion as its first PhI­II proves to be a bust

Steven Burke, Pro­teon

This morn­ing it was Pro­teon Ther­a­peu­tics’ $PRTO turn to join the queue of small biotechs to step on a land mine.

The Waltham, MA-based biotech’s shares cratered, plung­ing 70% in pre-mar­ket trad­ing af­ter the biotech re­port­ed that their Phase III tri­al of von­a­pan­i­tase (which used to be called PRT-201) failed the pri­ma­ry end­point on re­duc­ing the risk of what’s called pri­ma­ry unas­sist­ed pa­ten­cy — the length of time from fis­tu­la sur­gi­cal cre­ation to the first oc­cur­rence of a fis­tu­la throm­bo­sis or cor­rec­tive pro­ce­dure to re­store blood flow.

The drug was de­vel­oped to help pa­tients with chron­ic kid­ney dis­ease main­tain an open fis­tu­la to al­low ad­e­quate blood flow need­ed for he­modial­y­sis.

The re­searchers ze­roed in on signs of drug ac­tiv­i­ty, not­ing a 17% risk re­duc­tion on the pri­ma­ry end­point and so-called sug­ges­tions that the drug was work­ing for sec­ondary pa­ten­cy, the time to fi­nal fail­ure. And the com­pa­ny will look for some vin­di­ca­tion in an on­go­ing Phase III tri­al. But quite a few in­vestors don’t ap­pear ready to hang on for Round II.

More than 300 pa­tients were en­rolled in the first Phase III tri­al.

The mi­cro cap end­ed Mon­day with a mar­ket cap of $164 mil­lion, which large­ly went up in smoke.

“We are dis­ap­point­ed that the study missed the pri­ma­ry end­point. How­ev­er, it ap­pears that von­a­pan­i­tase had a drug ef­fect and we are en­cour­aged by the sec­ondary pa­ten­cy and fis­tu­la use for he­modial­y­sis find­ings in this tri­al, both of which we be­lieve are clin­i­cal­ly im­por­tant,” said Steven Burke, MD, Se­nior Vice Pres­i­dent and Chief Med­ical Of­fi­cer of Pro­teon Ther­a­peu­tics. “We plan to re­view the full da­ta set from PA­TEN­CY-1 and fur­ther in­ves­ti­gate these find­ings in our on­go­ing Phase 3 clin­i­cal tri­al, PA­TEN­CY-2.”

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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As­traZeneca gains EU nod for di­a­betes triple; Am­gen and Duke launch re­al-world PC­SK9 ob­ser­va­tion­al study

→ Weeks after winning EU approval to start marketing dapagliflozin as Forxiga, AstraZeneca has racked up another OK for a triplet combo involving the SGLT2 diabetes drug. Named Qtrilmet, the pill combines Forxiga with the DPP-4 inhibitor Onglyza (saxagliptin) and the bedrock drug metformin in a modified-release format. That 3-in-1 approach proved superior in reducing average blood glucose levels to a number of other dual combinations across 5 Phase III trials, including Forxiga plus metformin, Onglyza with metformin, or glimepiride with metformin.

Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is custom, an EMA panel on Friday issued its weekly recommendations on marketing applications submitted by drug developers. This week, the agency backed the use of five new therapies — including two Novartis drugs — but issued no negative reviews.

Novartis’ S1P drug for relapsing forms of multiple sclerosis (MS) drug, Mayzent (known chemically as siponimod), which was approved by the FDA in March — has been given the nod by the EMA. The Swiss drugmaker already sells its other MS drug, Gilenya, in both regions.

Atom­wise's X-37 spin­out gets $14.5 mil­lion to launch AI dis­cov­ery ef­forts

The folks behind Atomwise’s spinout X-37 like to think in cosmological metaphors, and you can think of their AI drug development model as probes sent into space from a central station. That station just got $14.5 million in Series A funding from DCVC Bio, Alpha Intelligence Capital and Hemi Ventures to back those missions.

X-37 uses Atomwise’s AI platform to identify drug targets and – unlike the parent company, which largely sticks to computers  – bring those into a wet lab and preclinical testing.  In addition to AI professionals, it’s led in by part by drug developers from Velocity Pharmaceutical Development.

Ab­bott Lab­o­ra­to­ries CEO Miles White pass­es ba­ton down to suc­ces­sor; Lon­za CEO Marc Funk hits the ex­it

→ Abbott Laboratories has named a successor to CEO Miles White after he announced that he was stepping down in March after 21 years of service. Robert Ford, the company’s COO and president, will take the helm. Ford is known for his work in the $25 billion merger between St. Jude Medical into Abbott in January 2017. White will remain with the company as executive chairman of the board. 

→ After snapping up Novartis’ Swiss facility, Novartis Center of Excellence, in July, Lonza has announced that their CEO, Marc Funk, is hitting the exit for “personal reasons.” Funk has been the CEO of the company for less than a year — brought onto the company back in March. In the meantime, chairman Albert Baehny will serve as interim CEO. 

BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: In a first, FDA green-lights use of a Chi­nese built can­cer ther­a­py — and more are com­ing

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

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